Biotechnology Focus June/July 2016

INSIGHTS FOR THE LIFE SCIENCE INDUSTRY

JUNE/JULY 2016 VOLUME 19, NUMBER 3

Partnering for Success:

Why Ontario’s competitive advantage is scientific excellence and a government that’s willing to

back it!

Publication Mail Registration Number: 40052410

BUILD I RENOVATE I EXPAND I MOVE

LAB SET-UP GUIDE THE COMPLETE CHECKLIST FOR SUCCESSFUL SET-UP

When Science Calls, We Deliver.

VWR can guide you through the entire lab set-up process, from project planning to completion. We make sure your new lab is set-up, stocked, and optimized for productivity when you arrive.

Get started today by requesting your 2016 Lab Set-Up Guide at vwr.com/labsetup.

Our 2016 guide includes 74 money-saving offers from leading brands, including our VWR Collection, totaling up to $62,500 in savings. You’ll also find new lab checklists and helpful information about our customer service teams, VWRCATALYST Laboratory Services, VWR Furniture, Sustainability with VWR, and more.

The VWR Collection offers the right products, at the right price, when you need them... with a name you trust. In your lab, you face the challenge of balancing the need to stretch your dollars with the need for quality and innovation. The VWR Collection helps you do both.

Be sure to ask about our New Lab Welcome Kit! • VWR Collection product samples • Fun giveaways • More resources for your new lab

Request your 2016 Lab Set-Up Guide!

Contact your VWR Sales Representative to learn more about starting your new lab with VWR.

Need Support with Cell Biology?

VWR offers complete solutions for your Cell Biology Workflow, including technical articles, selector guides, and exclusive limited time offers!. Read more at vwr.com/aynsolutions

contents

June/July 2016 – VOLUME 19 – NUMBER 3

FEATURES 10

OBIO’s “Innovation In Action” draws crowds at Queen’s Park

21

Highlights from a special day where OBIO got to showcase innovative Ontario bioscience companies and raise awareness around the challenges facing the sector

By Gail Garland

11

The Pace of Progress in Heart Regeneration

Compiled by Shawn Lawrence

25

13

Leading Change for Ontario’s Med Tech Sector

By Jason Field

26

18

JLABS comes to Toronto Ontario’s bioscience startups have a new place to call home as Johnson & Johnson Innovation has opened the doors to JLABS @ Toronto

The BRIDGE@CCRM: Where cell and gene therapies go mainstream Expectations are high that this new facility will usher in a new era of industrialization in the regenerative medicine field.

In this special Q&A, Bill Charnetski, Ontario’s first chief innovation strategist details his plans for Ontario’s healthcare innovation sector Compiled by Shawn Lawrence

Championing our success Life Sciences Ontario president Jason Field says in his column that it’s time we get loud and proud about Ontario life science innovations. We couldn’t agree more

Dr. Michael Laflamme is mixing old and new technologies to piece together the right combination of stem cells needed to repair the damaged heart

By Lisa Willemse

In conversation with the Minister As we’ve traditionally done in year’s past, we go one-on-one with Ontario’s Minister of Research and Innovation Reza Moridi to discuss the state of Ontario’s bioscience sector

By Shawn Lawrence

30

The Last Word Capitalizing on Canada’s contagiously creative life science sector and building a constellation of homegrown stars Raphael Hofstein and Elizabeth Monier-Williams

By Shawn Lawrence

www.biotechnologyfocus.ca

June/July 2016 BIOTECHNOLOGY FOCUS 3

PUBLISHER’S note PUBLISHER/ EDITOR-IN-CHIEF SENIOR WRITER CONTRIBUTING WRITERS

Public Private partnerships: Ontario puts its best foot forward It’s been a year of tremendous growth and change for the Ontario bioscience industry.

Likewise, we’ve also witnessed a remarkable transformation of the once struggling 20-storey MaRS Phase 2 building. It may be premature to say it has shed its ‘white elephant’ reputation, but more and more it is becoming a staging ground for sector growth and bringing world class companies to Ontario.

Flip through the pages of this issue and you’ll see exactly what we mean.

For starters, in January there were two significant funding announcements around regenerative medicine. The first, a partnership between the Government of Canada and GE Healthcare with each chipping in $20 million towards the creation of a new Centre for Advanced Therapeutic Cell Technologies at CCRM. Senior writer Shawn Lawrence’s piece on the BridGE@CCRM details how this new facility, that is located at the MaRS tower, intends to solve manufacturing challenges in the regenerative medicine space.

The second funding announcement saw the Ontario Government invest $25 million into the Ontario Institute for Regenerative Medicine (OIRM). This funding will support 150 leading stem cell and regenerative medicine scientists, clinicians and engineers in the province. Among the scientists is Dr. Michael Laflamme whose work using stem cells to repair hearts is featured in Lisa Willemse’s contributed article: The Pace of Progress in Heart Regeneration.

In further funding news, the Ontario Government in March partnered with Baylis Medical Company, investing $4.22 million through its Jobs and Prosperity Fund to help expand the company’s Mississauga R&D manufacturing plant. This expansion will allow for the near doubling of the company’s exports over the next seven years, while creating 84 new jobs.

The activity culminated with a huge celebratory event on May 11, as Ontario was finally introduced to Johnson & Johnson Innovation’s new JLABS @ Toronto incubator. Like the BridGE@CCRM, JLABS @ Toronto has taken up residence in the MaRS tower. Already, 22 emerging Ontario life science startup companies call it home. Be sure to check out our spotlight piece to see why this new facility is a real ‘game-changer’ to the local life science start-up scene.

In terms of our other stories, once again we go one-on-one with Ontario’s Minister of Research and Innovation Reza Moridi. Likewise in separate Q&A, we introduce you to Ontario’s new Chief Health Innovation Strategist Bill Charnetski He details why Ontario is a great place for health technology innovation and his vision for the province’s burgeoning medtech sector.

These are just some of the many stories we’ve packed into this issue. We hope you enjoy it.

4 BIOTECHNOLOGY FOCUS June/July 2016

Terri Pavelic Shawn Lawrence Gail Garland

Elizabeth Monier-Williams

Jason Field

Lisa Willemse

Raphael Hofstein

Director, Content & Business Development

José Labao

GRAPHIC DESIGNER CONTROLLER MARKETING MANAGER

Elena Pankova John R. Jones Mary Malofy

CIRCULATION DIRECTOR Mary Labao circulation@promotive.net Tel: 905-841-7389

EDITORIAL ADVISORY BOARD Christine Beyaert, Cohn&Wolfe; Pierre Bourassa, IRAP, Montréal; Murray McLaughlin, Sustainable Chemistry Alliance; Ulli Krull, UTM; John Kelly, KeliRo Company Inc.; Peter Pekos, Dalton Pharma Services; Brad Thompson, Oncolytics; Robert Foldes, Viteava Pharmaceuticals Inc.; Gail Garland, OBIO; Barry Gee, CDRD; Bonnie Kuehl, Scientific Insights Consulting Group Inc.; Raphael Hofstein, MaRS Innovation; Roberto Bellini, Bellus Health; Peter van der Velden, Lumira Capital; Albert Friesen, Medicure Inc.; Ali Tehrani, Zymeworks Inc.

Biotechnology Focus is published 6 times per year by Promotive Communications Inc. 23-4 Vata Court, Aurora, Ontario L4G 4B6 Phone 905-727-3875 Fax 905-727-4428 www.biotechnologyfocus.ca E-mail: biotechnology_focus@promotive.net Subscription rate in Canada $35/year; USA $60/year; other countries $100/year. All rights reserved. No part of this publication may be reproduced without written consent. Publications Mail Registration Number: 40052410 Return undeliverable Canadian addresses to: circulation dept – 23-4 Vata Court, Aurora, Ontario L4G 4B6 National Library of Canada ISSN 1486-3138 \ All opinions expressed herein are those of the contributors and do not necessarily reflect the views of the publisher or any person or organization associated with the magazine.

If you would like to order hard copy or electronic reprints of articles, contact sales@promotive.net

Transforming Ontario’s oncology innovations into commercial opportunities.

The Fight Against Cancer Innovation Trust (FACIT) is the gateway to the cancer research pipeline in Ontario, bringing together communities in the public and private sectors. We take the most promising ideas and discoveries in the fight against cancer and turn them into profitable opportunities that benefit patients, researchers, investors and our economy.

— To learn more visit facit.ca or contact info@facit.ca

FACIT is an independent business trust established by the Ontario Institute for Cancer Research (OICR) to undertake development and commercialization activities for Ontario assets related to cancer research, technologies, products and drug discovery.

R&D news Canada’s top student scientist recognized at Sanofi Biogenius Canada national final Iveta Demirova, 16-year-old, grade 11 student from New Westminster Secondary School in British Columbia, wins first place at the national final of the Sanofi Biogenius Canada (SBC) competition, which took place in Ottawa on May 2 and 3 (CNW Group/Sanofi Canada).

OTTAWA, ON – Iveta Demirova from New Westminster Secondary School in New Westminster, BC has been awarded top honours at the national final of the prestigious Sanofi Biogenius Canada (SBC) competition in Ottawa.

The 16-year-old, grade 11 student, was chosen by the judges for her research project exploring the development of a novel HIV-1 therapy.

The results of Iveta’s research project, completed with the support of mentor Dr. Ralph Pantophlet of Simon Fraser University, could offer numerous advantages to those living with HIV, which remains one of the world’s leading infectious diseases.

Although current treatment options have managed to successfully target and suppress the virus among patients, many individuals become resistant to treatment, and it is among this population that Iveta’s project could play a significant role.

“I was very pleased with the results of my research and I am hoping that my findings will have an impact within the field of HIV research, and, more importantly, in the lives of patients living with this disease,” she said. “I am truly honoured to have won the Sanofi Biogenius Canada competition, and thrilled to have the opportunity to rep6 BIOTECHNOLOGY FOCUS June/July 2016

resent the country at the 2016 International BioGENEius Challenge in San Francisco in June.”

One of the country’s most prestigious student competitions, Sanofi Biogenius Canada pairs exceptional young scientists at the high school level with academic mentors to pursue real-world research projects.

These enriching partnerships have resulted in many promising breakthroughs across various scientific fields.

Hosted at the National Research Council of Canada (NRC), the competition’s national final featured the winners of all nine Sanofi Biogenius Canada regional competitions across the country. The nine finalists presented their research projects to a judging panel of esteemed members of the scientific community, including Dr. Robert Tsushima, associate dean, Research and Partnerships, Faculty of Science, York University; Dr. Thomas Merritt, Canada Research chair in Genomics and Bioinformatics, Laurentian University; Dr. Lakshmi Krishnan, program leader, NRC and Michael McCluskie, senior research officer, NRC. “What distinguishes Sanofi Biogenius Canada from other science competitions is the real-life experience that participants gain by working in close collaboration with a mentor,” said Mark Lievonen, Canada Country chair, Sanofi and general manager, Sanofi Pasteur. “There is nothing more inspiring than having the opportunity to pursue groundbreaking research projects with experts and leaders in their respective fields. Not only is the depth of Iveta’s research project commendable, but it is a concrete example of the benefits of programs like SBC, which help transform passion into tangible results.”

Iveta receives a cash prize of $5,000, a portion of which will go to New Westminster Secondary School, and she will now progress to the 2016 International BioGENEius Challenge in San Francisco in June, where she will submit her work to a panel of pre-eminent international scientists.

Runners-up in this year’s national SBC competition were awarded cash prizes ranging in value from $1,000 to $4,000.

Second prize went to Melody Song, a student from Evan Hardy Collegiate in Saskatoon, SK. Melody earned high praise for her research project which focused on preventing disease in Faba bean crops, and was completed with the support of mentors Dr. Kirstin E. Bett and Dr. Hamid Khasaei of the University of Saskatchewan.

Third prize was awarded to Denis Drewnik, a grade 12 student from Sisler High School in Winnipeg, MB, for a project that examined how to protect the canola crop from the devastating Blackleg disease, which can reduce yield by up to 20 per cent and have a devastating economic impact. Denis’ project was supported by mentor Dr. Mark Belmonte of the University of Manitoba.

Dina Shehata from Holy Heart of Mary High School in St. John’s, NL earned the competition’s Commercialization prize.

The Commercialization prize recognizes the project with the most commercial potential and viability. Dina’s research focused on developing a low cost gel model for ultrasound training.

To see this story online visit http://biotechnologyfocus.ca/canadastop-student-scientist-recognized-sanofibiogenius-canada-national-final-2/

McMaster scientists uncover new way to grow rare life-saving blood stem cells HAMILTON, ON – Researchers at McMaster University’s Stem Cell and Cancer Research Institute have made significant steps forward in understanding the stem cells of the human blood system after discovering how a key protein allows for better control and regeneration of these cells.

This discovery, published in the scientific journal Nature, illustrates how a protein called Musashi-2 regulates the function and development of important blood stem cells.

This knowledge provides new strategies that can be used to control the growth of these cells — cells that can be used as therapeutics for a range of life-threatening diseases but are, in general, in very short supply.

The senior author is Kristin Hope, principal

R&D news

investigator at the Stem Cell and Cancer Research Institute and assistant professor with McMaster University’s Department of Biochemistry and Biomedical Sciences. The research also involved collaborators from the University of California San Diego, University of Toronto and the University of Montréal.

Hope says the discovery could be impactful for the tens of thousands of patients suffering from a range of blood-based disorders including leukemia, lymphoma, aplastic anemia, sickle cell disease and more.

“We’ve really shone a light on the way these stem cells work. We now understand how they operate at a completely new level, and that provides us with a serious advantage in determining how to maximize these stem cells in therapeutics.”

The research team specifically looked at stem cells from umbilical cord blood, a proven but under-utilized source of stem cells for the treatment of adult blood cancers. These stem cells have the potential to become an important therapeutic for the thousands of people suffering from blood cancers who are awaiting life-saving transplants.

Cells from umbilical cord blood have unique properties that make them easier to use for transplantation, including accessibility and adaptability. As a result, they allow for safer and more effective transplants.

The problem, Hope points out, is that there are very few stem cells available in individual cord blood samples — only about five per cent of all samples actually contain enough cells for a transplant. The team’s research into the importance of Musashi-2 and its role in expanding the number of stem cells in a given cord blood sample could help ease the current stem cell shortages.

Gene Yeo, associate professor at the Uni-

versity of California San Diego, co-corresponding author of the study, adds, “Most stem cell studies focus on proteins that bind DNA to control gene output. The prominent role we found for Musashi-2, a protein that instead binds to RNA, also underscores an urgency to study this second layer of gene regulation in stem cells.”

Hope says: “Providing enhanced numbers of stem cells for transplantation could alleviate some of the current post-transplantation complications and allow for faster recoveries, in turn reducing overall health care costs and wait times for newly diagnosed patients seeking treatment.”

“By expanding the stem cells as we have done, many more donated samples could now be used for transplants.”

The study was supported by the Ontario Institute for Cancer Research, the Canadian Institutes of Health Research, Canadian Blood Services, Health Canada, the National Institutes of Health and the California Institute for Regenerative Medicine.

To see this story online visit http://biotechnologyfocus.ca/mcmaster-scientists-uncover-new-way-growrare-life-saving-blood-stem-cells/

June/July 2016 BIOTECHNOLOGY FOCUS 7

BUSINESS corner Fonds de solidarité FTQ invests US$7.5 million in DalCor for ACS Phase 3 trials

MONTRÉAL, QC – The Fonds de solidarité FTQ is investing US$ 7.5 million in DalCor Pharmaceuticals for its Phase 3 trials of dalcetrapib in genetically distinct

patients with Acute Coronary Syndrome or ACS. DalCor was founded by Sanderling Ventures with the support of Québecbased investors.

“Our strategic approach in this sector continues to deliver results”, said Alain Denis, senior vice-president, Innovation, for the Fonds de solidarité FTQ. “By investing in funds such as Sanderling Ventures VII, we are attracting them to Québec and increasing the availability of capital. Banking on world-class research talent as well as research infrastructures and services, we are helping to strengthen Qué-

bec’s life sciences industry.”

“The work done by a team of researchers at the Montréal Heart Institute, led by Dr Jean-Claude Tardif and Dr Marie-Pierre Dubé, has shown that innovation based on research and precision medicine can represent renewed hope for patients suffering from diseases such as ACS,” he continued.

In recent years, the Fonds has namely invested in DalCor, Inception IBD, enGene, Milestone, Esperas, PRCL Recherche, Gladius, Thrasos, GlyPharma and Zymerworks.

Since 2012, the Fonds also invested in funds such as AmorChem II, CTI II, Forbion, Lumira, Versant V and Sanderling VII. To see this story online visit http://biotechnologyfocus.ca/ fonds-de-solidarite-ftq-invests-us7-5-million-dalcor-acs-phase-3-trials/

OBIO launches report on building Canada’s health science industry of the future TORONTO, ON – The Ontario Bioscience Innovation Organization (OBIO®) has released its report “How Canada Should be Engaging in a $9 Trillion Dollar Health Economy.” The report is the output of interviews and surveys of 125 CEOs of Canada’s health science companies looking at the future of the global industry and opportunities for Ontario and Canada.

The report makes recommendations to ensure Canadians derive the economic and patient benefits from competing successfully on the world stage. Hosted by Synaptive Medical, the launch was attended by over 50 health science industry leaders.

“In late 2015 and early 2016, Canada’s health science industry CEOs, shared their optimism, concerns and ideas with OBIO to ensure Canada can compete for a piece of the $9 trillion global health science economy,” said Gail Garland, president and CEO of OBIO. “Now we are pleased to share these ideas and our recommendations. Winning global jurisdictions have made the human health industry a priority and have appropriate policies and resources in place to ensure competitive success. A sustainable, successful Canadian Health Science industry represents significant economic potential 8 BIOTECHNOLOGY FOCUS June/July 2016

for Canada’s economy and return on investment in research. It is the path to ensure innovations help patients and can be used to reduce healthcare costs.”

Overview of the Report: • The potential to offset growing demand and rising costs through commercial enterprise and exports, represents a compelling reason for a strong healthcare industry.

• Canada has yet to identify and implement an industrial policy for a commercial, globally competitive, healthcare industry that will bring us into the future.

• Currently, Canada is reasonably competitive (but not differentiated) with its well educated workforce, tax policies, clinical trial capabilities and the quality of the federal regulatory agency.

• Canada has fallen behind on a number of metrics that are of concern if we are to compete in the global healthcare industry of the future. Key among these are: cost of research and doing business, speed and agility, health data infrastructure, innovative market access processes, an experienced labour force and access to capital for commercial purposes.

The report’s key recommendations focus on: 1. Creating an environment where health science innovations can thrive through market access and procurement policies.

2. Access to capital and supportive tax policies for companies to scale up and grow.

3. Access to experienced industry talent.

To see this story online visit http://biotechnologyfocus.ca/obiolaunches-report-building-canadas-healthscience-industry-future/

BUSINESS corner

Zymeworks lands second major antibody development partnership with GSK VANCOUVER, BC – Zymeworks Inc. has struck a second partnership deal with pharmaceutical heavyweight GlaxoSmithKline (GSK), for the research, development and commercialization of bispecific antibodies.

Under the agreement, GSK will now have access to its second Zymeworks platform technology, Azymetric™. The platform will be employed to develop new multiple bi-specific drugs across different disease areas.

Zymeworks will receive upfront and preclinical payments of up to US$36 million and is eligible to receive up to US$152 million in development and clinical milestone payments, along with commercial sales milestone payments of up to US$720 million, and tiered royalties on potential sales.

As previously announced in December 2015, Zymeworks and GSK entered into a collaboration and license agreement

to further develop Zymeworks’ Effector Function Enhancement and Control Technology (EFECT™) platform and to research, develop, and commercialize novel Fc-engineered monoclonal and bi-specific antibody therapeutics that have been optimized for specific therapeutic effects.

As part of this second agreement, GSK has also gained the right to combine the Azymetric™ platform with novel engineered Fc domains developed under the previously announced collaboration.

“We are excited to be expanding our relationship with GSK to include our Azymetric™ bi-specific platform.” said Dr. Ali Tehrani, president and CEO of Zymeworks.

“We view this new collaboration as evidence of our valuable role as a partner and the strength of our proprietary drug development platforms.”

Tehrani says that proceeds from this collaboration will be used to advance Zymeworks’ own pipeline of therapeutic candidates, including the Azymetric™ antibody ZW25 and the Azymetric™ antibody drug conjugate ZW33, into human clinical trials this year.

To see this story online visit http://biotechnologyfocus.ca/zymeworks-gsk-team-antibody-collaboration/

Keynote Speaker: ROBIN FARMANFARMAIAN SUMMIT 2016 YORK REGION

Exponential Health – Transforming Health Care

VP of INVICTA Medical, Faculty of Singularity University Other topics include: • Thought Leadership • Synthetic Biology and Biohacking • Future of Exponential Technologies • Lightning Round Innovations

MedEdge AD

Tuesday, June 14 • 8 a.m. to 2 p.m.

Richmond Hill Centre for the Performing Arts 10268 Yonge Street, Richmond Hill

Register now!

Learn, network and grow your business. Be part of the innovation revolution.

BusinessRichmondHill.ca/MedEdge Follow the conversation on

#MedEdge

June/July 2016 BIOTECHNOLOGY FOCUS 9

Special report

By Gail Garland, CEO, Ontario Bioscience Innovation Organization

ONTARIO

OBIO’s ‘Innovation in Action’ Draws Crowds at Queen’s Park On April 11, 2016: Ontario Bioscience Innovation Organization (OBIO®) hosted its annual Queen’s Park Advocacy Day attracting an impressive 150 attendees including 50 elected officials.

High profile meetings with the Premier and key Ministers took place throughout the day followed by a reception which raised awareness of the health science industry and showcased 15 health science companies including several from among OBIO’s CAAP entrepreneurs. Politicians from all Parties interacted with innovative health technologies being developed in Ontario and engaged with over 60 company representatives.

OBIO’s All-Party MPP Health Science Caucus was on hand to share their enthusiasm for the industry and support the sector.

The theme for the day grew out of the Ontario Highlights of OBIO’s 2016 report “How Canada Should be Engaging in a $9 Trillion Dollar Health Economy”. OBIO consulted with 125 CEOs from the human health industry on the future of the global healthcare economy, Canada’s opportunities and ideas to ensure our industry is competitive, as well as innovative. Winning global jurisdictions/environments were characterized by their market opportunities and processes, agility, expertise and science and by data and analytics infrastructure accelerating discovery, development and commercialization.

Currently, Canada and Ontario have competitive, well educated workforce, tax policies, clinical trial capabilities and federal regulatory agency but we need to pay attention in other areas.

OBIO’s policy recommendations address procurement and market access issues, capital for Ontario companies, globally competitive tax policies, building an experienced workforce through job creation and revisions to Government programs.

The potential to offset growing demand and rising health care costs through commercial enterprise and exports, represents a compelling reason for a strong healthcare industry.

Further, the commercialization of health technology is an opportunity to diversify Canada’s economy, create quality jobs and to ensure our highly educated workforce can stay here and build a future. OBIO delivered this message on behalf of industry to a receptive audience at Queen’s Park.

“OBIO’s Provincial Advocacy Day was the first phase of a larger initiative.” said Gail Garland, President and CEO of OBIO. “We have spent the last 9 months consulting with 125 CEOs from industry to look at the future of the global industry, identify the opportunities for Ontario and Canada and make recommendations to ensure we derive the economic and patient benefits from competing successfully on the world stage.

10

Biotechnology Focus / June/July 2016

“Ontario is a leader in life sciences. We are proud to foster a diverse economic climate for businesses to thrive and bring new products to market. That is why I was thrilled to meet with members of OBIO to advance our shared commitment to health care innovation and job creation in this vital sector.” — Kathleen Wynne, Premier of Ontario, MPP Don Valley West

We look forward to engaging with visionary stakeholders as we translate this information into action.”

In addition to his address to the crowd at the reception, Minister Moridi had this to say about the event:

“I would like to congratulate OBIO on its successful inaugural Queen’s Park Advocacy Day and their investments in Ontario’s life sciences companies over the past five years. Our government is committed to ensuring that Ontario’s world-class life sciences research is translated into innovative products and successful companies. Our government is proud to have supported OBIO’s efforts in helping promising companies become investment-ready.”

— Reza Moridi, Minister of Research and Innovation MPP Richmond Hill The Ontario Bioscience Innovation Organization (OBIO®) is a not-for-profit, membership-based organization engaged in the development of an integrated health innovation economy for Ontario and one that will become a global leader in providing health technology products and services to the international marketplace. OBIO advances this goal through advocacy, promotion and strategic leadership and via collaborative partnerships with industry, academia, patients and government.

For more information please visit www.obio.ca and follow OBIO on Twitter @OBIOscience

OIRM

By Lisa Willemse

ONTARIO

The pace of progress in heart regeneration

Dr. Michael Laflamme is mixing old and new technologies to piece together the right combination of stem cells needed to repair the damaged heart.

Heart repair has long been a challenge of modern medicine.

Comprised primarily of muscle, the heart is somewhat unique among our organs in that its resident population of stem cells are not terribly active once they’ve finished the job of building the organ prior to birth.

As a result, these lazy (the scientific term is quiescent) stem cells don’t jump to action after injury, as is the case in a heart attack. Instead, scar tissue develops from the dead and dying cells, which can place undue strain on other areas of the heart and lead to heart failure and other associated illnesses in the long term.

Over the years, two main approaches to dealing with this problem have emerged: the first seeks ways to encourage the resident population of heart stem cells to wake up and repair the tissue, and the second uses heart stem cells grown in the lab that can then be transplanted into the patient. It’s in this second approach that Dr. Michael Laflamme, principal investigator at the McEwen Centre for Regenerative Medicine, has directed his focus. Dr. Laflamme is leading a Disease Team project, funded by the Ontario Institute for Regenerative Medicine (OIRM) that seeks to convert scar tissue left behind after heart attack into new replacement muscle.

The replacement muscle comes in the form of new cardiac cells, grown from stem cells in the lab and then transplanted into the injured heart. Much like a puzzle, each piece requires a certain amount of specialized work, from the uniform type of heart muscle cells required for transplant, to the ability to efficiently scale these cells up into large numbers, and to the use of a new electrical mapping technology to determine what effect the transplanted cells have on the natural rhythm of the recipient heart.

To find this level of expertise and infrastructure, Dr. Laflamme, had to pick up his lab and move from America’s west coast to Toronto. He is unequivocal about the wisdom of his move.

“Many of the missing pieces for our work in Seattle were already in place here,” said Dr. Laflamme, who is also a member of the Toronto General Research Institute’s Cardiac Pathology team in the University Health Network. “Toronto has world class experts in stem cell biology; people that are working in the same area and are kindred spirits. I’m an applied sort of guy. I want to see these cells put to use fixing injured hearts, and I think we can do it better here than any other place in the world.”

In Toronto, Dr. Laflamme has already built on his earlier research conducted at the University of Washington, that, while successful in growing and grafting new heart tissue, showed less than optimal results. The research animals tended to develop arrhythmias (irregular heart rhythm), which Dr. Laflamme speculated was a result of the transplant having contained a mix of different types of heart cells, including pacemaker cells, which began working on their own rhythm rather than that of the host’s heart.

“This is where the imaging capabilities will be very helpful. We can record the electrocardiogram to see if the animal experiences arrhythmias, but we can even go further and we can actually map the electrical activity. ” — Dr. Michael Laflamme Biotechnology Focus / June/July 2016

11

CHANGE IN HEART After a heart attack, scar tissue can severely limit heart function and lead to heart failure and other associated illnesses. In Toronto, a team led by Dr. Michael Laflamme is testing how cultivated stem cells, injected into the damaged heart, can form new, healthy tissue.

STAGE 1

DAMAGED HEART

STAGE 2 VERSION 2

VERSION 1

A mixture of pacemaker, ventricular and atrial cells.

bioreactor

A purified selection of ventricular cells.

Two different populations of heart cells, each grown from stem cells, are cultured in bioreactors.

STAGE 3

The two different cell populations are transplanted into damaged hearts. Each transplant will grow new tissue and the two are compared.

STAGE 4

Imaging technologies measure heart function to assess the success of the transplants and to determine which cell population is better.

STAGE 5

The better of the two cell populations will be refined and tested, in preparation for clinical trials in humans.

This research project is funded in part through a Disease Team grant from the Ontario Institute for Regenerative Medicine.

www.oirm.ca

12

Biotechnology Focus / June/July 2016

OIRM

ONTARIO Enter Dr. Gordon Keller, director of the McEwen Centre and an expert in the growth and applications of embryonic stem cells.

Like Dr. Laflamme, Dr. Keller had long been interested in optimizing cardiac and vascular cells from embryonic stem cells for cardiovascular repair. “The key piece of technology that Keller’s group had,” explained Dr. Laflamme, “was a more precise way to grow the particular cell type that we need for replacing the heart muscle cells lost during a heart attack. We were already able to make human embryonic stem cells into heart muscle cells—cardiomyocytes—but his group had figured out how to take them one step further into ventricular cardiomyocytes.” Thus Keller could provide access to a population of pure cells, better suited to the purpose at hand.

Since the goal is ultimately to test the heart cells as part of a human clinical trial (the current OIRM Disease Team project will work with a pig model in preparation for this), Dr. Laflamme’s next challenge was finding a way to grow the large numbers of cells needed and ensure they were at a standard high enough for clinical use. Once again, Toronto had the answer, in the form of the bioreactors housed in the Centre for Commercialization of Regenerative Medicine’s (CCRM) facilities, conveniently located a short walk down the street.

“In Seattle, we were able to grow up the requisite number of cells [about 1 billion cardiomyocytes for each transplant] for large animal studies, but we were doing it by brute force, growing many dozens of flasks of cells,” recalled Dr. Laflamme. “But of course that’s not scalable, it’s not economic and it’s hard to imagine how you’d be able to convert that process into something that is clinical grade. CCRM can do this in a single bioreactor, so they’re obviously one of the key players on our disease team.”

The final piece of the puzzle is the expertise that Dr. Laflamme himself brings—a keen understanding of the electrical properties of the engrafted heart that, up until now, had been missing from Toronto.

Since the problem in earlier studies had been the development of arrhythmias in the recipient animals—a problem that is electrical at its core and that could potentially be remedied with the more appropriate cells produced in uniform batches, the last remaining question will be to determine if it works.

To answer this question, Dr. Laflamme found it necessary to back up all the way to his early career, when he was studying the electrical properties of cardiomyocytes at the single cell level. “I certainly wasn’t expecting to revisit these techniques,” he said.

“But, in 2002, when I began working with cardiomyocytes grown from human embryonic stem cells, we didn’t really understand their electrical properties. So I dusted off those old techniques and applied them to these new cells. I kind of came full circle.”

In Toronto, Dr. Laflamme will be applying these dusted-off skills as well as a new imaging technique that will allow his team to visualize the electrical activity in both the newly-formed heart muscle and the recipient heart. By combining this approach with EKG (electrocardiogram) recordings and catheter-based electrical measurements used in clinical practice, they will be able to better understand the electrical behavior of the engrafted hearts. He’ll be testing both the cocktail of imprecise cardiac cells used in the earlier studies and the more refined ventricular cardiomyocytes grown in the large-batch bioreactor, and looking for new heart muscle that’s firing and contracting in synchrony with the host muscle.

OIRM

Dr. Keller had long been interested in optimizing cardiac and vascular cells from embryonic stem cells for cardiovascular repair. — Dr. Gordon Keller “This is where the imaging capabilities will be very helpful.

We can record the electrocardiogram to see if the animal experiences arrhythmias, but we can even go further and we can actually map the electrical activity. We can challenge the heart to stimulate arrhythmias to see whether grafts formed with one type of cell are more vulnerable to those formed using our newer, more precise cells,” Dr. Laflamme said.

Should the imaging provide the answers they seek, Dr. Laflamme and his colleagues will be one step closer to a human clinical trial, which will also be launched in Toronto.

Lisa Willemse is a communications professional with 18 years’ experience working in the technology, child development and health research fields, and is currently a Senior Communications Advisor with the Ontario Institute for Regenera-

ONTARIO

tive Medicine. With a background in fine art, communications and journalism, Lisa continues to moonlight as a writer, photographer and editor, contributing to a range of Canadian and US-based publications.

In 2014, she was alumni-in-residence for the acclaimed Science Communications program at the Banff Centre. She is also involved in several volunteer initiatives, including serving as a Board member for Science Borealis, a Canadian science blog aggregator. Follow her on Twitter and Medium @WillemseLA.

To see this story online visit http://biotechnologyfocus.ca/pace-progress-heartregeneration

CELEBRATING

7YEARS

Of Delivering High Impact Results

Ontario Bioscience Innovation Organization Advocacy Action Access

OBIO RESPONDS TO INDUSTRY NEEDS: Capital Access, Innovation Adoption and Interconnectivity Initiatives.

OBIO IS A POWERFUL FACILITATOR & KNOWLEDGEABLE RESOURCE: For our member companies, partners and sponsors.

BUILDING AN ONTARIO that will lead the way in providing health technology, products and services to the international marketplace. Join today and experience the benefits of membership.

www.obio.ca

@OBIOscience Biotechnology Focus / June/July 2016

13

Health Innovation Compiled by Shawn Lawrence

ONTARIO

Leading change for Ontario’s Med Tech Sector Q&A with Ontario’s Chief Health Innovation Strategist William Charnetski In September, 2015 the Ontario government took a significant step towards bolstering Ontario’s health technology sector naming William Charnetski as the province’s first-ever Chief Health Innovation Strategist.

In this special Q&A, Charnetski details his plans going forward in this role, his office’s priorities when it comes to the health technology sector and how he plans to continue to support healthcare innovation in the province.

The subject matter of this work is very interesting to me. I’ve been looking at integrated health solutions for the past ten years and prior to that I was involved in work looking at how to use Canada’s investment in the healthcare system as an economic driver. Back then, it was difficult to move that idea forward as there was strong resistance from health officials to approach healthcare from that perspective.

When I returned from London, UK 18 months ago, I was looking to do two things in my next job. First, I’ve always been very interested in public policy, and I knew the time was right to try and “make a difference”. At the same time, I wanted to leverage the global experience and expertise I had acquired abroad. When the opportunity came up to lead the work to drive collaboration across Ontario’s health care system to accelerate the adoption and diffusion of new innovative health technologies with the purpose of not only improving patient outcomes, but also growing jobs in Ontario and building a health technology ecosystem, it pushed all the right buttons. I believe this work is important and that we can make a difference by both leveraging global expertise and bringing global best practices to bear here.

William Charnetski

14

Biotechnology Focus

Q: You’ve had an interesting career path, working at the highest levels of business, law and government, even working with one of the world’s largest pharmaceutical companies at AstraZeneca in UK. Tell us how you came into this role as Ontario’s chief health innovation strategist and why you took the job?

June/July 2016

Health Innovation

ONTARIO Q: What do you think motivated the Government of Ontario’s decision to name a Chief Health Innovation Strategist?

The government was visionary in looking at how it could use its $52 billion investment in the healthcare system as an economic driver. At its core, that was the motivation. It’s clear that if Ontario is going to win in the knowledge based economy, as opposed to some of the low costbased industries in which it has excelled in the past, it has to invest in innovation.

There is an incredible opportunity to do so in Ontario’s health care industry. We will leverage that investment and grow a health technology ecosystem in which we create jobs and wealth in the province in one of the fastest growing job and wealth creators of the 21st century.

The other contextual point is the critical need to preserve the sustainability of the public healthcare system. To do that, it will be important to introduce innovation into the system more quickly and more broadly than has been done in the past.

That also was a driving factor behind the decision to create this role.

Q: Can you briefly outline your mandate as chief health innovation strategist and take us through your daily routine in this role?

We’ve developed what we call a purpose statement and it is self-explanatory. The purpose of our work is to drive collaboration across Ontario’s healthcare system to accelerate the adoption and diffusion of new innovative health technologies and processes with three objectives: to improve patient outcomes, add value to the system, and grow jobs in the province.

In fact, it’s that third objective, growing jobs, that distinguishes us from many other public and private organizations in the province that are involved in health and innovation.

The uniqueness of this role is that we are focused on growing Ontario-based

companies, including those who already export their health innovations globally, but cannot sell them in Ontario. The challenge for many health technology-based companies is that they often are unable to penetrate the market in their own backyard. Our purpose statement encapsulates what we’re trying to accomplish for them.

There is no “usual day” in my role, which is part of what makes it so interesting and rewarding. I love my job and say so nearly every time I have a public speaking engagement of which I have many. What we do straddles the public sector (i.e., the Ministry of Health and Long-Term Care within which I sit on the Deputy Minister’s Executive Leadership Team) and the private sector and broader public sector. The latter group includes the various healthcare providers, universities, colleges, research institutions, incubations labs, innovation centres, entrepreneurs, innovators, multi-national companies, and health tech/device companies doing business in Ontario.

A day in my job may involve internal meetings with Ministry colleagues where my role is to help ensure we are all considering how innovation can be introduced into healthcare delivery in the province.

It is also important to build relationships with Health Ministry colleagues because, of course, whatever we set out to do must also be a priority for them. Otherwise, nothing will happen as they ultimately have the delivery accountability. Our office does not. So it is critical to our success to collaborate well with them.

A day in my job may also involve external meetings with any of those public or private institutions I’ve described. We’re either hearing from a company about their successes or frankly the challenges and the barriers that they face and how we can help them. We’re hearing from healthcare providers and listening to what their priorities are and sharing with them some ideas on how we could broker relationships between them and innovators.

Moreover, raising public awareness is another part of our function, and this

Our focus is on how to help make that happen by using enabling or virtual technologies like digital or mobile health. — Bill Charnetski

includes giving speeches or participating in panels with a clear purpose to help achieve our priorities and objectives.

Q: Can you tell us about these priorities and objectives?

From the very beginning, I’ve said to people that we will judge ourselves by the impact we have had, not by the activity we generate.

To have that impact, it’s very important to focus our efforts. There’s a saying that if everything is a priority, nothing is. If you’re actually going to make an impact, you have to focus on a few strong priorities. So we’ve decided to focus on five.

The first is to optimize pathways to adoption and diffusion for innovative Ontario health technologies. This focuses on having the healthcare system drive demand and using the pull of a single payer system.

Our second priority is to enable effective procurement of innovation by shifting the health care system to strategic, value-based procurement and removing barriers for small- and medium-sized enterprises to participate.

Procurement is meant to be a driver of innovation, but in Ontario, it hasn’t been seen that way. The environment is very constrictive, and the rules are very narrow and often interpreted conservatively, making it difficult for the procurement agent or healthcare provider to procure innovation.

It’s a very cost focused system where the focus is on driving down costs. Now the good news: A supply chain management review is now underway, including work of an expert panel that will be making recommendations for change to the procurement model.

Our third priority is a critical one for most healthcare systems globally, namely, home and community health. We call it “Better care closer to home” and our focus is how to make it happen by using virtual, mobile and digital health technologies.

This is fertile ground for Ontario health tech companies and there are many that have been successful in marketing these products around the world. However, they struggle with adoption in Ontario and I hope to change that.

The fourth priority is empowering On-

Biotechnology Focus

June/July 2016

15

Health Innovation

ONTARIO Our role in this is to help the people doing this type of work to collaborate closely with innovators that provide these types of innovative solutions.

And finally, the last priority is to enhance aboriginal health outcomes. — Bill Charnetski tarians through digital health by building a dynamic market of ehealth tools and supports to navigate and personalize their path to health and wellness, including for example the use of electronic health records. The principle is that we should have a patient-centric approach to the use of personal health information to allow patients and their circle of care to make better and faster care decisions.

Our role is to help the people doing this type of work and to collaborate closely with innovators that provide these types of innovative solutions.

The fifth priority is to enhance Aboriginal health by advancing opportunities for innovation to address health challenges in their communities. This is a broad and important topic and there are huge opportunities before us to use new madein-Ontario health technologies and apply them to enhance health outcomes for First Nations people across the province.

Q: You’ve mentioned the challenges and barriers innovators in this space face. What are some examples?

We hear about procurement issues, both the rules as they are drafted and the behaviors associated with assessing and applying those rules in practice.

I think the biggest concern we hear expressed revolves around procurement practices that either make it very difficult for Ontario’s smaller- and medium-sized enterprises to qualify or participate in a tender, or the act of participating is just onerous.

The procurement system in Ontario is relationship based, it’s not necessarily evidence based. Larger companies are able to employ many people to build the necessary relationships in such a system while small- and medium- sized companies may have only one or two people and are not able to blanket the province in the same way.

16

Biotechnology Focus

June/July 2016

Then, increasingly, hospitals are bundling the goods and services that they’re asking for in tender, making it impossible for small- and medium-sized enterprises to qualify.

Even when a small or medium sized enterprise can qualify for a tender, the procurement process often requires hundreds of pages in response to Requests for Proposals and a long time investment.

Again, while larger companies are able to do because of their bigger workforces and portfolios, it is virtually impossible for smaller companies to participate.

There’s also a risk aversion in Ontario to go with a smaller and lesser known company. There are other challenges, such as bringing innovative devices or processes to the healthcare market and realizing that the doctors fee schedule that governs the reimbursement and payment of physicians for their services doesn’t even contemplate the particularly innovative approach or device being put forward.

Quite literally, a doctor will not get paid for adopting the innovative technology being offered. These are some of the barriers that we see over and over again.

Q: Staying on this topic of procurement, the Government of Ontario announced in May of last year its intention to launch a $20 million Health Technology Innovation Fund as a means to back the adoption of made-in-Ontario technologies. Fast forward to the present and you’ve just announced you will soon be accepting applications for this Fund. Tell us about it and who it’s geared towards?

The $20 million Fund is designed to support pre-market evaluations and early adoption of innovative Ontario health technologies. Through it, we will invest $5 million over each of the next four years to accelerate innovation across multiple health care settings.

In the spirit of the importance of prioritizing our work to have the greatest impact, instead of spreading the resources across all five priority areas, we’re going to focus on better care closer to home for the fund’s first round. It will support home and community care such as virtual, digital and mobile technologies and work towards delivering better coordinated and integrated care in the community.

Eligibility for funding will require strong collaboration in what we call a Health Innovation Team. Depending on the application stream, a Health Innovation Team may consist of an Ontario based health technology business, health procurement professionals in provincially funded health delivery organizations, researchers with expertise in research and evaluation design and implementation, and most importantly, whoever will be using the technology such as a clinician, doctor or patient.

A note on the Ontario based health technology businesses: these will include either small to medium sized enterprises or a large multi-national business, as long as more than half of their R&D and manufacturing occurs in Ontario.

The fund will have three streams. The first stream is called Prototype. Innovators should apply if they’re looking to test a new product or feature of their product. They will have the opportunity to work with a healthcare provider(s) to get valuable input on the design. Each team must have a strong business plan, including a feasibility study and problem validation, and the end result will be a new technology that is market ready with proof of its potential value in Ontario’s healthcare system.

The second stream is Premarket Evaluation. Innovators will work with healthcare providers in a health innovation team to test the feasibility of use of their product on a day-to-day basis including training and changes in workflows. The activities from this stream should result in the development of an evidence package that can support procurement of the product to the healthcare provider’s hospital, clinic, or a home care setting. The stream also includes an economic analysis that examines the efficiency of the technology and potential cost savings to the system, as well as a plan that outlines the systems and processes required for wide scale adoption.

The third stream is called Early Adoption. Innovators will work with their

Health Innovation

ONTARIO health innovation team to refine the evidence package and develop a plan to help facilitate adoption of their product in multiple healthcare locations. That includes strategic or value based procurement. Here results will include successful adoption, multiple healthcare locations and a report on the change and knowledge transfer required to successfully adopt the new technology.

Health innovation teams can only apply to one stream at a time.

For the purposes of the program, medicines, vaccines, drug efficacy research will not be supported. We’ll have an expert panel that reviews proposals, and panel membership will depend on the type of proposals we receive. Details on applications will be available on July 4.

Q: The other major component of your recent funding announcement was that you will soon be recruiting Innovation Brokers. How do they fit into what you’re hoping to accomplish?

The creation and hiring of “Innovation Brokers” was also one of the key recommendations made by the Ontario Health Innovation Council – in addition to the creation of my role. I’m very excited by this concept and the process of hiring our first two Innovation Brokers is well underway.

This office was created to be an outward facing organization, and as is the case with any high performing social enterprise, whether it is in the public, political or private sphere, the notion of very knowledgeable community representatives is critical and highly valuable.

We want people who are able to bring insight from a particular geography or sector into the strategic and tactical conversation.

This is hugely valuable, and that is what these Innovation Brokers will be. They will apply that insight to work with innovators and health care providers to create an even greater culture of innovation across the province, principally by enhancing connections.

Moreover, their job or brokering function is to remove barriers to the scaling and adoption of innovation, and to link the health tech companies with potential opportunities from healthcare providers. They will be our eyes and ears in the community.

They will also be the people on the proverbial ‘other end of the phone’ when an entrepreneur or innovator is trying to figure out their way through the Ministry of Health and Long-Term Care landscape. Similarly, they will be there to inform the healthcare providers what certain companies are doing.

Q: You have a big task ahead of you. What is the hoped for outcome and how will you know you’ve succeeded?

Our goal is to build a health technology ecosystem for Ontario and we will judge our success on metrics.

Those metrics will be derived from questions such as: • Have we actually had technologies introduced into the system?; • Has that improved patient outcomes?; • Has it created new companies and jobs in Ontario?; • and Has as it led to additional investment in Ontario?

In addition, we will put patients first in our work. When I described my work to a friend who was a very smart professional but not in the health care industry, she asked a question of me that was so profound our team has put it on the wall in our office: “From a patient’s perspective, in two years, what will I see

that is different as a result of your work?” Like everyone in the Ministry of Health and Long-Term Care, that governs our work.

The $20 million Health Technologies Fund is an important step, but it is only one tool to help us achieve those objectives.

Ultimately, we will succeed or fail based on how we measure up to those questions. And I am convinced that, with all the resources we have across the province, which includes world class hospitals, research institutions and universities, full government support in a single payer system and hundreds of passionate hard working people already driving health innovation in the province, we will succeed.

In particular, I believe there will be a tipping point. We will not have to change everything. We just have to change a few things, and there will be a tipping point where a few changes will have significant benefits for the health care system, for patients and for the economy.

Ultimately, I believe we will see significant improvement in the ability of Ontario companies to grow and prosper because of Ontario’s health care system and not in spite of it. At the same time, we will see improved outcomes that will benefit all Ontario patients.

To see this story online visit http://biotechnologyfocus.ca/leading-change-forontarios-med-tech-sector

www.kalgene.com “KalGene is determined to develop therapeutics to treat Alzheimer’s.”

- Dr. T. Nathan Yoganathan, CEO, KalGene Pharmaceuticals Inc.

KalGene Pharmaceuticals moving to new JLABS @ Toronto at MaRS to accelerate development of Alzheimer’s therapeutic KalGene Pharmaceuticals is developing a novel therapeutic molecule to treat Alzheimer’s disease. KalGene is collaborating with NRC, McGill, the Montreal Neurological Institute and CIMTEC to advance the therapeutic development.

KalGene plans to initiate Phase 1 clinical trials in 2017.

For additional information on KalGene’s efforts to develop new treatments for Alzheimer’s disease, please contact nathan@kalgene.com

Biotechnology Focus

June/July 2016

17

JLABS @ Toronto

By Shawn Lawrence

ONTARIO

JLABS @ Toronto Ontario’s life science startups have a new place to call home Things just got a little more crowded on the 13th floor of the MaRS West Tower as Johnson & Johnson Innovation has officially opened the doors to its newest startup incubator, JLABS @ Toronto.

The arrival of JLABS on the Ontario life science scene was greeted with tremendous fanfare, as approximately 2,000 people jammed the entire first floor of the MaRS building as part of a spectacular launch event held on May 11, 2016. Many came out to see the lucky 22 companies selected to be its first residents, while others wanted a first glimpse inside the state of the art facility. While not all were lucky enough to get the grand tour, those who did, got more than they bargained for, learning that there’s plenty of substance behind the hype around this 40,000-square-foot life sciences incubator.

As JLABS @ Toronto site head Rebecca Yu explains, the companies taking up residency are about to step into a whole new world. Having worked as director, strategic health technology assessment in the Government Affairs Department at Janssen (Canada), a division of Johnson & Johnson, prior to stepping into her new role with JLABS @ Toronto in December, 2015, she’s witnessed firsthand how the JLABS model works and why it has been so successful in other markets.

“The idea is for a startup company to come into the space and have everything they need so they can focus their efforts on what’s truly important, taking their scientific discoveries forward” she says.

She adds that tenant companies benefit from the capital-efficient and resourcerich environment that JLABS provides.

“For us it’s about giving companies the opportunity to accelerate the development of their technologies by making sure everything, including the physical and operational side of the lab is taken care of and that their business development needs are met.”

According to Yu, these are the founding principles behind all of Johnson & John-

18

Biotechnology Focus / June/July 2016

son’s JLABS incubation sites across North America. Among them are sites in San Diego (flagship), San Francisco, South San Francisco, Boston and Houston. Together, they house more than 120 early-stage companies advancing bio/pharmaceutical, medical device, consumer and digital health programs. Within each of these facilities, companies share lab space and offices, work in modular lab suites and have access to scientific, industry and capital funding experts.

This mix of companies is no accident adds Yu, “That’s how we like to see the ecosystem. By having companies with different backgrounds or representing different sub sectors together, working in the same space, they can actually learn from each other and co-create value.”

Yu believes both the formula and the success enjoyed at other JLABS sites will easily be replicated at JLABS @ Toronto.

To help run the facility, Yu has an inhouse operations team that includes both a manager and an office leader. The team oversees everything at the site ensuring that the existing infrastructure and state of the art equipment is in working order.

They also train tenant companies on how to use this equipment, which includes such things as cell culture, western blot and cell imaging, multimode plate readers, cell sorters and a host of othe analyzers.

Also, resident companies have a device and digital prototype lab with even more highly specialized tools at their disposal.

As an added caveat, JLABS @ Toronto tenants will get to share a receptionist who is able to take calls on their behalf and manage their meetings with potential customers and investors.

In terms of user fees, they are customized to the services provided or required.

Further on the physical aspects of the

lab, resident companies have flexible options to lease as much lab space as they need. As mentioned, the labs themselves are modular and scalable. Users can remove or add benches and even walls as they see fit. It’s all about accommodating the client company’s needs, Yu explains.

“You can come in small and grow as your company grows. It’s very customizable. In San Francisco for example, we thought originally we could host about 40 to 50 companies, but in the end we maxed out at 27 simply because companies took up larger individual spaces,” says Yu. And if privacy is a tenant’s priority, JLABS @ Toronto does offer options here as well.

“There are certain labs that are enclosed for example, and likewise, they have lockers and cabinets to lock away and secure their belongings at the end of every work day,” she says.

But if Yu has one recommendation, it’s that resident companies should still take full advantage of the open concept labs and workstations.

“What we find with the open concept model is that tenants are actually talking to each other and discussing what they are each doing and finding common ground. They come into this space to cocreate and co-think, and hopefully come out with a new product as a result of this convergence.”

Then there are the other perks beyond the physical elements to being part of JLABS @ Toronto. These include unrivaled

ONTARIO

access to unique educational programs and networking events that have both a scientific and business focus.

For example, there are workshops for resident companies with external investors from both within and outside of the region. This includes a funding series with venture capitalists, granting agencies, corporate ventures and other corporations.

Likewise there are sessions scheduled externally with other industry players and experts as well as internally with Johnson & Johnson staff.

“These are the huge value adds of our program, having the capability of bringing in these external folks, including our competitors coupled with those that we bring in directly from Johnson & Johnson to speak with entrepreneurs, educate and mentor them,” Yu says.

She adds that these mentors can offer insight into what works and what doesn’t when building up a company.

“It could be as simple as understanding what the right business model is for a specific technology, and if a business model is going to make sense. It could be something like where to look for investments and resources or finding ways to broker relationships. It’s a very robust curriculum and I think it’s invaluable,” she says.

Moreover, resident companies at JLABS @ Toronto also have the opportunity to collaborate with IBM Canada to access its entrepreneurship programs and services, including IBM Watson cognitive business technology and IBM Bluemix cloud-based development platform.

It all ties into perhaps the most important key differentiator of JLABS, its no strings attached motto.

On this front Yu is quite clear, selected companies remain independent and Johnson & Johnson does not claim any rights or affiliation.

“We don’t own any of the IP and we don’t have first right. There’s no obligation

for Johnson & Johnson to sign with the company, and there’s also no obligation for them to sign with us. We only deal with non-confidential information, and over and above that, we actually proactively introduce our companies to our other pharma and medical devices companies, so that we cultivate relationships and get these companies through to the commercialization stage.”

That’s not to say partnerships can’t or won’t happen, but rather, it’s just that it’s more likely that the majority of resident companies will remain independent. For example, Yu estimates that at its existing six JLABS incubators, Johnson & Johnson has collaboration partnerships with only about 30 per cent of the companies.

For JLABS @ Toronto itself and reaching full capacity, it’s expected that the site will be even larger than the ones in Boston, Houston and San Francisco. Maximum capacity is pegged at 50 companies, but that number is a moving target as it will depend on the footprint size of the companies taking up residence. Yu does say proudly that JLABS @ Toronto has seen the fastest company uptake of all the global sites.

“Given we had a very short runway and we didn’t start the selection process until November of last year, there’s been an incredible amount of interest in coming into our space,” she says.

On how the first 22 companies where chosen, Yu says that applicants were reviewed by a selection committee made up of Johnson & Johnson experts from its R&D and Innovation Center.

“They looked at whether they were developing a transformational technology, if it met an unmet need and if it was compelling science. They also looked at the credibility of the company itself, and the entrepreneur. To a lesser extent, they looked at the funding they had available and if there were investors already supporting the company. At the end of the

day, we wanted companies that could come in and truly benefit from the right coaching, mentoring and support we provide. That for us is much more important than the numbers.”

With its present selections as well as future choices, Yu says JLABS @ Toronto will focus in on companies who represent Toronto’s research strengths.

“It’s like that with all our JLABS sites.

For example in Houston they have the MD Anderson Institute, so oncology is big focus area. I’d say the areas of focus here will include neuroscience, oncology, cardiovascular disease, infectious disease and immunology. On the medical device side: diagnostics, surgical devices, and orthopedics. And on the consumer health side, we’ll be looking at oral and digital health companies. These are all areas that Toronto has a significant presence in.”

It is the strength in all these areas that Yu believes drove Johnson & Johnson’s decision to establish this on-the-ground research presence here. It’s also why the new JLABS facility has struck partnerships with six hospitals in Toronto, including the Centre for Addiction and Mental Health, The Hospital for Sick Children, Sinai Health System, St. Michael’s Hospital, Sunnybrook Health Sciences Centre, and University Health Network.

“I think it’s fairly clear we’re coming into an area where you’ve got a huge critical mass and the science here is incredible. You’ve got the University of Toronto who has spun out a number of our resident companies, the MaRS Discovery District, the MaRS building itself which houses everything, and all the hospitals all within walking distance. And of course the financial centre of Canada is only a subway stop away.”

She adds that both Johnson & Johnson and Janssen Canada have strong existing ties here, having been very active in the past on the collaboration front. They are well aware of the environment. Moreover, in terms of planting the seeds for JLABS @ Toronto, the Ontario government with its $19.4 million commitment made through its Strategic Partnerships Stream of the Jobs and Prosperity Fund certainly played a role.

“And it’s not just the financial piece, but also the way they pitched Ontario as a place to do business,” she says.

The metrics that will determine if this initiative is a success have already been set out.

Biotechnology Focus / June/July 2016

19

JLABS @ Toronto selected companies to be the first residents of JLABS @ Toronto JLABS @ Toronto Resident

About the Company

6biotech

Accelerating the discovery of new drugs through the creation of technologies designed for high-value data acquisition that mimic complex in vivo biology.

Abcombi Biosciences Inc.

Providing technology platforms that can be tailored and applied to any disease treatable via prophylactic vaccination.

Biotagenics

A microbiomics platform company developing novel therapies and diagnostics targeting intestinal microbial dysbiosis in inflammatory bowel disease.

Cogniciti Inc.

Reducing the time and cost of volunteer recruitment for clinical trials through a science-validated online test for brain health.

DNAstack

Developing a cloud-based platform for genomics data analysis.

IGY Immune Technologies & Life Sciences

Designing and manufacturing technology and processes to strengthen and supplement the body’s natural immune system against broad and targeted bacterial, viral and autoimmune ailments.

Immune Biosolutions

Harnessing chicken antibodies to overcome challenges of anti-GPCR antibody generation and providing new antibody candidates for difficult druggable targets.

KalGene Pharmaceuticals

Developing a new therapeutic to bring back the memory of patients with Alzheimer’s disease.

Ketogen Pharma Inc.

Maximizing the therapeutic potential of the ketogenic diet to develop a new class of anti-epileptic medications.

Proteorex Therapeutics Inc.

Unlocking a treasure trove of new drug targets by targeting protein complexes in human diseases with a next generation small molecule platform technology.

Realist Pharma

Developing oncology-focused therapeutics that treat a wide range of cancers with its main programs being REAL-1 (a “next gen CAR-T” cell therapy) and REAL-2 (NCE cancer vaccines), both of which target >10 highly prevalent tumor markers.

Seegene

Proprietary priming chemistries that make multiplexed PCR easy, effective and affordable.

SYNG Pharmaceuticals

Developing the first diagnostic blood test for endometriosis and an innovative non-hormonal therapy.

Translatum Medicus Inc. (TMi)

Dedicated to saving eyesight with a first-in-class immunomodulator therapy for patients with “dry” Age-Related Macular Degeneration.

Ubiquitech

An early-stage drug discovery company focusing on identifying and developing next generation cancer therapies.

App4Independence*

A4i is a digital health innovation company that provides clinically proven mobile interventions to significantly enhance treatment engagement and quality of life for individuals with psychosis and other major mental health challenges.

AVROBIO*

An innovative gene therapy company focused on immune-oncology and rare diseases.

Chestnut Pharmaceuticals*

Developing novel therapeutics for metastatic cancers employing a comprehensive approach to reverse the metastatic state of tumor cells.

Nanovista Inc.*

Achieving high-precision personalized cancer therapy with multimodal nano imaging agents: lead application in image-guided surgery.

Neutun*

Creators of a seizure tracking tool that uses devices patients already own and wear.

PinPress*

Regular molds have one shape; PinPress is a shape-shifting tool that gives you a thousand molds in one.

Suncayr*

Helping people enjoy the sun with a smart indicator of ultraviolet light that changes colour to warn people of their sun exposure.

“Whether it’s companies coming in and we play a role in facilitating their success, bringing jobs and investments to Ontario, or seeing collaborations, especially if that collaboration is with Johnson & Johnson, ultimately our end goal is to the technology, whether it be a drug, device or some other products to the patient who has the unmet need. If we can do that, then we’ll know we’ve been successful.”

Based on how things went for JLABS @ Toronto on opening night, they’re off to a good start.

20

ONTARIO

Biotechnology Focus

June/July 2016

To see this story online visit http://biotechnologyfocus.ca/ jlabs-toronto-ontarios-lifescience-startups-have-a-newplace-to-call-home

Ministry of Research and Innovation Compiled by Shawn Lawrence

ONTARIO

In Conversation with

Ontario’s Minister of Research and Innovation Reza Moridi As we have traditionally done in year’s past, we go one-on-one with Ontario’s Minister of Research and Innovation Reza Moridi to discuss the state of Ontario’s life science industry.

Q: What are the Ministry’s goals for this year’s BIO International Conference in San Francisco?

BIO is a fantastic opportunity for Ontario to showcase and promote its strengths in biotechnology to the world, and for the province’s life sciences companies to reach new markets and customers. Ontario’s presence at this conference will reflect its standing as home to one of North America’s largest and most vibrant life sciences sectors, which fosters a unique spirit of collaboration I’m proud of.

We are excited to continue to lever our reputation as a jurisdiction at the forefront of biotechnology development to generate new investment leads.

Q: You personally have remained very consistent in your messaging throughout your tenure as the Minister of Research and Innovation that collaboration between academia, the research community, industry and government is paramount to Ontario maintaining its standing as one of the leading life science jurisdictions in the world. On this front, what initiatives that the Ministry has had a hand in over the past year are you most proud of?

Perhaps one of my proudest moments would be our role in working with Minister of Health and Long-Term Care Eric Hoskins, the Ontario Health Innovation

Council and our medical technology community to create the Office of the Chief Health Innovation Strategist and a $20 million fund to support this work. This tremendous opportunity helps us strengthen our health innovation ecosystem: collaboration between government, industry, health providers and patients will transform patient care and build the economy for tomorrow.

We have also made many investments in Ontario, including: • Helping young innovators and aspiring entrepreneurs across the province create the next wave of highly skilled jobs and innovative products. The Ontario Government is investing more than $80 million for innovation-focused funding and $565 million over the next two years in the Youth Jobs Strategy, as announced in the 2015 Budget.

• The Ontario Government, University of Toronto, and MaRS Discovery District (MaRS) launched a collaboration with Janssen Inc. for the successful Johnson & Johnson Innovation JLABS incubator model in Toronto. The new facility, called JLABS @ Toronto, is the first JLABS to open outside the United States. JLABS @ Toronto will provide startups with many of the advantages of being part of an established innovation centre, such as access to talent and mentors, large existing firms and research universities, capital and convergence opportunities with other sectors. When

complete, the 40,000-square foot facility will include cutting-edge, modular and scalable lab space, equipment, with access to scientific, industry and capital funding experts.

• The province is contributing $15 million over 10 years to the Lazaridis Institute for the Management of Technology Enterprises in the heart of the Kitchener-Cambridge-Waterloo Region, dubbed ‘Canada’s technology triangle.

The institute will help bolster Ontario’s emerging technology sector by teaching the skills required to grow successful start-ups ready to prosper in the global marketplace.

• Ontario is investing in Stem Cell Research by providing $25 to the Ontario Institute for Regenerative Medicine to study chronic disease treatments. This funding will support new treatments and therapies for people living with chronic diseases, such as cancer, multiple sclerosis and diabetes.

Biotechnology Focus

June/July 2016

21

Ministry of Research and Innovation

ONTARIO We are also committed to sustaining and strengthening Ontario’s world-class research and science environment. For example: • In 2015, Ontario invested $209 million in 280 research projects through the Ontario Research Fund and the Early Researcher Awards programs, building on current, ongoing and innovative research, fostering new discoveries and supporting knowledge- based jobs across the province.

• The Ontario Brain Institute’s objective is to maximize the impact of Ontario’s excellence in brain research and care.

OBI activities centre on high-impact translational programs, informatics and analytics platform (Brain-CODE), mechanisms to engage and support industry, training and entrepreneurship, and communications, knowledge translation and evaluation. To that end, OBI is bringing together 200 researchers from 35 institutions, 40 companies and 20 patient advocacy groups.

Brain Code will help accelerate brain research including clinical, imaging and neuro-

psychology assessments.

• Clinical Trials Ontario will attract increased international clinical trials investments to the province by streamlining the ethics review and contract approval processes for industry-led multicentre clinical trials. CTO has created an electronic clinical trial research ethics approval system that has already significantly reduced the time it takes for a clinical trial to obtain ethics approval.

Q: What differentiates Ontario from other life science jurisdictions and how do we stack up against international competition?

Ontario is the largest life sciences jurisdiction in Canada with more than 50 per cent of the total Canadian life sciences economic activity. In order to sustain this standing, the government recognizes Ontario’s capacity to compete in a fiercely competitive global economy depends on how well we can harness our research, innovation and entrepreneurship strengths.

That is one reason why we support the full innovation continuum – accelerating the translation of scientific discoveries from the laboratory to the marketplace.

Q: In your discussions with Ontario companies, and even multinational companies with subsidiaries here, why do they say they choose Ontario?

Companies recognize Ontario’s strategic investments to help mobilize and prepare researchers, entrepreneurs and companies to succeed, compete and create jobs of the future. They are also aware of our investments – more than $3.8 billion across13 years – in research and innovation to attract talent and investment, provide global solutions and create good jobs.

Q: One of your goals is to attract businesses and foreign investment into Ontario’s life science sector and you mention a number of initiatives that have been launched to accomplish this.

Is the world getting the message that Ontario is a place to do business?

Advancing the Success of Ontario’s Life Sciences Sector Ontario’s Life Sciences Sector is a Top 3 North American jurisdiction Ontario’s life sciences industry ranks in North America’s top three by number of establishments, contributing $21.6 billion to Ontario’s GDP.

You'll find this and other fully-researched benchmarks documented in our 2015 Life Sciences Ontario Sector Report, a first-in-class look at Ontario's life sciences sector and its competitive standing against other international jurisdictions.

Life Sciences Ontario supports and advances the commercial success of the province's life sciences sector, through advocacy and collaboration with government, industry, academia, and other organizations across Canada and internationally.

22

Biotechnology Focus

June/July 2016

Innovation Lives Here Problems get solved here.

Awards are won here.

Melinda Richter, Head Johnson & Johnson Innovation, JLABS

Innovation that leads to advances in life sciences requires a vast network of partners – hospitals, research centres, universities, technology incubators, start-ups, scientists and multinationals – sharing knowledge and expertise.

Ontario has one of the world’s most diverse and thriving innovation networks. That’s why Johnson & Johnson Innovation recently chose Toronto to open their first JLABS incubator outside the United States.

It’s also a major reason why Graham Collingridge, this year’s recipient of the world’s largest prize in brain research, recently moved to Toronto.

Dr. Graham L. Collingridge Chair, Department of Physiology, University of Toronto Senior Investigator, Lunenfeld-Tanenbaum Research Institute, Mount Sinai Hospital

Insulin and stem cells were discovered here. We have a storied history of biomedical excellence, but are very much focused on the future. We’re taking neural surgery to new levels of precision. We’re exploring ways to heal the previously incurable.

We’re ensuring cancer has no place to hide.

And with all of these advances, lives won’t just get longer – they’ll get better. And that’s the true measure of progress.

Cameron Piron, President Synaptive Medical (Neurosurgical Technologies)

Visit Ontario at BIO 2016 in the Canada Place pavilion, Booth #5944

InvestInOntario.com/BIO2016 Paid for by the Government of Ontario Photos courtesy of: (top) Johnson & Johnson Innovation, JLABS; (centre) University of Toronto; (bottom) Synaptive Medical

Ministry of Research and Innovation

ONTARIO Ontario is building a more dynamic, competitive environment for job creators with the newly launched, $400 million Business Growth Initiative. The initiative focuses on three areas: creating culture of innovation, helping more businesses scale-up, and modernizing regulations.

The new JLABs facility at MaRS (JLABS @ Toronto) is an initiative under the Strategic Partnership Stream of the Jobs and Prosperity Fund. The Strategic Partnership Stream was designed to encourage open innovation technology partnerships, allowing companies, research institutions, suppliers, investors and customers to work together and establish industrydriven strategies.

The province’s investment in JLABS@ Toronto will provide a valuable long-term asset for Ontario’s innovation ecosystem in the form of a state-of the art lab space for life sciences start-ups. The facility will also attract global venture capital interest and give start-ups access to global commercialization and entrepreneurship experts, including access to Johnson & Johnson’s global resources that span 57 countries and five other JLABS facilities.

In addition, it will provide training and expertise for entrepreneurs in the life sciences sector.

Furthermore, since JLABS committed to Ontario, we have seen an uptake in the number of multinational companies visiting the province to understand our science and meet emerging entrepreneurs.

Q: One significant change this year was the appointment of Bill Charnetski to the role of chief health innovation strategist. What were the reasons for creating this position, and why was Mr. Charnetski the ideal candidate to fill this role?

We created this position because it was one of the key recommendations of the Ontario Health Innovation Council’s report to champion Ontario as a leading centre not only for new and innovative health technology, but also for bringing that technology to market both here in Ontario and globally.

We were excited to welcome Mr. Charnetski to the role, given he is an accomplished national and global executive with more than a decade of experience in developing integrated health solutions in Canada and around the world in the global pharmaceutical industry.

24

Biotechnology Focus

June/July 2016

The government established the Ontario Health Innovation Council (OHIC) to accelerate the adoption of new technologies in our health-care system and support the growth and competitiveness of Ontario’s health technology sector. — Reza Moridi Q: In a sense, is the creation of this role an example of the type collaboration you wish to see across Ontario’s Ministries and a means to further your collective impact on the sector?

It certainly is. This work requires partnerships and collaboration between ministries including Economic Development, Research and Innovation and Health and Long-Term Care. Building bridges between these three ministries is key, and I’m proud to say we’ve created multiple meaningful opportunities to collaborate.

Our shared goals include creating a strong medical technology business environment in Ontario to ensure its continued growth and advance prosperity in Canada; position Ontario as a global leader in life sciences and a destination of choice for medical technology investment and development; and maximize the industry’s contribution to high quality and sustainable health care.

We are finding ways to improve outcomes for patients. Together, we increase economic activity and create prosperity by leveraging the health-care system to strengthen our medical technology sector, and in so doing, improve the quality of health-care delivery service through the adoption of innovative health technology.

Q: Staying on the home front, at the LSO Awards Gala held in February, you reaffirmed the Province’s commitment to the creation of a public/private life sciences working group, to strategize how Ontario can further the sector’s growth. How much progress has been made here?

The Ontario Government supports Ontario’s life sciences sector. Over the last decade, the provincial government has invested almost $2 billion in life sciences.

And Ontario remains committed to attracting investments in research and development to improve the industry’s competitiveness. From 2010 to 2014, the Ontario Government has invested in 44 life sciences

companies supporting the creation of 1,030 new jobs, the retention of more than 1,400 existing positions and leveraging a total investment of nearly $700 million.

The government established the Ontario Health Innovation Council (OHIC) to accelerate the adoption of new technologies in our health-care system and support the growth and competitiveness of Ontario’s health technology sector.

Last fall, we passed the Better Business Climate Act, which commits Ontario to acting as a catalyst to spur the development of clusters. Life science is a priority cluster and we will work with businesses and other entities to develop plans to continue to develop the industry.

Ministry staff are creating a working group to look at ways to grow and scale Ontario’s life science companies. Industry, with support from Ministry staff will develop options and recommendations to improve the sector broadly. I remain interested in this Ministry initiative, and will monitor its work closely.

Q: At this same event, you went on record citing the need for new facilities to help attract world-class biotech events to Toronto, and put Ontario on the global life sciences map. Is it safe to say you hope to see Ontario hosting an event like BIO during your term as Minister and is it possible?

Yes, I would welcome having more of these events here. Ontario is home to a large and vibrant life sciences sector, which represents a huge economic growth opportunity for Canada and Ontario, in particular.

Given the large public investments already made in the sector, attracting world-class biotech events is a natural next step.

To see this story online visit http://biotechnologyfocus.ca/ in-conversation-with-ontariosminister-of-research-andinnovation-reza-moridi

Special report

ONTARIO

By Jason Field, President & CEO, Life Sciences Ontario

Championing our successes: Getting loud about Ontario life sciences innovation Did you know that Ontario’s life sciences sector is one of the top clusters in North America? Ontario ranks as one of the three most significant life sciences jurisdictions by number of establishments, and among the top 10 by number of workers.

Conservative estimates put annual revenues for Ontario’s life sciences sector at $40.5 billion - directly contributing $21.6 billion to our province’s GDP. The sector’s job growth since 2001 is 25.1 per cent, outpacing the provincial average of 15.9 per cent. And, these jobs are higher paid: 26.5 per cent higher than the provincial average, in fact.

If these figures aren’t widely known, it’s because our sector has traditionally had little in the way of benchmarking, and, by that token, no real way to measure its success. Last year, Life Sciences Ontario undertook an industry-first study – our 2015 Life Sciences Sector Report – which helped quantify Ontario’s achievements and verify that our province is a force to be reckoned with when it comes to innovation in biotech, pharmaceuticals, medical technologies, and the agri-food industry.

The superheroes of our sector are the companies and organizations across Ontario creating the innovations that will secure our health and economic prosperity for generations to come – both within Canada and on a global scale. Who are they? What are their stories? Why aren’t they household names – or at least better known than they are, currently?

I believe part of the problem is this: science typically takes a back seat in popular culture. Researchers toil behind the scenes, working in labs, rarely seen or recognized. The other part of this disconnect seems to be a healthy (or perhaps unhealthy) dose of Canadian modesty.

But consider this list: Insulin. T-Cell receptors. Stem cells.

Safer heart surgeries. These are made-in-Ontario innovations with massive global reach.

Their achievements are vast, and too many to list here, but here are some key innovations happening in Ontario’s life sciences sector as we speak: • Ottawa company eSight has developed mind-blowing digital eyewear that allows the legally blind to see • Synaptive Medical’s “neural GPS” is helping surgeons reduce risk and better navigate the brain in 3D; this technology is now in use in hospitals as far away as Pakistan • Profound Medical’s unique, less-invasive cancer therapy is improving outcomes for prostate cancer patients • Guelph biotech company PlantForm is using tobacco plants to produce low-cost treatments for cancer and HIV/AIDS • Toronto company Xagenic has developed a game changing device for diagnostics, with a handheld, lab-free tool that delivers results in only 20 minutes And this is only a short list. Everywhere we look, Ontario companies are pushing boundaries to create world-changing technologies.

Prime Minister Justin Trudeau recently stated that Canada is widely recognized for its resources, but we should be also known for our resourcefulness.

So, let’s get loud about our Ontario innovations.

If you’re an Ontario innovator, stand up and be counted. We will amplify your voice. Tweet us with the hashtag #OntarioInnovation, make yourself at home in the Ontario Pavilion, and join us at our international networking event.

And, if you’re an investor, there’s never been a better time to partner with Ontario’s world-class life sciences sector.

Biotechnology Focus

June/July 2016

25

BridGE@CCRM

By Shawn Lawrence

ONTARIO

The BridGE@CCRM:

Where cell and gene therapies go mainstream On January 13, 2016, the Government of Canada and GE Healthcare announced that they were each investing $20 million into the creation of a new Centre for Advanced Therapeutic Cell Technologies.

Located in the heart of downtown Toronto in the MaRS West Tower, the new facility, dubbed BridGE@CCRM, is scheduled to be up and running by the late fall and expectations are that it will usher in a new era of industrialization in the regenerative medicine field.

The Canadian Press Images/Centre for Commercialization of Regenerative Medicine/Salvatore Sacco

As Michael May, president and CEO of the Centre for Commercialization of Regenerative Medicine (CCRM), explains it will be a unique venue where solutions like scalable manufacturing processes for cell therapies are developed to help regenerative medicine companies, both big and small.

“We’re taking a unique approach to developing and testing new manufacturing and workflow solutions,” says May, adding that the way it has been done in the past, companies take a bright idea, create a prototype and throw it over the fence and see what customers think. “Here companies like GE and others will work handin-hand with the customers and end-users on the ground in creating the prototypes, and thus enable scalable, commercialized manufacturing of such therapies.”

26

Biotechnology Focus

June/July 2016

“We have built a strong industry consortium of nearly 50 companies to help drive a collaborative approach to realizing the potential of regenerative medicine, of which GE Healthcare plays a leading role” — Michael May Moreover, he says with GE as a partner, there is huge potential to capitalize on existing expertise and credibility in the industrialization space.

“I think the wonderful thing about this collaboration is that GE is one of the most significant infrastructure companies in the world. It already enables industrialization in so many other major industries. GE

Healthcare’s Life Science business already provides tools and technologies to people throughout the various parts of the market including basic research all the way through to clinical production and manufacturing,” he says.

On the other side of the table, Phil Vanek, general manager of cell therapy technologies at GE Healthcare’s Life Sciences busi-

BridGE@CCRM

ONTARIO “A lot of times when people think of scaling up, they think just of producing more cells, but scale up for cell therapies also includes additional upstream and downstream processes.”

— Phil Vanek

ness, is equally boastful of the merits of partnering with CCRM on this initiative.

Vanek cites Canada’s rich history in regenerative medicine and existing critical mass of companies in the space as driving factors behind the collaboration.

“Undoubtedly Toronto’s existing excellence in this field, and CCRM are well known in our industry.”

He adds that BridGE@CCRM fits very nicely with GE’s cell therapy business ambitions, but more importantly it is the right response to a growing need in the industry. Specifically that due to cell therapy’s relatively recent emergence as a therapeutic area, it is under-developed in terms of the industrialization technologies available to end users in this space.

He believes there’s a huge opportunity for BridGE@CCRM to fill this gap.

“It all starts with what’s currently available to cell therapy makers for meeting their manufacturing needs. The choices are very limited. As an example, autologous cell therapies or what we call vein-to-vein individualized treatments currently rely on tools adapted from other industries such as cord and blood processing, banking, cell culture etc. For many, it’s like cobbling together a factory floor with parts that aren’t designed to work together.”

Vanek says BridGE@CCRM will give GE Healthcare the chance to work directly with end users who are bringing cell therapies to market. It will also allow GE to be more efficient in advancing new technologies. A key part of the collaboration is that it is open to other partners and that the expectation is that therapeutic providers, companies or clinical researchers who have products that they want to bring through the clinic would utilize this centre to future proof their processes.

“We’re absolutely looking for other companies, whether they’re therapeutic companies that need those solutions or companies developing them,” states May. “That is why this centre is so unique, because it gives companies in the trenches reach to the customers, clients, patients and clinical trials.”

According to Vanek, GE’s willingness to work with other companies is two-fold. It comes down to, first, being on the front lines and finding those solutions while creating a place where prospective technologies can be tested out in a real world setting, with access to experts, ideas and customers, and getting to know what the real pain points are so that BridGE@ CCRM can tackle them.

“We recognize that within any manufacturing workflow there will ultimately be multiple components from multiple different providers of tools and technologies, and not just from GE,” he says. “We acknowledge that we won’t develop everything, so in certain areas we want to collaborate and try new things out, and work in a real-world cell therapy production environment workflow.”

Both May and Vanek stress this will be a safe environment for third parties to come and work with GE Healthcare and CCRM. On the topic of those points of pain, Vanek and May say one must first understand what exactly cell therapies are.

As the name implies, they are therapies that use cells to treat disease or replace tissue and there are many different types.

In many cases, cells are genetically engineered to make the product. This is where many of the logistical challenges exist, say both men, at the interface of biology and engineering, especially in terms of scale up and scale out. One major challenge that must be overcome is developing a therapy that provides the right kind of cells in the right quantity.

In terms of what is meant by scale up and scale out, an example of the latter is autologous therapies, where cells are removed from a patient and then returned to the patient after they’ve been manipulated to serve a certain function. Vanek breaks it down further into processes.

“A lot of times when people think of scaling up, they think just of producing more cells, but scale up for cell therapies also includes additional upstream and downstream processes,” he says.

“Upstream processes are all about iso-

lating from a mixture of cells the right cells you’re interested in, or selectively sourcing the cells and banking them.

Downstream processes include things like further purifying the cells, freezing them or preserving them so that they can be transported all over the world.”

In terms of challenges to scale up, both say that current manufacturing technologies simply can’t keep up with the high demand for quantity or throughput.

“Whether for financial reasons, or limitations in the technologies, we don’t have the ability to produce enough cells to treat the patients that need them,” says May. “One challenge is that most cells are grown on petri dishes in two-dimensions, on a surface, and when they fill up the surface you scrape them off and regrow them and you’re limited there by the surface area. So to get billions and billions of cells, you have to have rooms full of petri dishes, which are very expensive.”

Vanek offers up immunotherapy as a poster child for this expensive bottleneck.

“There’s lots of great clinical success and many companies have been formed on the backbone of this T-Cell immunotherapy technology, but if you look at where we are today in the market, it’s in the hundreds of patients that have been treated and yet the therapeutic promise for these therapies is going to have to address hundreds of thousands if not millions of patients,” says Vanek. “Ultimately, we have to take the industry from a position where we have the capability and wherewithal to treat hundreds to the point we’re able to treat the millions and that means improving the manufacturing process.”

Another challenge they hope to tackle at the new centre is finding ways to integrate the data side into the liquid side of the manufacturing process.

“The world is becoming much more digitized and so are the data on these cells, their genomics, proteomics, etc. They all need to be processed and integrated to do this optimization,” says May. On the workings of how the deal came Biotechnology Focus / June/July 2016

27

BridGE@CCRM together, May points to CCRM’s unique consortia model. He explains that CCRM was created five years ago to accelerate the development of regenerative medicine as well as cell and gene based products.

One of the important characteristics of its model was building very large and robust academic and industry networks. One of the major industry partners happens to be GE Healthcare.

“We have built a strong industry consortium of nearly 50 companies to help drive a collaborative approach to realizing the potential of regenerative medicine, of which GE Healthcare plays a leading role,” he says. In fact the two sides had talked often about combining their common interests and complementary expertise in the past.

CCRM Preliminary Program Layout Plan “As with most of the big initiatives, and increase productivity, establishing MaRS 10th Floor you engage with potential partners many clusters and creating collaborations betimes and then champion collaboration, tween the private sector and research and and if the timing is right, good things can post-secondary institutions. happen,” says May. “This is exactly how “The project was assessed against the things played out with GE and we both AMF guidelines and determined to be saw an opportunity to resource this in a good prospect for investment on the collaboration.” basis of three criteria: level of innovation, He adds that GE’s deep knowledge of market relevance and the potential for the bioprocessing industry, combined with spill-over economic benefits,” said the its global scale and health-care insights, spokesperson. made it the ideal anchor partner for the Among the spill-over economic benefits centre. But both parties knew they needed that are anticipated from BridGE is the one more party at the table to get the projcreation of 209 full-time jobs as well as ect launched. the maintenance of an additional 180 full“Ultimately, we tried to figure out a way time permanent jobs in Ontario. The presthis could possibly be funded and there ence of a successful global company like was the call from the Canadian governGE Healthcare is also considered a huge ment around the FedDev initiative to say, win for Ontario in terms of strengthening ‘hey, if you can bring a company to the its economic position. Moreover, experts table that’s willing to match the governalready forecast that the global market ment investment, we would be very interfor cell-based therapies will surpass the ested in having a manufacturing oriented US$20 billion mark by 2025, with an anposition in Toronto,’” recalls May. nual growth rate of 21 per cent. BridGE@ With GE as that partner, government CCRM presents a huge opportunity for readily backed the centre, and an inCanada to capture this growing market. vestment was made through the FedUltimately, says May, by having aceral Economic Development Agency for cess to new technologies developed at Southern Ontario (FedDev Ontario) from BridGE@CCRM, there is also potential its Advanced Manufacturing Fund (AMF). down the road to eventually build even The official news announcement for this larger manufacturing facilities in Ontario new facility drew so much attention that for this burgeoning subsector. even Canadian Prime Minister Justin “The idea being that processes develTrudeau was in attendance for the funding oped at BridGE@CCRM would be transannouncement. The reason being, regenferred over to these plants that could be erative medicine is a priority even at the built here if all goes well,” May says. On federal level for a host of reasons. this point, May says that the manufacturAs one FedDev Ontario spokesperson ing success of BridGE@CCRM has potenexplains, BridGE meets many of the objectial to create stickiness for other compatives of the AMF fund, specifically in terms nies in the regenerative medicine space. of supporting the development of cutting“If they manufacture here, they’ll likely edge technologies to improve processes stay here,” he says.

ONTARIO

Adjacent Tenant Space

28

Biotechnology Focus

June/July 2016

Legend

Programme Requirements

Meeting Offices Cubicles Carrels Kitchenette / Lounge Lab Lockers Filing Copy Core /Adjacent Tenant Storage/Mech/Comms

Room Area

Exec Office Offices Cubicles Carrels Boardroom Meeting Rooms Kitchenette Lockers Storage Filing Cabinet Pods Printer Pods IT Closet

# Required 4 6 15 55 1 5 1 70 1 2 2 1

Actual 4 6 11 45 1 5 1 70 1 6 2 1

He adds also that the new technologies and devices tested at the facility by academic partners might also one day be bundled to create new companies. It all fits very well into the larger CCRM puzzle of creating a sustainable regenerative medicine cluster.

Already we’re seeing the model work.

For example, on CCRM’s 40,000 sq. foot floor at MaRS is BridGE@CCRM and CCRM already has a partnership with UHN to operate a GMP facility to make cells for early stage clinical trials. Also sharing floor space is the University of Toronto’s Medicine by Design, a $114 million academic initiative to create new discoveries and technologies at the front end of regenerative medicine. And again on the same floor there’s the headquarters for the Ontario Institute for Regenerative Medicine (OIRM), a provincially funded initiative that brings together all of the institutions and most of the scientists in the field. CCRM is the commercial partner for both of these initiatives: Medicine by Design and OIRM. “It really is part of our overall strategy to leverage this activity and collaboration into bringing the best investments to town, and we don’t like to sit still,” says May. He adds that while there are a lot of collaborations going on the world where one plus one equals two, everything that CCRM is doing here is driving towards one plus one equals 10.

“I think that bringing all these things together, with everything else we’re doing, demonstrates that it’s possible,” says May. “Canada discovered stems cells, and so really we should be a leader at making them and I think this facility is a a means to make this happen.”

Vanek is equally excited by all this activity.

“This partnership has been well received by the industry and GE leadership.

We’re very excited about cell therapy and this is a great first step for GE to help industrialize the cell therapy industry.”

To see this story online visit http://biotechnologyfocus.ca/ made-in-ontario/

CALENDAR JUNE 2016 June 5-9 Canadian Chemistry Conference and Exhibition Venue: Halifax, NS Tel: 613-232-6252 Web: www.csc2016.ca

September 13-15

Tel: 604 681 2153 x 109 Fax: 604 681 1049 Email: IUBMB2016@icsevents.com Website: iubmb2016.org

Biopharm America Venue : Boston, MA Web : http://www.ebdgroup.com/bpa/index. php

SEPTEMBER 2016

new card:Layout 1 1/31/2013 9:09 AM Page 1

OCTOBER 2016

June 6-9

September 12-15

2016 BIO International Convention Venue: San Francisco, CA Web: www.convention.bio.org/2016

48th Annual Canadian Mineral Analysts (CMA) Conference and Exhibition Venue: Sudbury, ON Tel. 705-670-5643 Fax. 705-670-3047 E-mail: ed.debicki@ontario.ca Web : www.2016cma.com

June 11-14 CALAS Annual Symposium Venue: Toronto, ON Tel: 416-593-0268 Web: www.calas-acsal.org/symposium

October 16-19 Canadian Chemical Engineering Conference Venue : Quebec City, QC Tel : 613-232-6252 Web : www.csche2016.ca

SRC101

June 12-15 Canadian Society for Microbiologists Annual Conference Venue: Toronto, ON Tel: 613-421-7229 Email: info@csm-scm.org Web: www.csm-scm.org/english/conf_upcoming.asp

June 14 MedEdge Summit Venue: Richmond Hill, ON Livestream: https://livestream.com/accounts/1705310/events/4100246 Web: www.businessrichmondhill.ca/MedEdge/

June 14-16 Canadian Biosafety Symposium Venue: Montreal, QC Tel: 306-966-8496 Web: http://biosafety.icid.com

June 23 RESI (Redefining Early Stage Investments) Venue: Toronto, ON Tel: 617-600-0668 Email: resi@lifesciencenation.com Web: http://www.resiconference. com/#!mars-2016/qpp86

JULY 2016 July 17-19 Labcon 2016 Venue: Charlottetown, PEI Tel: 905-667-8688 Email: labcon@csmls.org Web: http://labcon.csmls.org/en/

I wish to receive/continue to receive a complimentary subscription to

print

digital

No

both

Signature:____________________________________Date: ___________________________

Name:_________________________________Title: ____________________________________ Company: ______________________________Dept: ___________________________________ Business Address : _______________________________________________________________ City:_____________________________Prov: __________Postal Code: ____________________ Telephone: ___________________________Fax: ______________________________________ E-mail: ________________________________________________________________________ On occasion, BIOTECHNOLOGY FOCUS will send third-party information on products & services related to the lab and life science industries. These may be cancelled at any time. Please check here if you do NOT wish to receive these.

JOB FUNCTION 50 C Level Management 50a Engineer 51b Principal Investigator / Research Scientist 51c Professor / Faculty 53a Group / Project Leader 53b Procurement / Supply Chain 53c R&D Director / Manager 54 Legal / Financial / Administration / Regulatory / Communications

55 58 59 60 62 63 64 65 99

Post Doc / PhD Student Professional Services / Consulting Lab Technician / Research Assistant Sales / Marketing IT Management Nurse / Practitioner Pharmacist Physician Other (Specify):

50 50a 50b 50c

Academic Biotech Organization Clean Tech organization Contract Research / Manufacturing Organization 50d Diagnostics Organization 50f Food processing / manufacturing 50g Instrument Manufacturer / Distributor

50h Packaging / Distribution 50i Pharmaceutical Organization 50j Professional Services (legal, financial, consulting, recruiting, regulatory, communications) 52 Clinical Research / Hospital 53 Research Institute 55 Government Agency 99 Other (specify):

C86 Buying Influence A Authorize

B Recommend

C87 Which products are used in your lab?

A B C D E F G

Analysis Instruments Basic Lab Equipment Chemicals/Biochemicals Chromatography – Gas Chromatography – Liquid Filtration, Water Purification LIMS

H I J K L M Y

Liquid Handling & Sample Prep Microscopes, Optics, Cameras Safety & Hygiene Spectroscopy Testing Systems/Equipment Vacuum Equipment None of the Above

C88 Product Development Stage (check all that apply)

COMPANYs PRIMARY BUSINESS ACTIVITY

July 17 -21 International Conference of Biochemistry & Molecular Biology Venue: Vancouver, BC

Yes

BIOTECHNOLOGY FOCUS Format Preference:

C Specify

A Research/Development B Pilot/Scaleup

C Production/Manufacturing D Tech Transfer E Not applicable

C89 Are you building a new lab?

Yes

No

C90 We have the following enewsletters: 1 2 3 4

Biotechnology Focus eBulletin Laboratory focus eBulletin BioPharma BioMedical

5 Health Care 6 Agri-Food 7 Clean Tech 8 Industry Inte

For a quick response please fax: 905-727-4428 or e-mail: circulation@promotive.net Biotechnology Focus / June/July 2016

29

| By Dr. Raphael Hofstein President & CEO, MaRS Innovation and Elizabeth Monier-Williams Director, Marketing & Communications, MaRS Innovation

LAST word

Canada’s Contagiously Creative Life Sciences Sector

T

here’s a historical trend in Canadian culture with farreaching effects for the way we do business, ranging from entertainment to academia to science. Namely, it’s that until quite recently, Canadians weren’t into cultivating national star systems.1 As Gideon Hayden recently wrote for TechCrunch, if you’re a Canadian tall poppy who made it big, there’s a good chance you struck out on your own by leaving the country. After you succeeded in Hollywood, Silicon Valley or another business arena of your choice, Canada recognized your achievements and welcomed you home with open arms.

In the life sciences, as in music, Olympic podiums and other areas of Canadian life, this stereotype is changing. As we build more robust technology clusters, we’re becoming more comfortable with building Canadian excellence here, within our borders.

We’re also seeing signs that the innovation economy is indeed on the cusp of helping our nation make the transition, in Prime Minister Justin Trudeau’s words, “from a nation of resources to one known for its resourcefulness.” That means supporting home-grown ideas, technology and companies.

Here are some of those telling signs in Canadian technology development, business transactions and public recognition.

First, scientists from Toronto’s Sunnybrook Health Sciences Centre made history when they used ultrasound to non-invasively penetrate the blood-brain barrier, a last frontier in international efforts to more effectively transport chemotherapeutic agents across the blood-brain barrier and into the brain tumours of patients. Their elegant solution to the pervasive challenge of getting drugs inside such tumours will lead to new, more effective treatments with fewer side effects. While it will be years before their discovery reaches patients, Triphase Accelerator’s recent advances in other clinical trials for brain cancer treatments, made in partnership with Celgene, are part of the growing signal that we can expect huge growth in Canada’s life sciences in this area.

In Vancouver, Zymeworks orchestrated one of the largest life sciences transactions for Canada last year through their acquisition of Kairos. The deal, facilitated by our colleagues at the Centre for Drug Research and Discovery (CDRD), will augment the efficiency and efficacy of Zymeworks’ biologics, designed for high accuracy to focus on antibody drug conjugates (ADC).

Within MaRS Innovation’s portfolio, Encycle Therapeutics’ nacellins are penetrating cell membranes to focus on intracellular disease-related targets. This Canadian company, emerging from the laboratory of Dr. Andrei Yudin at the University of Toronto, attracted investment from Canadian and international venture groups for their recent seed round, led by Japan’s Takeda Ventures. Another developing story revolves around Vasculotide, the lead asset of Vasomune Therapeutics from Sunnybrook Health Sciences Centre, which demonstrates promise in stabilizing patient vasculature (e.g., kidneys, lungs, etc.) in diseases associated with vascular leakage.

Further afield, Canadian researchers are also leading technology developments in immuno-oncology, the art of training the immune system (T-cells) to recognize cancer cells as foreign to the body and therefore suitable for immune attack. Many Canadian start-ups are emerging in this scientifically hot area, as well as in

30

Biotechnology Focus

cancer stem cells, which represent another notable hot spot. In April 2016, Abbvie-Abbot acquired California-based Stemcentrx in a $5.8-billion transaction (USD) because of their progress in attacking cancer stem cells, the progenitors of cancer cells. Likewise, fibrosis represents a significant unmet medical condition in need of next-generation medical treatments; companies like Novosis, emerging from MaRS Innovation’s research community, offer world-class approaches for fibrosis control. We can expect more national interest in commercializing technology in all of these promising research areas.

Global trends were also prevalent in the Gardiner Foundation’s awards this year. The award committee of Canada’s most significant prize in science signalled their awareness of world scientific events by recognizing all five researchers who were instrumental in developing CRISPR. The Globe and Mail’s Ivan Semeniuk described this discovery as “a revolutionary gene-editing technique that carries enormous potential for many areas of biology and medicine.”

As recently as last January, Prime Minister Trudeau signalled Canada’s growing strengths in regenerative medicine by joining with Minister Navdeep Bains and Minister Chrystia Freeland to open the Centre for Commercialization of Regenerative Medicine’s GMP facility in Toronto. Expect to see movement in companies like ExcellThera, which specializes in the advanced technology that enables selective expansion of stem cells extracted from cord blood, to be among the facility’s first projects.

To build a constellation of Canadian life sciences stars, earlystage companies need a continuum of support as they launch, develop and scale. The federally-funded Centres of Excellence for Commercialization and Research (CECR) program is unparalleled in how it embraces, packages and advances the most promising technologies. As a CECR, MaRS Innovation believes that early partnerships with industry are crucial to our ability to accelerate these bright lights. We formed strategic partnerships with six global pharmaceutical companies; the ensuing constructive relationships allowed us to elevate their awareness of the innovation happening in Canada. Last month’s arrival of Johnson & Johnson’s JLABS @ Toronto incubator is indicative of the growing international awareness of, and interest in, the high calibre of Canada’s discovery work.

Einstein famously said, “Creativity is contagious. Pass it on.” In 2016, there’s every indication that Canadian creativity is becoming contagious, and making its impact felt across the life sciences.

How will you pass it on?

References 1. Reporter, TV host and blogger Elaine Lui has written extensively on this topic in the entertainment context. See http:// www.laineygossip.com/Martin-Short-honoured-with-LifetimeAchievement-Award-at-the-Canadian-Screen-Awards-and-Introfor-March-14--2016/43314.

Got something to say? Please send your comments/letters to biotechnology_focus@promotive.net

June/July 2016

15165

Give our kids every chance to get better.

PUT YOUR MONEY WHERE THE MIRACLES ARE.

JENNIFER LOPEZ ACTRESS, MUSICIAN, TV PERSONALITY, MOM

Like all moms, I’m always concerned about my children’s well-being. But sometimes they get sick. Sometimes they get hurt. That’s why I’m so grateful we have children’s hospitals. If any child needs a miracle, they’ll do everything in their power to make one happen. Please join me in giving sick and injured children every chance to get better. Put Your Money Where the Miracles Are. Give to your Children’s Miracle Network member hospital.

Children’s Miracle Network® raises funds and awareness for 170 member hospitals, 14 of which are in Canada. Donations stay local to fund critical treatments and healthcare services, pediatric medical equipment and research. Its various fundraising partners and programs support the nonprofit’s mission to save and improve the lives of as many children as possible. Find out why children’s hospitals need community support, identify your member hospital and learn how you can Put Your Money Where the Miracles Are, at childrensmiraclenetwork.ca and facebook.com/CMNHospitals.

Give Today

ChildrensMiracleNetwork.ca

15165 JLOPYM_fullpage_color_CA_GENERIC.indd 1

to your children’s hospital foundation

12/17/15 1:59 PM

NEW: CryoCube® ULT Freezers

Efficiency Reinvented CryoCube Ultra-low Temperature Freezers CryoCube ultra-low temperature freezers combine maximum sample security with improved functionality. New advancements decrease power consumption and make CryoCube freezers among the most energy efficient in the industry.

Eppendorf quality means years of trouble-free operation and dependable support.

> New automatic vent port on front door allows for easy re-entry into your freezer while also improving energy efficiency > New ergonomic handle requires less force and improves freezer access > New magnetic closures on insulated inner doors further increase ease-of-use

www.eppendorf.com • 800-263-8715 130.A1.0109.B © 2015 Eppendorf AG.

C130.A1.0109.B.CA-LFO.indd 1

Full page tabloid ad Laboratory Focus – LFO

7/9/15 12:14 PM