Biotechnology Focus June/July 2018 by Promotive Communications

INSIGHTS FOR THE LIFE SCIENCE INDUSTRY

JUNE/JULY 2018 VOLUME 21, NUMBER 3

Ont editario ion

Going deep into

the human microbiome with Adapsyn Bioscience

INSIDE:

Q&A with Ontario’s first Chief Scientist Tackling Alzheimer’s Pursuing Longevity in Cell Therapies

Publication Mail Registration Number: 40052410

29 Ontario steps up to play on the global stage Features 12 Hope beyond the hype: The pursuit of longevity in cell therapies By proving that cell therapy can be effective, researchers have won an early battle – but to win the war they must ensure that this therapy is effective and accessible for the tens of thousands of recipients in the years to come. By Erika Siren

23 Going deep into the human microbiome with Adapsyn Bioscience Adapsyn Bioscience mines the landscape of highly-evolved small molecules from microorganisms to uncover a diverse portfolio of novel drugs to treat a wide spectrum of diseases. By Michelle Currie

29 Ontario steps up to play on the global stage 16 KalGene Pharmaceuticals and its partners bring an innovative, collaborative approach to tackle Alzheimer’s disease KalGene Pharmaceuticals has taken the road-less travelled approach to its clinical program, looking to slow down disease progression by developing a treatment that can penetrate the blood-brain barrier. By Anthony Boone

18 Q&A with Molly Shoichet: Ontario’s first Chief Scientist This past year, the government of Ontario founded a brand-new position to provide a platform for which Ontario can continue to grow its innovation-based economy and promote Ontario as an integral research hub and a top destination for global talent. Compiled by Michelle Currie

There have been many investments made and new capital that will fuel innovation within the province and economy. By Michelle Currie

30 Seizing the day: How can we capitalize on Ontario’s life sciences opportunity A surge of momentum for our companies, along with a provincial election, has created an unprecedented opportunity to capitalize on Ontario’s life sciences economy. By Jason Field

Departments 7 Research News 9 Business Corner

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Innovative Ontario companies in the health sciences space

June/July 2018 BIOTECHNOLOGY FOCUS 3

june/july 2018 | volume 21 | number 3

www.biotechnologyfocus.ca

Contents

PERSPECTIVES 11 Brampton’s health and life sciences sector is powering opportunities and success in the Canadian and global life sciences ecosystem

ONTARIO

Punching above its weight, the city is creating a foundation for success and building strong health partnerships that surround innovation wellness through preventative care. By Michelle Currie

15 Use it or lose it: The future of Canada’s health science industry Strong health science clusters contribute enormous benefits to their economies and societies; and while Ontario’s health science industry is beginning to mature, there remains significant barriers to scaling up. By Gail Garland

22 The oncology entrepreneur’s secret weapon: navigating commercialization with FACIT For many entrepreneurs and universities, commercialization is a serial process that follows an invention disclosure to the Institution. Fortunately, oncology entrepreneurs in Ontario have a secret weapon: FACIT. By David O’Neill

25 Small biotech start-ups drive innovation Small start-ups, not big pharmaceutical organizations, are now at the heart of innovation in health-related biotech. By Karen Ramlall

26 Regenerative medicine experts in Ontario aim to strike the right balance by stimulating commercialization and reducing healthcare costs Stem cell researchers in Ontario, supported by the Ontario Institute for Regenerative Medicine, are making strides towards treatments that could have a big mpact on the province’s economy. By Krista Lamb

28 The battle to control CRISPR IP continues The potential applications of CRISPR system have led to a significant amount of interest in protecting the intellectual property associated with this technology. By Ainslie Parsons, Carmela De Luca 4 BIOTECHNOLOGY FOCUS June/July 2018

ONTARIO:

LIFE-CHANGING SCIENCE

LIVES HERE

The technological convergence that intertwines genomics, artificial intelligence and biotech is allowing Ontario to perform life-changing science. From regenerative medicine to synthetic biology, we are transforming the landscape of biotechnology. In the words of Ontario’s first Chief Scientist, Dr. Molly Shoichet, “What could be more exciting than inventing the future?”

#OntarioBIO2018

VISIT ONTARIO IN BOOTH 551 InvestInOntario.com/BIO2018

Paid for by the Government of Ontario.

PUBLISHER’S note PUBLISHER/ EDITOR-IN-CHIEF Writer CONTRIBUTING WRITERS

Terri Pavelic Michelle Currie Anthony Boone

Gail Garland Karen Ramlall David O’Neill Krista Lamb Erika Siren Jason Field

It’s that time of year again where we are putting the spotlight on Ontario and showing the world at this year’s BIO event in Boston that Ontario really is ‘Yours to discover’. There has been some dramatic growth and new opportunities within the last year that our authors have decided to share with all of you. One of those that arrived on the horizon was the creation of a new position within Ontario’s government to help glue the public, government, and life sciences sector together to advance Ontario on the world stage. Molly Shoichet, Ontario’s first-ever Chief Scientist, goes in depth about the province’s vision as well as her own in the Q&A held with Biotechnology Focus’ writer, Michelle Currie. With an ever-increasing aging population, and roughly 46 million people worldwide suffering from dementia, Anthony Boone takes us away to explore the fundamental work KalGene Pharmaceuticals plays at breaking down the blood-brain barrier in their road-less-traveled approach to its clinical program to end this debilitating disease in his article: KalGene Pharmaceuticals and its partners bring an innovative, collaborative approach to tackle Alzheimer’s disease. After years of intensive research and development, cell therapies are finally having their moment. By proving that cell therapy can be effective, researchers have won an early battle – but will they win the war? Erika Siren walks us through what is ongoing with cell therapy across the country in her article Hope beyond the hype: The pursuit of longevity in cell therapies. We go deep into machine learning and artificial intelligence when speaking with Adapsyn CEO, Nathan Magarvey. A spinout company from McMaster University that focuses on the human microbiome by translating genes to proteins to small molecules. Read the article by Michelle Currie, Going deep into the human microbiome with Adapsyn Bioscience. Strong health clusters can contribute enormous benefits to economies and societies, which is what Gail Garland discusses in her article Use it or lose it: The future of Canada’s health science industry. Although Ontario’s health science industry has begun to mature, there is still much work to do. Within the article are some of the industry’s recommendations. The Centre for the Commercialization of Antibodies and Biologics (CCAB) Communications manager writes about how it’s small biotech start-ups that truly fuel innovation, not big pharmaceutical organizations. She goes into detail to let us know what their labs are bringing to the table for the life sciences sector in Canada in the article Small biotech start-ups drive innovation. The Government of Ontario surprised us again this year with the announcement of their venture capital fund that will provide significant financing devoted to the life sciences sector. We have also seen international capital coming into the province with the collaboration between MaRS Innovation and South Korea. Read more about it in the article Ontario steps up to play on the global stage. Then to top off another Ontario-based issue, we have Life Sciences Ontario (LSO) president and CEO, Jason Field, describing how a momentum for our companies, along with a provincial election has created an unprecedented opportunity to capitalize on Ontario’s life sciences economy.

Ainslie Parsons Carmela De Luca GRAPHIC DESIGNER CONTROLLER MARKETING MANAGER

Elena Pankova John R. Jones Melisa Sukhdeo

CIRCULATION DIRECTOR Mary Labao circulation@promotivemedia.ca Tel: 905-841-7389

EDITORIAL ADVISORY BOARD Barry Gee, CDRD; Christine Beyaert, Roche Canada; Nadine Beauger, IRIC; Peter van der Velden, Lumira Capital; Jason Field, Life Sciences Ontario; John Kelly, KeliRo Company Inc.; Raphael Hofstein, MaRS Innovation; Gail Garland, OBIO; Albert Friesen, Medicure Inc.; Andrew Casey, BIOTECanada; Ulrich Krull, UTM; Peter Pekos, Dalton Pharma Services

Biotechnology Focus is published 6 times per year by Promotive Communications Inc. 1-226 Edward Street, Aurora, ON L4G 3S8, 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 department – 1-226 Edward Street, Aurora, ON L4G 3S8 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.

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6 BIOTECHNOLOGY FOCUS June/July 2018

R&D news Positive results from Prometic’s pivotal phase III trial

Prometic Life Sciences announce positive clinical data from its pivotal IVIG phase 3 clinical trial, meeting its clinical primary and secondary endpoints in adult patients suffering from primary immunodeficiencies (PID). The clinical data was presented at the

Clinical Immunology Society Annual Meeting in Toronto on April 27-28 2018. The primary endpoint is the rate of clinically documented serious bacterial infections (SBIs), defined as bacterial pneumonia, bacteremia, and septicemia, osteomyelitis/ septic arthritis, bacterial meningitis or visceral abscess. The FDA Guidance for Industry on studies required to support marketing of IGIV states: “…a statistical demonstration of a serious infection rate per person-year less than 1.0 is adequate to provide substantial evidence of efficacy”. Since there were no SBIs observed during the study, Prometic IGIV 10% clearly meets this requirement. Secondary endpoints including episodes of fever (≥100.4°F), number of missed days, number of days of hospitalization due to infection, number of days on antibiotics, number of infections other than SBI, and trough IgG level were comparable between Prometic’s IGIV and commercial drugs. Only 4.94 days/subject/year were lost from work with Prometic IGIV 10%, which was signifi-

CCRM:

Commercializing Living Therapies CCRM is a not-for-profit organization that enables our partners to develop and commercialize cell and gene therapies, and regenerative medicine technologies. Our business consulting services include regulatory affairs, technology reviews, market assessments, patent strategy, and defining commercial paths. Our development lab services include cell reprogramming and engineering, technology translation, and protocol/process development. Our Centre for Advanced Therapeutic Cell Technologies specializes in current and future challenges in cell and gene therapy manufacturing, with expertise in scale up/out manufacturing and bioreactor optimization, media development, and downstream product concentration, purification and packaging. Coming in 2019, we will offer contract manufacturing of cell and gene therapies for early-stage clinical trials in our Good Manufacturing Practices (GMP) facility. Learn more at ccrm.ca.

cantly less than the rate observed while on a commercial product. The proportion of infusions for which at least one treatment-emergent adverse events (TAAE) that was reported was well within FDA guidance threshold across all time points within 72-hours post-infusion. Overall Prometic IGIV 10% appeared to be well tolerated and was not associated with any SBIs. “The results with Prometic IGIV 10% met the FDA Guideline requirements for both safety and efficacy. This is the second plasma derived therapeutic clinical program to generate positive pivotal phase 3 results” says Pierre Laurin, president and CEO of Prometic. “This demonstrates the ability of our PPPS platform to generate positive data from both rare proteins targeting rare diseases as well as more commodity like proteins”. To see this story online visit https://biotechnologyfocus.ca/positiveresults-from-prometics-pivotal-phaseiii-trial/

CCRM Supports Your Regenerative Medicine Innovations from Concept to Market CCRM offers business and technical services tailored to your company’s stage, timelines, ﬁnancing and needs. Our deep expertise in the manufacturing workﬂow provides a comprehensive approach from discovery to commercially-ready manufacturing solutions. CONTACT US ccrm.ca cdmo@ccrm.ca 416-978-3751 June/July 2018 BIOTECHNOLOGY FOCUS 7

R&D news University of Toronto researchers on their way to revolutionizing burn care

From left to right, Associate Professor Axel Guenther, Navid Hakimi and Richard Cheng have created the first ‘skin printer’ that forms tissues in situ for application to wounds (Photo Credit: Liz Do) Skin is constructed of three layers – the epidermis, dermis, and hypodermis – that could all be damaged in patients with deep wounds. The current ideal treatment for deep skin wounds is called split-thickness skin grafting. This method of treatment takes healthy donor skin and grafts it onto the surface epidermis and part of the underlying dermis. However, with large deep wounds, a significant amount of healthy donor skin is required to

graft all three layers and is typically not available. This leaves a partially covered wound or an improperly covered wound that leads to poorer healing outcomes. This ‘skin dispenser’ could offer a solution to these barriers, and works a lot like a white-out tape roller, but on a much larger scale with a microdevice that forms tissue sheets instead of a tape roll. Vertical stripes of “bio-ink” made up of protein-based bio-

materials including collagen run along the inside of each tissue sheet. Collagen is the most abundant protein in the dermis, and fibrin, a protein involved in wound healing. “Most current 3D bioprinters are bulky, work at low speeds, are expensive and are incompatible with clinical application,” explains Guenther. “Our skin printer promises to tailor tissues to specific patients and wound characteristics,” adds Hakimi. “And it’s very portable.” The handheld device is the size of a small shoebox and weighs less than a kilogram. It also requires minimal operator training and eliminates the washing and incubation stages required by many conventional bioprinters. Although many tissue-engineered skin substitutes exist, they are not yet widely used in clinical settings. This entices the researchers to add several capabilities to the printer, including expansion of the coverable wound size. With the team combining forces with Sunnybrook Hospital, they have more plans to perform in vivo studies with clinical trials on humans. This brilliant technology has the opportunity to literally shape skin and revolutionize the face of burn care. To see this story online visit https://biotechnologyfocus.ca/university-of-toronto-researchers-on-their-wayto-revolutionizing-burn-care/

Health Canada approves Latuda to treat adolescents with bipolar depression Sunovion Pharmaceuticals Inc. announces that Health Canada has approved the Supplemental New Drug Submission (SNDS) that expands the use of Latuda (lurasidone HCI) to include the acute management of depressive episodes associated with bipolar I disorder in adolescents (13 to 17 years of age). Latuda is currently indicated in Canada for the management of the manifestations of schizophrenia in adults and adolescents (15 to 17 years of age) and the acute management of depressive episodes associated with bipolar I disorder in adults. Bipolar disorder is a chronic mental health condition that can affect individuals of all ages, and is characterized by potentially debilitating severe mood swings, including periods of depression and mania. Bipolar disorder is the fourth leading cause of disease burden among children and adolescents worldwide. 8 BIOTECHNOLOGY FOCUS June/July 2018

In Canada, more than 730,000 people aged 15 years and older report symptoms that meet the criteria for bipolar disorder. The SNDS is supported by data from a Phase 3 clinical study of children and adolescents (10 to 17 years of age) with bipolar depression. In this study, Latuda was associated with statistically significant and clinical-

ly meaningful improvement in bipolar depression symptoms compared to placebo and was generally well-tolerated. “This approval marks an important milestone for the mental health community in Canada, where few approved treatment options are available to adolescents with bipolar depression,” says David Frawley, executive vice president, chief commercial officer at Sunovion. Latuda comes with some potentially serious side effects, including stroke, neuroleptic malignant syndrome, uncontrolled body movements, and increased mortality in elderly patients with dementia. To see this story online visit https://biotechnologyfocus.ca/healthcanada-approves-latuda-to-treat-adolescents-with-bipolar-depression/

BUSINESS corner Zymeworks and Celgene to expand bispecific antibody collaboration Vancouver-based Zymeworks Inc., a biopharmaceutical company, and the biotech giant Celgene Corporation announce that they have expanded their collaboration agreement for the research, development, and commercialization of bispecific antibody therapeutics using Zymeworks’ Azymetric platform. “The team at Celgene has made excellent progress developing bispecific and multifunctional therapeutic candidates built on our industry-leading Azymetric platform and we are delighted to expand and continue our relationship with them,” says Ali Tehrani, Ph.D., Zymeworks’ president & CEO. “We are proud of the fact that all six of our pharma partnerships involving the Azymetric platform remain active and are advancing innovative therapeutics towards clinical trials.” The Azymetric platform enables the transformation of monospecific antibodies into bispecific antibodies, giving the antibodies the ability to simultaneously bind two different targets. Azymetric bispecific technology enables the development of multifunctional biotherapeutics that can block

multiple signaling pathways, recruit immune cells to tumours, enhance receptor clustering degradation, and increase tumour-specific targeting. These features are intended to enhance efficacy while reducing toxicities and the potential for drug-resistance. Azymetric bispecifics have been engineered to retain the desirable drug-like qualities of naturally occurring antibodies, includ-

ing low immunogenicity, long half-life and high stability. In addition, they are compatible with standard manufacturing processes with high yields and purity, potentially significantly reducing drug development costs and timelines. Under the terms of the original collaboration agreement signed in 2014 which enabled Celgene to research and develop multiple bispecific antibodies based on the Azymetric platform, Celgene has now exercised its right to increase the number of potential products it can develop and commercialize from eight to ten, and extended the research program term by two years. Zymeworks will receive an expansion fee and is now eligible to receive up to US$164 million in development and commercial milestones for each of up to 10 products plus royalties on worldwide sales. In total, Zymeworks is now eligible to receive up to US$1.64 billion in future payments for the entire collaboration. To see this story online visit https://biotechnologyfocus.ca/zymeworks-and-celgene-to-expand-bispecificantibody-collaboration/

bereskinparr.com June/July 2018 BIOTECHNOLOGY FOCUS 9

BUSINESS corner Joint venture in BC funds $45-M to STEMCELL technologies

Canada’s largest biotechnology company, STEMCELL Technologies, announces a $45 million joint funding agreement between the Government of Canada and British Columbia for the company to build a state-of-the-art manufacturing facility. This agreement is opening the doors wide to accelerate STEMCELL by providing cutting-edge reagents and tools to support life science researchers around the world working in the cell therapy and regenerative medicine fields. The $138 million project will take 5 years to build and will manufacture products at the higher regulatory compliance standard required to support clinical trials in the rapidly evolving sciences of cell therapy, tissue engineering, immunotherapy,

gene therapy and regenerative medicine, with the eventual aim of curing cancer and other serious diseases. This initiative will provide and retain biotech jobs on Canadian soil and show the world that Canada is strong in the future of regenerative medicine. It will also ensure that products invented in Canada will continue to be commercialized and manufactured on Canadian soil before being sold to researchers around the world. The $45 million government investment— divided evenly between the Provincial and Federal Governments—is combined with STEMCELL’s own investment of $93 million to complete the facility between now and 2023. The Provincial portion results from the need to support well-paying, high-tech

manufacturing jobs that will remain anchored in BC by this new advanced manufacturing facility. “This is the kind of opportunity our government looks for—one that can help make a real difference in the health and quality of life for people here at home and abroad,” says The Honourable Bruce Ralston, British Columbia Minister of Jobs, Trade and Technology. “As we continue to work with companies and partners in life sciences research, we are helping create a global hub for B.C. that will allow us to create jobs for British Columbians and attract more talent and investment to our province.” The Federal contribution will come from the Strategic Innovation Fund Program managed by the Ministry of Innovation, Science and Economic Development. “Canadians paved the way in stem cell research; now we’re investing in STEMCELL Technologies to help commercialize this success,” states The Honourable Navdeep Bains, Minister of Innovation, Science and Economic Development. “The Government of Canada’s Strategic Innovation Fund investment will create nearly 700 jobs and advance life-saving innovations that will secure STEMCELL’s place in the global supply chain for years to come.” To see this story online visit https://biotechnologyfocus.ca/jointventure-in-bc-funds-stemcelltechnologies-with-45-m/

Montreal-based company BioAmber files for bankruptcy BioAmber files for a voluntary petition for relief under chapter 11 of the United States Bankruptcy Code and it’s two Canadian subsidiaries, BioAmber Sarnia Inc. and BioAmber Canada Inc. file for a Notice of Intention to make a proposal under the Bankruptcy and Insolvency Act. The company has one of the largest succinic acid production facility’s in the world in Sarnia, Ontario that opened in the spring of 2015, employing roughly 60 people. Their mission was to be a fast-growing producer of chemical intermediates that uses sugars instead of fossil fuels and sell competitively priced, sustainable chemicals with strong profit margins to have the cleanest environmental footprint in the industry. Appearing to have been riding the edge of 10 BIOTECHNOLOGY FOCUS June/July 2018

the knife for the past several years, recently losing their founding president and being delisted from the Toronto and New York Stock Exchange, it has been a trying time for this Canadian company. They lost buckets of money back in 2013, but investors kept on coming regardless. Still rolling with the punches, BioAmber believes that filing for these procedures will be in the best interest of the stakeholders and will best facilitate its efforts to renegotiate its debt and raise the funds needed to continue operation. “This process will provide BioAmber with the time and stability to restructure its finances,” says Richard Eno, chief executive officer of BioAmber. “This restructuring, combined with the significantly improved

cost structure we anticipate, will position BioAmber to emerge as a much stronger company which will be better positioned to meet the growing global demand we see for our product.” There can be no guarantee that the company will be successful in securing further financing or achieving its restructuring objectives. Failure by the company to achieve its financing and restructuring goals will likely result in the company and/or its subsidiaries being forced to cease operations and liquidate its assets. To see this story online visit https://biotechnologyfocus.ca/montreal-based-company-bioamber-files-forbankruptcy/

Perspective By Michelle Currie

ONTARIO

Brampton’s Health and Life Sciences sector is powering opportunities and success in the Canadian and global life sciences ecosystem Brampton has become a game-changer in the Canadian and global life sciences space. Punching above its weight, the city is creating a foundation for success and building strong health partnerships that surround innovation wellness through preventative care. Clustered into the country’s largest life science hub, Brampton is the second fastest growing city in Canada, traversing many transcontinental highways, next door to Pearson International Airport and located in the centre of the innovation super corridor makes this a leading location for companies and the economy to flourish and grow. A recent phase 1, $500-million investment into the new Peel Memorial Centre for Health and Wellness, which is part of the William Osler Health System, truly makes this centre the nucleus for the area. With four available acres on the site to host future medical lab incubator space for digital health companies and other medical-related firms, it is rife with opportunity. Phase 2 has been approved for development and could be another $300 million investment of the expansion of the

facility, which will lead to further development opportunities. Brampton separates themselves from the pack with their focus on preventative care. They want to be a facilitator to make Brampton an attractive and livable space not only for investing companies, but for everyone that shares the space as outlined in Brampton’s Vision 2040. Already, the city has been beginning to see clustering of companies with some of them working together to create an illustrious economy for the whole community. Brampton strives to connect the ecosystem by partnering with life science organizations across the GTA to provide ample opportunity for support and growth. Ryerson University is also moving into Brampton and will be bringing with them potential for more start-up companies and learning opportunities – especially in the digital health space or precision medicine – to partner with Osler, Ryerson as an incubator, or a global partner. This growing economy is leading the way to a successful life sciences ecosystem for all parties involved and for all of those who live in this thriving city. Brampton.ca/business

BRAMPTON’S

Health & Life Sciences Sector

Feel free to judge us by the companies we keep Join industry-leading businesses like Medtronic Canada, Dynacare, Taro Pharmaceuticals and Canadian Blood Services who call Brampton home. A young, educated, highly skilled and multicultural workforce. A strong economy. World-class transportation corridors to major markets. Room to grow. Need we say more? Discover why we’re the best business decision you’ll ever make. brampton.ca/business

Brampton, Ontario, Canada

BRAMPTON

ECONOMIC DEVELOPMENT & CULTURE

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2018-04-10 2:57 PM

stem cells

By Erika Siren

Hope Beyond the Hype: The Pursuit of Longevity in Cell Therapies

fter years of intensive research and development, cell therapies are finally having their moment. In the past year, the FDA has approved two CAR-T cell (chimeric antigen receptor T-cell) therapies, with stem cell treatments in hot pursuit as they enter late stage clinical trials. While regulatory body recognition is an important milestone for the field, great challenges in long-term sustainability of the industry remain. By proving that cell therapy can be effective, researchers have won an early battle – but to win the war they must ensure that this therapy is effective and accessible for the tens of thousands of recipients in the years to come. Delivering these therapies at this scale in a reliable and affordable manner will require a paradigm shift in not only manufacturing, but the subsequent supply chain and patient care that follow. Unlike proteins and small molecule drugs, cells are living entities derived from living donors. Once harvested from the patient, they continue to be influenced by environmental and temporal changes during the manufacturing process. When generating large batches of cells, this responsiveness leads to variability that can result in major batch-to-batch differences in product quality. Even with careful handling this variation can increase phenotypic variability and more importantly, hinder their therapeutic potency. The variability in large-scale manufacturing has been especially challenging in the expansion of mesenchymal stem cells (MSC’s). Some researchers believe that it is this variability which is responsible for the ongoing difficulties in achieving statistically significant efficacy in human trials. With the founding of institutions like Canada’s Centre for Commercial-

Biotechnology Focus

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stem cells

“For patient specific therapies like most current CAR-T’s then you’re trying to scale out the process and make it robust and repetitive. In MSCs you have a bulk product that can treat many thousands of patients with one donor – that’s’ a scale-up”. — Dr. Peter Zandstra

ization of Regenerative Medicine (CCRM), STEMCELL Technologies recently announced $138 million cell manufacturing facility in Burnaby, B.C, and biotech companies like Mesoblast, RoosterBio, and Excellthera, there is major work being done to address the manufacturing problem. The success of cellular scale-up is assessed through three major critical quality attributes (CQA): identity, purity, and potency. Identity and purity are relatively easy to assess in the lab, but reliably determining the potency of a dynamic, living therapeutic is still not quite where it needs to be. Dr. Peter Zandstra, Chief Scientific Officer for the CCRM and Chief Technical Officer of Excellthera explains: “Many of the markers to assess cell therapies right now are surrogate markers, meaning they don’t directly measure the therapeutic function of the cell but instead, correlate to it.” Zandstra emphasizes that this is especially a problem in clinical translation as at the laboratory stage, “we’re still one step away from biology that were looking for in the patient”. The potency problem can also vary widely not only from batch-to-batch, but also patient-to-patient, as often cell therapies are reprogramming the patient’s existing immune system to elicit a therapeutic effect. In predicting potency for different patient populations, Phil Vanek, General Manager Cell Therapy Strategy in GE Healthcare, suggests translating big data analytics into predictive models may ultimately be the answer: “We need the collection of large biological data sets that track the appropriate inputs and outputs of the therapy…we can then mine that data to better predict what the outcome will be with certain process and patient inputs”. In fortifying the cell therapy industry, there is also incentive to streamline the supply chain responsible for transporting the cells before and after expansion. These processes diverge according to the two major streams in cellular manufacturing: scale-out and scale-up. Zandstra explains: “For patient specific therapies like most current CAR-T’s then you’re trying to

scale out the process and make it robust and repetitive. In MSCs you have a bulk product that can treat many thousands of patients with one donor –that’s’ a scaleup”. While scaled up cells can be shipped in a similar manner to cell lines used in basic research, the supply chain is much more complex for scaled-out therapies as the cells must be transported — quite literally — from vein to vein in a timely manner. The supply chain for cell therapies involves health systems, hospitals, developers, manufacturers, as well as traditional logistics providers and distributors. Vanek notes, “one of the reasons why these cell therapies are so expensive is because the elements of the supply chain process are all disconnected today…from a supplier perspective, it’s very inefficient and inefficiency is driving up cost”. Companies like Gilead subsidiary, Kite Pharma are hoping to break down supply chain silos by controlling the entire vein-to-vein process. Through their Kite Connect service, all aspects of the supply chain will all be overseen by one company to help reduce redundancies and alleviate inefficiencies. In another approach, Asymptote — a subsidiary of GE Healthcare — is working to improve the ‘cold-chain’ technology which cryopreserves the cells for the entire transportation process in an endeavor to provide a little more flexibility in the supply chain timeline. In another effort to simplify the supply chain process, scientists are hoping to transform scale-out strategies into a more flexible scale-up process. Currently, scaled-out therapies like CAR-T cells rely on a single donor that ‘matches’ the immune profile of the recipient. To circumvent the matching process, most scaledout therapies are autologous, meaning the patient themselves serves as the donor. To shift toward a scaled-up version of CAR-T cells, a single donation must be able to make bulk quantities of cells for multiple patients. The donated cells however, must universally match the immune profiles of all recipients. This endeavor is being hotly pursued by the aptly named Universal Cells, a biotech company based in Seattle that uses gene editing to ‘scrub’ stem cells from immune recognition. Zandstra adds this could also improve issues related to variability in patient-specific cells: “You get cells from young and old patients, and individuals in different stages of the disease process which generates a lot of Biotechnology Focus

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stem cells

variability. If you can move to a more universal donor source of cells and then do your large batch scale-up, you can reduce a lot of that batch-to-batch variability”. Automation is another strategy that is hard to ignore when searching for ways to improve cellular manufacturing. Cleanrooms and laboratory technicians are still the workhorses of the field, but with yearly operational costs that often exceed $400,000 USD, this aspect of the manufacturing process is viewed by some as the most pressing obstacle in sustainable and economical cell therapy production. In response, Biospherix Ltd. has developed a ‘cytocentric approach’ which replaces cleanrooms and techni-

cians with a ‘hands-off’ automated system for cell culture. Vanek is quick to point out however, that there is still some work to be done before automation can be fully implemented: “Automation is what you do when you’ve locked down a process to reduce variability…[currently] you have patients entering a manufacturing process with different genetic, environmental and treatment backgrounds — simply stated: we need to understand a lot more about the biology to determine the predictability of a process. Once we get that then automation shouldn’t be a problem” Beyond the cell product, the delivery and aftercare of patients following treatment is also a major consideration in longevity.

The complex nature of this treatment has for the most part, limited cell therapies to areas with the facilities to assess and monitor cell therapy recipients. In addition to the obvious impact this has on accessibility, added cost to the healthcare system is also a significant obstacle. Dr. Michael Rudnicki, Director of the Stem Cell Network in Ottawa, Canada suggests that health economists may have a major role in properly assessing the issue for the long-term. “We need to evaluate the cost to our society of supporting someone with a disease, versus the cost of delivering healthcare”. Rudnicki adds: “The government looks at the silo of the healthcare system. What they don’t look at is that someone with a [life threatening] disease has an economic impact: being taken out of the workforce, not paying taxes, and [consequently] their kids may not get an education”. Freeing individuals from debilitating diseases and treatments goes far beyond saving a life; these advances have the potential for a major societal and economical impact that may in fact, overcome the steep costs upfront. Even so, there are high hopes that the costs of cell therapies will also improve with time. Vanek surmises “With the efficiency of supply chain, reproducibility, competition from multiple vendors — all those economic elements will come together [to decrease cost]”. Though complex, cell therapies have the potential to define a new pillar in modern medicine. Scalable solutions for the outstanding issues related to the manufacture, delivery and accessibility of cell therapies however, is paramount for a therapy that ‘sticks’. For those tasked with ensuring their continued success and mass adoption in modern healthcare, the work has just begun.

Erika Siren is a Ph.D Candidate in Biomaterials Chemistry at the University of British Columbia’s’ Centre for Blood Research. She is based out of Vancouver, British Columbia.

To see this story online visit https://biotechnologyfocus.ca/ hope-beyond-the-hype-pursuitlongevity-in-cell-therapies/

Biotechnology Focus / June/July 2018

Perspective By Gail Garland

ONTARIO

Use It or Lose It:

The Future of Canada’s Health Science Industry Strong health science clusters in the US, UK, Sweden and Germany have contributed enormous benefits to their economies and societies. From high-value knowledge economy jobs and domestic prosperity, to improved health outcomes, higher patient satisfaction via the practice of “smart healthcare”, and overall health system cost savings, innovative health technologies and a robust industry that produces them deliver transformative change and prosperity. A recent analysis of the US biotech landscape by Atlas Ventures concluded that Boston and San Francisco are the two clusters that dominate the health science industry. They combine a unique blend of biomedical science, venture capital, entrepreneurial talent, risk-taking culture, and geographic density. “Beyond having great science and the right ‘pixie dust’ in the local environment, two fundamentally important ingredients to the success of any cluster are capital and talent.” R&D industry jobs have grown by 30 per cent in the successful clusters over the last decade, while it has shrunk or flatlined in other regions, including Europe. Research on clusters indicates that their success relies on the interconnection between seven different sectors which include established companies, start-up businesses, universities or other research institutions, support groups or ‘champions’, state/provincial governments, municipal governments, and federal governments and their policies. A 2003 presentation to the Competitiveness Institute on behalf of the Ontario government included a benefit of the development of clusters is that they enhance productivity and therefore prosperity. The same presentation suggested that actions governments can take to support cluster development included early experimentation with new products, procurement of products, and enacting progressive laws and regulations. OBIO’s most recent report, ‘Use It or Lose It: Industry Solutions to Ontario’s Commercialization Gap’, is based on a recent survey of 135 health science industry executives in Ontario. The report found that while Ontario’s health science industry is beginning to mature, there remain significant barriers to scaling up. Ontario’s home-grown companies face obstacles that prevent them from maturing here, commercializing local intellectual property and growing into anchor companies. While both access to capital and access to talent continue to stand out as barriers to scaling, industry increasingly has concerns about the overall ecosystem and access to local markets. As one CEO expressed, “[For Ontario] to become a successful health science hub, a lot of work has to be done. Funding is the most pressing challenge, access to the healthcare system is a close second.” As discussed in OBIO’s ‘Use It or Lose It’, Ontario’s health science industry has five recommendations for policy makers to continue the growth and maturation of the sector. In addition to recommendations that allow companies to extend their runway and generate cash flow, this year industry emphasized the importance of go-to-market issues like access to local hospitals and health service providers, and the development of infrastructure to facilitate access to the local market for the purpose of clinical trials or pilot projects to generate evidence and refine technology.

Industry’s five recommendations: • Establish a health innovation capital fund that invests directly in health science companies. • Work with industry to establish a network of early adopter institutions and invest in infrastructure and programming to pilot new drugs and technologies at Ontario institutions. • Consolidate government funding programs and bring application and review processes into line with standard market terms and global investment practices. • Simplify procurement processes and facilitate adoption and dissemination of new drugs and technologies throughout the healthcare system. • Adopt globally competitive tax policies to attract investment and help companies to grow. Ontario’s health science industry has struggled to attract necessary capital, create value in local technologies and scale companies to create anchors for new start-ups. Consultations with industry over the years have consistently found access to capital as a major barrier to scale up. Access to talent has also been identified as a key barrier to commercializing and scaling up. Ontario’s industry is maturing and there are emerging success stories that highlight other aspects of the ecosystem that need to be addressed. Foremost among these is the need for a system of early adopter health care providers with expertise in piloting, adopting and championing new technologies. In order for Ontario’s health science industry to produce anchor companies, the local ecosystem needs to support early experimentation with innovative technologies. In a virtuous circle, anchor companies can generate the right talent which in turn attracts capital and investment. OBIO’s latest consultation with industry moves the field forward as the industry matures and offers recommendations to address challenges in scaling from start up to commercialization and growth that will benefit Ontario patients, healthcare providers and the economy. Use it or lose it. The opportunity is there. About OBIO® The Ontario Bioscience Innovation Organization (OBIO®) founded in 2009, is a not-for-profit, membership-based organization engaged in strategy, programming, policy development and advocacy to further the commercialization of Ontario’s human health technologies positioning Ontario as a leader in the international marketplace. OBIO advances this goal through collaborative partnerships with industry, the investment community, academia, the health system and government. For more information, please visit www.obio.ca and follow OBIO on Twitter @OBIOscience. Gail Garland is the founder and CEO of OBIO, a not-for-profit, membership-based organization championing the development of an integrated health innovation economy for Ontario.

Biotechnology Focus

June/July 2018

KalGene Pharmaceuticals By Anthony Boone

ONTARIO

KalGene Pharmaceuticals and its partners bring an innovative, collaborative approach to tackle Alzheimer’s disease For companies working to treat dementia the costs are high — but more than worth the toll paid by patients living with the disease.

s life expectancies around the globe continue to go up, the accompanying steady rise in senior populations has given way to a newly intensified global focus on dementia in recent years. Dr. Margaret Chan, director-general of the World Health Organization, underscored the urgency of current and future needs in her 2015 address to the first WHO Ministerial Conference on Global Action Against Dementia, warning of the “tidal wave of dementia coming our way”. Adding, “I can think of no other disease where innovation, including breakthrough discoveries, is so badly needed.” A year earlier, public research agencies in the UK had pledged more than £200 million to the cause and last November Bill Gates personally invested $50 million in the Dementia Discovery Fund expressing on his blog, “Of all the disorders that plague us later in life, one stands out as a particularly big threat to society: Alzheimer’s.” A big part of that threat is the associated costs of caring for afflicted patients. Until we discover a way to prevent the disease, the health system costs to care for and support patients bear the brunt of the impact. In 2017, the US spent $259 billion on hospital, hospice and long-term care for dementia patients. This is expected to increase to $1 trillion by 2050. An estimated 46 million people worldwide suffer from dementia. According to the Alzheimer’s Society of Canada some

Dr. T. Nathan Yoganathan, KalGene president, and Dr. Danica Stanimirvic, director of the NRC, Translational Bioscience Department, on the signing of the NRC KalGene collaborative Alzheimer’s disease partnership.

564,000 Canadians suffered from dementia in 2016 and by 2031 that number will nearly double to 937,000. The associated costs to the health care system for treatment, care and support is estimated at $10.4 billion and will grow to $16.6 billion by 2031. Clearly the need to improve treatment and to pursue the eventual prevention of Alzheimer’s disease is immense—both from an economic and social perspective. Industry response has been to step up

research efforts to accelerate the development process. In the US, National Institute of Health funding has increased in the past five years from $503 million per year to $1.391 billion per year. While Canada’s public research agencies are working to increase current research funding levels of just under $50 million, the private sector is stepping up to bridge the gaps and support promising new technologies with the potential to change the way we treat the disease.

An estimated 46 million people worldwide suffer from dementia. According to the Alzheimer’s Society of Canada some 564,000 Canadians suffered from dementia in 2016 and by 2031 that number will nearly double to 937,000. 16

Biotechnology Focus

June/July 2018

KalGene Pharmaceuticals

ONTARIO

KalGene Pharmaceuticals and NRC team.

One such company is KalGene Pharmaceuticals, a privately held company working in partnership with leading clinician-scientists and academic institutions. The company has taken the roadless-travelled approach to its clinical program, looking to slow down disease progression by developing a treatment that can penetrate the blood-brain barrier. This breakthrough puts KalGene on the road to success according to president and chief scientific officer Dr. T. Nathan Yoganathan. “A major obstacle to treating neurodegenerative diseases like Alzheimer’s is delivering therapeutics past the blood-brain barrier,” says Dr. Yoganathan. “Using technology licensed from the National Research Council of Canada, we’re overcoming that obstacle. As a result, our development candidate is able to pass through the blood-brain barrier, rapidly inducing CSFamyloid-beta clearance. Studies to date have indicated dramatic plaque removal.” KalGene has worked closely with the McGill center for Ageing over the past 2 years to characterise the therapeutic molecule in a battery of test resembling the proposed clinical study. They have received contributions from Weston Brain Foundation, Ontario Brain Institute, Brain Canada and CQDM for this study. KalGene’s preclinical success has not gone unnoticed. Late last year, the firm received significant financial support from a syndicated investment led by Lumira Capital. Also participating in the transaction were Anges Québec, Anges Québec Capital, Accel-Rx Health Sciences Accelerator and a number of Canadian family offices. “We know so much more now about this terrible disease than we did just a few

years back,” says Jacki Jenuth, partner at Lumira Capital. “Kalgene will benefit tremendously from these new insights especially as it relates to patient recruitment with their partners at McGill.” For François Gilbert, CEO of Anges Québec and Anges Québec Capital, KalGene’s novel approach was key to their decision. “We were impressed by the quality of the science behind KalGene and their approach to Alzheimer’s treatments,” he says. “We know that important challenges lie ahead in this race to tackle Alzheimer’s disease, but we are confident that the KalGene team has what it takes to succeed.” Dr. John Gillard, KalGene’s highly experienced VP of Product Development, shares that enthusiasm largely because of KalGene’s ability to attract and work with key partners, including the National Research Council of Canada and McGill University, where a number of the lead clinicians and researchers are located. “The scientists there deserve a lot of credit for what we’ve accomplished,” he says. “No doubt, their contributions have allowed us to move forward.” Accel-Rx sees the work that KalGene is doing as exemplary of the potential of Canada’s health sciences environment to nurture and grow a vibrant sector. “When our ecosystem aligns—scientific expertise, industry-led strategy, private capital—it provides a catalyst for companies like KalGene to drive hard to deliver on tremendous promise,” says Natalie Dakers, president & CEO, Accel-Rx. “When promising therapies are commercialized, ultimately it’s the patient who wins.” “We are extremely confident with our approach—and that we are carrying out our work in the right place,” says Dr. Yoganathan. “The national ecosystem

in which we conduct our research, with its government support, with the participation of talented scientists from other institutions, with the financial support of private and public foundations, is an ecosystem ideal for the breakthroughs we need to confront and eventually overwhelm this terrible disease.” The company expects to conduct the first clinical trial of its Alzheimer’s therapeutic candidate at McGill University in 2019. Safety studies are expected to last about three months and the efficacy studies a little less than a year.

Public-Private Partnerships Prove Promising As researchers continue to grapple with the Alzheimer’s puzzle, countries around the world are increasingly looking towards public-private partnerships as the most effective strategy to stave off a health tsunami that will surpass cancer and heart disease as the population ages. Recalling the model to battle polio in the mid-20th century, nations are combining the funding strength of the public sector, research breakthroughs of academia and the developmental/commercial acumen of the biopharmaceutical industry to accelerate the path to treatment and care.

Anthony Boone is a writer and communications professional with experience in the health sciences and clean tech sectors.

To see this story online visit https://biotechnologyfocus.ca/ kalgene-pharmaceuticals-andits-partners-bring-an-innovativecollaborative-approach-to-tacklealzheimers-disease/ Biotechnology Focus / June/July 2018

Interview

Q&A with Molly Shoichet Compiled by Michelle Currie

Ontario’s first Chief Scientist

ONTARIO

This past year, Ontario created a brand-new position to provide a platform to support the growth of its innovation-based economy and promote Ontario as an integral research hub and a top destination for global talent. Molly Shoichet was appointed and is now the first ever Chief Scientist of Ontario. Molly is an award-winning researcher and will be the glue that converges the government, the scientific community and public, by championing high quality science and promoting Ontario to the world’s best researchers.

Biotechnology Focus had the opportunity to speak with her recently about her new position and what we can expect to see come from it.

“As a society, investment in research is crucial, as it leads to invention, innovation, and commercialization. We need to continue investing but we also have to invest more.”

Biotechnology Focus

June/July 2018

Interview

ONTARIO Q: What was the vision that created this position?

I think that the chief innovation officer in Israel was a big inspiration for this position as he is doing great things around innovation and the economy, rooted in science. That was the spark that led to a greater discussion and to a survey of what is going on in the rest of the world regarding chief scientists. By seeing what was going on in the UK, Australia, New Zealand, US, even in Canada – because we have had a chief scientist in Quebec for the last six years – helped shape what would be great for Ontario.

Q: Was this position based off

templates from around the world?

You always want to see what’s going on globally, look at best practices and make it right for Ontario. I think we realise that, in Israel, there’s more of a chief innovation officer rather than a chief scientist, and that wasn’t quite what the need was in Ontario. Really this role is an advisory role, similar to the federal Chief Science Advisor, rather than an operational one, such as the Chief Scientist position in Quebec, which includes a significant operational component. Of course, it’s always a good idea to see what everyone else is doing, learn from that and then ask, ‘what do we think is right for Ontario?’

Q: What would you like to achieve at this year’s BIO conference in Boston? We are passionate about Ontario’s Culture of Science and we’d like to share that message while we’re here at BIO. We start in a position of strength, with a fantastic ecosystem that begins with superb education and great science centres, like the Ontario Science Centre and Science North. We have internationally renowned universities, with 7 of the top 15 universities in Canada being in Ontario and the University of Toronto being ranked among the top 20 universities internationally for the past decade. We have strength in life sciences, artificial intelligence and advanced manufacturing. We have a dynamic private sector that gets stronger

every year. With increased opportunities for investment, our start-ups are scaling up. We will build on these strengths to enhance Ontario’s culture of science and the knowledge-based economy.

Q: What attracted you to the role of Chief Scientist?

Well, I think this is always a good question because I have an amazing job being a professor, discovery is so exciting, and we always think about the opportunity to invent the future. So, I am really happy that I have been able to hold onto that, and coming into this role it was really important for the chief scientist to be a scientist. But what was really exciting to me about this role, was taking everything I have learned about bringing teams together and communicating that to society. To do that for the province of Ontario was that opportunity to do something bigger and also the opportunity to give back to the community in a different way. What really excited me was the challenge. I have to tell you, I’ve only been here since January, so since then I have been on this enormous learning curve and I’ve gone out and met with as many people as I can. I have a much better understanding of the role of the government. Everyone has been very welcoming and they share that excitement that I have to work together in trying to do something better and in doing something differently. We would like to have an open-door policy and see ourselves as being the champion for research and a champion for bringing the community together around the knowledge-based economy.

Q: I see you have a background in regenerative medicine. What interested you in this form of research? What would you say your area of expertise would be? I think when any of us look back on our career, any of us can connect the dots, but at the time they’re just dots. For me growing up I was always interested in medicine, and when I got to university I had a better understanding of what research is, and even when I graduated with

“But what was really exciting to me about this role, was taking everything I have learned about bringing teams together and communicating that to society. To do that for the province of Ontario was that opportunity to do something bigger and also the opportunity to give back to the community in a different way.” my undergraduate degree I had applied to med school and grad school. Actually, I deferred med school and went right to grad school and just got really excited about the idea of “inventing the future” – future of medicine, future therapeutics. My first job out of university was at a biotech company and I think I was just lucky. That company was doing cell therapy – this was before we called it regenerative medicine. My Ph.D. was actually in polymer chemistry, so it was in more of the engineering and materials side of things, but the company needed the materials to protect the cells from the immune system. I think I was just very lucky to have joined a biotech company in the greater Boston area. It was a field that was emerging and I found myself in the field of regenerative medicine and was smart enough to stay in it. When I came to Toronto, there were already a couple of people in engineering working in this field – of course, there’s a huge richness in stem cell biology in Toronto since the 1960’s. This has just been a great area, and we’re working in areas where there are no, or few, therapeutics available – we’re trying to overcome stroke, spinal cord injury, and blindness. With regenerative medicine, there’s that hope of not just treating the symptoms of disease, but stopping them

Biotechnology Focus

June/July 2018

Interview

ONTARIO chief scientist’s office is a small team, but with enormous goals, that we can only achieve if we all come together. People aren’t going to come together unless they’re inspired by a common purpose. That’s why I say it’s not about me, or even the team here, it’s about bringing the community together.

Q: Could you tell me a bit about Ontario’s Life Sciences Economy?

Molly Shoichet and team. Photo: the Government of Ontario

“We would like to have an open-door policy and see ourselves as being the champion for research and a champion for bringing the community together around the knowledge-based economy.” and reversing them. Giving someone back their vision or their cognitive abilities, in itself is very inspiring. Our lab is highly collaborative, highly multidisciplinary. We’re working on strategies to enhance cell survival and enhance integration. So that’s our area, really that connectivity. All of our inventions go back to my core expertise, which is designing materials for applications in medicine.

Q: What do you envision happening

in the life sciences community as we move forward?

I think we have great expertise in the life sciences – over the last 15 years more than 50 per cent of Ontario’s research investments have been in life sciences – so we can recognise that as our strength and our opportunity. We see great expertise in genomics and synthetic biology. Great expertise in regenerative medicine and cell

therapy. There are overlaps obviously, and great expertise in artificial intelligence. In that, Ontario just has a strong history in cell therapy, artificial intelligence, and big data. So, we’re building from a strength, and we’re building from decades of investment in research to now being able to start connecting those dots in research and the knowledge-based economy. As most of us know, it is not a straight line. As a society, investment in research is crucial, as it leads to invention, innovation, and commercialization. We need to continue investing but we also have to invest more. I think of it as a spiral, reaching for bigger, better, smarter, and more efficient strategies and knowledge. So, it’s that idea that we’re really excited about – convergence.

Q: What are your aspirations for Ontario/Canada?

It’s not about me, but about all of us coming together. Even our team here in the

Ontario is an economic powerhouse in Canada. It invests the most in life sciences out of all the provinces and his home to 51 per cent of Canada’s life sciences R&D. Ontario’s economy in the life sciences sector in North America is also remarkably strong. Ontario places 7th in terms of employment and 8th by the number of firms in North America, so we need to wave our Ontario flag proudly. It’s important for global business leaders to know that Ontario is the 7th largest economy in North America. To sustain the levels of innovation in the life sciences sector, you need a strong ecosystem made up of a number of different partners, including hospitals, research centres, technology incubators, universities and much more. The key for successfully growing Ontario’s life sciences ecosystem can be linked to the incredible amounts of collaboration that has grown organically between all the different partners. This unique and enhanced level of collaboration has positioned Ontario as a global centre of research excellence in cancer and stem cell research; diagnostic imaging; gene therapy and neuroscience. With over 1900 firms, 60,000 employees and 18,000 researchers and staff making up Ontario’s life science sector, the one defining asset that has made this sector a world leader is the strength and diversity of our science based communities and networks. Science and research provides the sector with a level of adaptability that ensures their strategic investments continuously innovate and commercialize those successes.

To see this story online visit https://biotechnologyfocus. ca/q&a-with-molly-shoichetontarios-first-chief-scientist/

Biotechnology Focus / June/July 2018

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PERSPECTIVE By David O’Neill, PhD

ONTARIO

The Oncology Entrepreneur’s Secret Weapon: Navigating Commercialization with FACIT

For many entrepreneurs and universities, commercialization is a serial process that follows an invention disclosure to the Institution. While this traditional model can work in many established fields of engineering and IT, moving cancer research from bench to bedside is a much more challenging and complex commercial path. Fortunately, oncology entrepreneurs in Ontario have a secret weapon: FACIT. As a strategic partner to the Ontario Institute for Cancer Research (OICR), FACIT integrates early commercial planning alongside the research strategy. Well prior to inventions, FACIT collaborates with entrepreneur-scientists to plot a course for industrial development, adoption and reimbursement. FACIT addresses the local gap in seed capital investment and on occasion provides interim management of spin-outs to help companies grow strong roots in Ontario. These are critical resources needed to help make the commercialization journey successful and ultimately achieve patient impact. FACIT’s model has proved effective time and again over the past decade.

Helping companies break out — and therapies break through

Dr. Scott Tanner was one of the earliest recipients of FACIT support. He recalls it was critical to enabling his company, DVS Sciences, secure Series A financing and commercialize its measurement technology. “We went through three transitions getting off the ground, and each time we ran out of money,” Tanner recalls. “That’s when many companies fail. We didn’t — because we had a dedicated team and because FACIT took the long view. They saw the opportunity in us that was still five years down the road and supported us so we could raise the money to realize our potential.” As DVS Sciences expanded, the company brought manu-

facturing infrastructure and jobs to Ontario, which remain in the province. FACIT redeployed the returns generated on its initial investment to support a next wave of Ontario innovations. Among those today is a series of molecules that inhibit the WDR5 protein linked to various cancers. OICR Drug Discovery Program Director Dr. Rima Al-awar was part of the team that developed the novel class of WDR5 inhibitors, licensed in 2016 to Propellon Therapeutics — a FACIT spin-out company. FACIT seed-financed Propellon and provides corporate and operational support to help commercialize the WDR5 innovation. “FACIT became involved at the optimization stage of the chemical series,” says Al-awar. “They were extremely engaged, and have placed Propellon in an excellent position to attract potential investors and partners to ultimately bring this asset to market and positively impact the lives of cancer patients.”

Ready to receive

FACIT is also building the commercialization pathway to include locally based receptors. Triphase Accelerator Corporation is a leading example of this. A clinical accelerator created by FACIT in partnership with MaRS Discovery District and MaRS Innovation, Triphase advances promising cancer therapies into — and through — clinical development. In 2016, a novel brain cancer treatment developed by Triphase was sold to the global pharmaceutical firm, Celgene Corporation. That acquisition brought substantial returns to Ontario and has positioned Triphase strongly as a receptor for other next generation breakthroughs.

Building entrepreneurial capacity in Ontario

To date, the unique FACIT/OICR model has spawned 14 startups and generated significant economic benefits for Ontario. Tanner says an important feature of that model is its holistic, entrepreneurial focus. “When we talk about commercialization, we tend to focus on markets, venture capital and Since its inception, FACIT has actively helped bridge so on. But before any of that, you need the oncology commercialization gap in Ontario. people: staff, researchers, coordinators, advisers. FACIT understands this. They help assemble talented, capable teams that can build viable companies.” While less of a secret these days, this weapon continues to serve entrepreneurs, patients living with cancer, as well as the Ontario economy. David O’Neill is the President (Acting) of FACIT

Learn more about the unique FACIT/OICR model and its benefits for Ontario at facit.ca.

Biotechnology Focus

June/July 2018

microbiome By Michelle Currie

ONTARIO

Going deep into the human microbiome with

Adapsyn Bioscience In the modern world, artificial intelligence (AI) and deep learning have helped to accelerate important advancements in technology across a wide-range of industries, with the biotechnology sector being a prime example of such transformative applications. In Hamilton, Ontario, one biotech company is harnessing the power of deep learning and AI to accelerate the discovery of new pharmaceuticals from microorganisms found in nature and even the human body.

dapsyn Bioscience, a spinout company from McMaster University, mines the landscape of highly-evolved small molecules from microorganisms to uncover a diverse portfolio of novel drugs to treat a wide spectrum of diseases. Adapsyn was incorporated in 2016, and soon after its inception drew the attention of pharmaceutical giant Pfizer, entering into a collaboration to accelerate their drug discovery efforts. The company now has 12 full-time employees, in addition to several others who are on a part-time or contract basis. “I’m one of those Canadians that was in the U.S. for a long time, and then decided to come back to Canada,” says Adapsyn founder, president & CSO, Nathan Magarvey. “I came back with aspirations of setting up a company to engage in research here rather than the U.S. Adapsyn was a growing body of work that was established from my research lab, and the lab was increasingly taking on industrial work. It started getting a bit too much for an academic lab, so that’s when I said it’s probably time to spin out and make it grow. Building a start-up is an experience unto itself, and something I always wanted to do.” Subsequent to the research deal formalized with Pfizer, the company raised venture capital from Genesys Capital in Toronto, a mainstay in the Canadian biotech sector, and Pfizer Ventures out of the U.S. The company strengthened its board of directors by adding representatives from these firms and additional industry experts. “The microbiome is increasingly a focus for the company,” says Magarvey. “We’ve been engaged in the microbes of the planet and sifting through those genomes to identify molecules. What Adapsyn

Biotechnology Focus / June/July 2018

microbiome

ONTARIO

“With our technology, we never find the compound we found before, and given that around 1 in 200 molecules become clinical candidates, we can really ramp up programs to push those novel entities into therapeutic models.” — Nathan Magarvey does better than anyone is translating genes to proteins to small molecules. Those evolved small molecules are highly valuable against a whole spectrum of human therapeutic drug targets and can be particularly useful as anti-infective and immunomodulatory agents. Adapsyn is able to translate the gene sequence directly to the small molecule structure, and can in turn determine which structures are new and which ones are old, all from the genome sequences that we have. We can then predict from that data which ones do new things and which ones do things we have seen before from known compounds.” This is really at the heart of the machine learning and AI that the company uses to inform its wet lab activities – namely, purifying and testing novel molecular entities for therapeutic potential. The company has created a data set of all-natural products discovered to date, and uses this to relate predicted novel compounds to past discoveries, thereby allowing the company to infer potential uses of newly discovered natural products. “For a long time, we would focus on microbes and plant sources. But the challenge with that, by modern day standards you are a prisoner of the past. All the molecules that were identified previously, if you use old methodologies, you end up discovering the same molecules that you

previously found. With our technology, we never find the compound we found before, and given that around 1 in 200 molecules become clinical candidates, we can really ramp up programs to push those novel entities into therapeutic models,” says Magarvey. Adapsyn’s technology allows them to survey genomes of microorganisms, numbering over a hundred thousand to date, and rapidly pinpoint novel chemistries. With this pace of discovery, the company has seen a flourish of interest from many pharmaceutical companies. While some companies have enormous depositories of biological material from which to screen, they are inadequately-equipped to rapidly assess their chemical novelty. Adapsyn has proven they possess the technological platforms necessary to unlock this potential. “This is high science. This is a merger of chemistry, microbiology, genomics, and computer science,” says Magarvey. “Canada does well in certain elements of each field. The Canadian research community is also recognized for its creativity in the connection disparate research fields. Adapsyn is an exemplary showcase of this creativity and scientific translation. Striving to address the unmet medical need is where we can make a transformative impact on Canada and globally.” “It’s fascinating for us to appreciate

Adapsyn’s technology allows them to survey genomes of microorganisms, numbering over a hundred thousand to date, and rapidly pinpoint novel chemistries. With this pace of discovery, the company has seen a flourish of interest from many pharmaceutical companies. 24

Biotechnology Focus

June/July 2018

now all the medicines that are left in nature that are awaiting discovery to address more complex diseases,” comments Magarvey. “We don’t appreciate this in society, but a lot of our medicines – not just your holistic things – but medicines that we take are actually derived from living creatures on the planet. With our new technology, we can sort through all that information so rapidly, that now we see all these new opportunities.” Drug discovery can be a long, complex and expensive process, but with Adapsyn’s technology, it could take less time from bench to the bedside. “We’re getting better and better at discerning the molecular targets of those predicted molecules by using deep learning,” Magarvey says. “That’s really important, because obviously you have to be able to quickly identify what the molecular targets would be of molecules and their value as therapeutic entities. So, we’re using cellbased, high-content screening, in combination with our predictions, and deep learning is having a big role in elucidating the mechanisms of action of these novel compounds.” Adapsyn Bioscience is altering the drug discovery landscape, combining genomic and metabolomic data with AI and machine learning. Their unique approach will help fuel the future of natural products research, ultimately leading to the development of new pharmaceuticals that will impact global healthcare and address the treatment of diseases that currently lack effective therapies.

To see this story online visit https://biotechnologyfocus.ca/ going-deep-into-the-humanmicrobiome-with-adapsynbioscience/

PERSPECTIVE By: Karen Ramlall

ONTARIO

Small biotech start-ups drive innovation Small start-ups, not big pharmaceutical organizations, are now at the heart of innovation in health-related biotech. In fact, 62% of drugs approved in 2017 in the U.S. originated in smaller companies, according to HBM Partners, a healthcare investing firm.1 This doesn’t surprise the new CEO of the Centre for the Commercialization of Antibodies and Biologics (CCAB). Robert Verhagen, who joined the organization in November 2017, has seen dramatic changes over his biotech career. “Some of the most innovative ideas are coming from small companies. They are increasingly willing to take risks and raise money to pursue ideas toward the development of new therapies.” With more than 25 years of leadership experience in the pharmaceutical and diagnostics fields, Verhagen has seen these changes up close and, in his new position, he sees an opportunity to broaden CCAB’s focus and evolve it into an organization that assists with the development of novel therapies through the support and creation of start-up companies. Under his direction, CCAB will also extend its technical offerings to help these emerging companies and the research community as a whole as it launches C-Lab – which offers custom products, services & solutions and operates within CCAB – and begins distribution of reagent antibodies from its portfolio.

C-Lab – helping our partners develop better antibodies & proteins

C-Lab offers tailored antibody and protein production services, characterization, and solutions. The C-Lab team has extensive experience in producing high-quality, fully human antibodies and other proteins for both academic and industry partners. It offers a range of services, which include: • Antibody and Protein Production • Custom Solutions for Optimized Protein Purification • Antibody Engineering • Antibody Characterization • Plasmid Services To date, the C-Lab team has purified more than 2,000 antibodies and other proteins for research and drug development purposes. C-Lab combines recombinant synthetic technology and a transient transfection platform, which offers both reproducibility and scalability. As a result, C-Lab can customize its process to suit a client’s needs, from ug to g amounts of protein. One of C-Lab’s specialties is its versatile collection of antibody format species and isotypes – a portfolio the team is constantly expanding to meet scientific and customer needs. C-Lab works with a wide range of academic and industry clients and is looking to expand its services to a wider range of biotech start-ups. Currently, C-Lab is working with Pionyr Immunotherapeutics, which is developing cancer immunotherapies to target the tumour microenvironment. Michel Streuli, Pionyr’s Senior VP, says of the relationship: “The agility and the can-do attitude of the C-Lab group, paired with their scientific expertise, has enhanced Pionyr’s ability to develop our therapeutic programs. We look forward to continuing to work with the team as they launch C-Lab to the broader scientific community.”

Antibody Reagents: CCAB offers a robust portfolio of validated research-grade antibodies, specializing in isoform specificity within protein receptor families.

Antibody and Protein Production: C-Lab pairs its unique mammalian expression vector with its Expi293 cell line, licensed from Life Technologies, to offer a variety of scales and high-throughput options and achieving titres up to >400 mg/L.

High-quality reagents make excellent research tools

The launch of C-Lab coincides with the start of distribution of reagent antibodies from CCAB’s portfolio of monoclonal researchgrade antibodies against an array of human targets involved in various diseases such as cancer and autoimmune disorders. Through CCAB’s selection process, antibodies that exhibit competitive binding properties in flow cytometry and ELISA assays are tested further and then optimized for reagent use. For distribution of the reagents, CCAB has established a partnership with MédiMabs Antibodies. The Montreal-based company, experienced in marketing antibodies produced by hybridoma methods, was looking for opportunities to expand its portfolio to include synthetic antibodies and identified CCAB as an organization that could help them provide more comprehensive sets of protein family profilers. “MédiMabs is looking forward to working with CCAB since our organizations have great synergy and compatible technologies,” Martin Wong, MédiMabs Chairman says. “We are confident that this partnership will be a win-win for both companies and together we can deliver new and unique antibodies to life sciences researchers”. Establishing collaborations with other Canadian small and medium-sized enterprises, like MédiMabs, lines up with the goals of CCAB as it looks to expand its presence in the ecosystem. With these new initiatives, and likely more to come, it is an exciting time for CCAB and the small companies it is looking to foster.

Reference:

1. Dr. Ulrich Geilinger. Trends in US New Drug Approvals. HBM Partners, January 2018.

Karen Ramlall is the Communications Manager at CCAB. info@ccabcanada.com www.ccabcanada.com https://twitter.com/CCABcan https://ca.linkedin.com/company/ccabcan Biotechnology Focus / June/July 2018

PERSPECTIVE

By Krista Lamb

ONTARIO

Regenerative Medicine Experts in Ontario Aim to Strike the Right Balance by Stimulating Commercialization and Reducing Healthcare Costs

Stem cell researchers in Ontario, supported by the Ontario Institute for Regenerative Medicine, are making strides towards treatments that could have a big impact on the province’s economy.

Biotechnology Focus / June/July 2018

In 2000, when Dr. Duncan Stewart, president and scientific director for the Ontario Institute for Regenerative Medicine (OIRM), co-founded his small biotech company, Northern Therapeutics, he knew the potential for regenerative medicine was enormous. Now, after many years of hard work he has started to see that potential realized. Northern Therapeutics, in partnership with a U.S. biotechnology company, recently launched a multi-centre phase II clinical trial across Canada, with four sites in Ontario, utilizing a cell and gene therapy to treat pulmonary hypertension. In addition, OIRM is working with both academic and funding partners in California to extend this trial into the United States, further increasing its impact. “These types of investments show the value international investors place in Ontario’s ability to create breakthrough technologies in the regenerative space,” says Dr. Stewart.

ONTARIO “All of the cells for this trial will be manufactured in Ontario at The Ottawa Hospital, where there is the needed expertise for manufacturing the large number of cells required for these trials and the logistical experience essential to transporting cells across the country. This is an opportunity for the biotech industry in Ontario to showcase the world-leading work being done in this province.” Commercialization, which has an ongoing positive impact on the Ontario economy, is a focus for OIRM. “Ontario is a leader in the area of regenerative medicine,” says Sandra Donaldson, vice president and COO of OIRM. “Part of OIRM’s mandate is to develop partnerships worldwide that will help to expand the impact of Ontario-generated technologies in this market.” In addition to working with Dr. Stewart to support his commercialization efforts, OIRM was one of the first to fund the groundbreaking work of UHN’s Dr. Michael Laflamme, whose team is using stem cells to repair damaged heart tissue. Laflamme recently partnered with BlueRock Therapeutics—a $225 million investment by Versant Ventures and Bayer—to commercialize this treatment. “OIRM launched in 2014,” says Donaldson, “We’re pleased to already be seeing so much success. It’s clear that one small investment in these technologies can be a catalyst for

oirm-BiotechAd-May2018.pdf

2018-04-24

expanded partnerships and growth. We have the expertise to launch international multi-centre trials which encourages adoption of successful technologies and commercializing them in global markets.” OIRM is also focused on ways in which treatments developed using stem cells can reduce healthcare costs in the province. Projects like that of Dr. Freda Miller from The Hospital for Sick Children in Toronto offer another example. Dr. Miller and her team are researching the use of the generic drug metformin for endogenous white matter repair of the brain in youth. Because this treatment takes place early in the lifespan and uses a commonly available drug, it has the potential to have a substantial long-term impact in the health of those with Multiple Sclerosis, at a greatly-reduced cost in comparison to current treatments. “When OIRM awarded $3.6 million in grants in 2017 with the support of the Ontario Ministry of Research, Innovation and Science, we considered many factors,” says Donaldson. “We wanted to know that each project had the potential to lead to positive outcomes for patients. We also looked at whether these treatments, if successful, could help reduce the economic burden on our healthcare system or lead to commercialization opportunities. We are pleased to see so many of them showing promise so early in the process.”

11:15 AM

CELL THERAPY CLINICAL TRIALS IN ONTARIO 2018–2019

CMY

BRAIN AND/OR NERVES

HEART

Phase I using a diabetes drug for brain repair in children with Multiple Sclerosis: Toronto

Phase II using genetically modified stem cells following a heart attack: Ottawa and Toronto

Phase II using stem cells for Multiple Sclerosis: Ottawa

Phase II using stem cells for advanced heart failure with left ventricular assist device: Toronto

Phase II using a diabetes drug for brain repair in children with Cerebral Palsy: Toronto Phase III using a diabetes drug to stimulate brain repair for malignant brain tumours: Toronto

GENETIC DISORDERS Phase I using genetically modified stem cells for Fabry Disease: Toronto

LIVER Phase II-III using stem cells to induce tolerance in recipients of liver transplants: Ottawa and Toronto

13 trials – 11 active 2018, 2 pending 2019 13 partners 4 Ontario cities 200+ patients Billions of stem cells

JOINTS Phase I-II using stem cells for knee osteoarthritis: Toronto Phase III using a cartilage tissue implant grown from the patient’s own cells: London

LUNG Phase I using stem cells for bronchopulmonary dysplasia in pre-term infants. Ottawa (Pending 2019) Phase II using genetically modified stem cells for pulmonary arterial hypertension: London, Ottawa, Toronto and Hamilton

CRITICAL CARE Phase II using stem cells for septic shock: Hamilton, Ottawa, Toronto (Pending 2019) All trials lead by OIRM investigators

@oirmnews

All trials sponsored/funded by some or all of the following: UHN, Ottawa Hospital Research Institute, SickKids, Dr. Gary Levy, Dr. Jas Chahal, Histogenics, CIHR, Stem Cell Network, Ozmosis Research Inc., Dr. Annetine Gelijns, National Heart, Lung and Blood Institute, Northern Therapeutics and OIRM

Biotechnology Focus

June/July 2018

PERSPECTIVE By Ainslie Parsons and Carmela De Luca

ONTARIO

The Battle to Control CRISPR IP Continues The CRISPR/Cas9 system allows specific “editing” of genes in living cells and organisms using in part a cell’s own machinery. This system has already revolutionized genomic engineering and is expected to have profound implications for disease treatment, agriculture and beyond. Not surprisingly, the powerful potential applications of CRISPR have led to a significant amount of interest in protecting the intellectual property associated with this technology. Currently, a patent battle is ongoing in the United States between the University of California (UC), Berkeley and the Broad Institute in Cambridge, Massachusetts. Briefly, in May 2012, a group of researchers associated with UC Berkeley and Umea University in Sweden filed a patent application in the United States Patent and Trademark Office (USPTO) describing a CRISPR/Cas9 system that could cut DNA in vitro. In December 2012, a second group from the Broad Institute also filed a patent application in the USPTO. Unlike the UC application, which only included data showing the use of CRISPR/Cas9 in prokaryotic cells, the Broad application showed that the CRISPR/Cas9 system could be used in eukaryotic cells to modify DNA in vivo. While the Broad application was filed after the UC application, it issued to patent first in April 2014 with claims covering methods of editing genes in eukaryotic cells using CRISPR/ Cas9. In early 2016, UC Berkeley requested that a patent interference be initiated (these proceedings are now obsolete under the current “first to file” system in the United States), claiming that the UC team invented the CRIPSR/Cas9 system and that the disclosure from the Broad Institute that the system worked in eukaryotic cells was merely an obvious extension of the UC team’s work. In a February 2017 decision, the United States Patent Trial and Appeal Board rejected the University of California’s argument, meaning that the Broad patent remains valid, and the

Biotechnology Focus / June/July 2018

UC application can proceed. The decision is currently being appealed before the United States Court of Appeals for the Federal Circuit but even if the decision is upheld, it is unknown what patent claims will ultimately issue from the UC family of applications. More recently, the battle has expanded beyond the United States as a number of CRISPR patents have been granted in Europe. In March of this year, the European Patent Office revoked one of the Broad Institute’s key CRISPR patents for an invalid priority claim. As a result, the issued claims were found to lack novelty over a number of intervening disclosures. The Broad Institute is appealing the decision. It is far from clear who will ultimately prevail in the CRISPR/ Cas9 dispute, and it is possible that victory will be shared. While the current licensing landscape for CRISPR/Cas9 is complex, licensing opportunities do exist. Indeed, the value of CRISPR, coupled with the number of CRISPR-related patents and applications, have led some to suggest that “patent pools”, agreements between two or more patent owners to license their IP together, may be well suited to this technology. In addition, improvements and alternatives to CRIPSR/ Cas9 are in development. There is no doubt that CRISPR will continue to break new ground, both scientifically and on the intellectual property front.

Ainslie Parsons, Associate at Bereskin & Parr LLP

Carmela De Luca, Partner at Bereskin & Parr LLP

By Michelle Currie

Ontario steps up

to play on the global stage

It has been a year of good fortune for the Ontario life sciences sector. There have been many investments made and new capital that will fuel innovation within the province and economy.

ntario moved forward with their plan for a venture capital fund to help companies access the capital they need to grow their business, create jobs, and grow on a global level. They invested $50 million specifically for the growth of the Ontario life sciences sector. The Ontario Capital Growth Corporation (OCGC) plans to identify fund managers to partner with other institutional investors such as corporations, banks and pension funds. The new life sciences venture capital fund is to respond to the challenges faced in raising capital by innovative, highpotential life sciences companies to scale up and reach global markets. “Providing much needed capital to growing life sciences companies is a crucial step towards a strong and sustainable life sciences ecosystem,” comments the Honourable Reza Moridi, Minister of Research and Innovation. “Supporting high-potential life sciences companies will create good jobs and help commercialize technologies and services for improved healthcare at home and around the world.” The fund will also help businesses foster new discoveries, including new technologies, treatments and cures for illnesses while supporting high quality, knowledgebased jobs for people across the province. It will also drive Ontario’s ability to attract and retain talent. “Capital financing, including venture capital, is essential for many innovative companies,” says Steve Romanyshyn, OCGC president and CEO. “OCGC is pleased to play a role helping Ontario-based companies scale-up and succeed at home and in global markets.”

The OCGC, venture capital agency of the government of Ontario, was created to promote and develop the venture capital sector in Ontario so that more high-potential technology companies have access to the capital needed to grow and prosper. Supporting innovation in the life sciences is part of Ontario’s plan to create fairness and opportunity during this period of rapid economic change. As well as the government of Ontario’s contribution a $100 million partnership has come to life between MaRS Innovation and Korea Health Industry Development Institute (KHIDI) to establish and foster international research and commercialization opportunities for Ontario. MaRS Innovation hosted a delegation for KDIHI back in 2016 to present some of Ontario’s most promising portfolio companies and technologies. Since then, that visit has led to further dialogues discussing how both countries could innovate and co-commercialize. MaRS Innovation has met senior Korean government officials at both the municipal and national level to enhance and explore new initiatives and forge ahead with the mission to accelerate scientific collaborations with the highest commercial potential. Collaborating researchers that KHIDI identified with projects of high commercial potential and MaRS Innovation have ventured on the third mission to Seoul to participate in the 2018 BIO Korea International Convention – one of the largest life sciences and healthcare conference in Asia – to focus on research and investment opportunities. The mission is a continuation of the concerted efforts by Premier Wynne and Minister Moridi

during their recent visits to South Korea in 2016 and 2017, and potentially could create a new Global Venture Fund between MaRS Innovation and South Korea to scale-up and commercialize Ontario life sciences innovations and companies. “This current, and third, mission to Seoul builds on the partnership between MI and KHIDI which started in 2016,” says Rafi Hofstein, president and CEO of MaRS Innovation. “With us from Toronto on this trip we have a wonderful group of high ranking scientists and researchers who, together with their distinguished Korean counterparts, have brought several very promising scientific technologies to the point where they can now be pitched to Korean investor groups. I am also happy to note that, outside the scope of the MI-KHIDI arrangement, South Korean fund managers and other investors are taking note of the entire MI portfolio. This level of interest in a relatively short time speaks to the quality of science and innovation coming out of the research labs of our Member institutions in Ontario, and MI is very pleased to be meeting with potential investors on their behalf. “ Ontario is the largest life sciences jurisdiction in Canada with more than 50 per cent of overall Canadian revenue. Therefore, it should come as no surprise that within Canada we are seeing more investment to develop novel technologies and companies within the province, and more interest and capital from international sources. Ontario is stepping up its game and is ready to play as a contender on the global stage.

To see this story online visit https://biotechnologyfocus.ca/ ontario-steps-up-to-play-on-theglobal-stage/ Biotechnology Focus

June/July 2018

| By Dr. Jason Field

LAST word

Seizing the Day: How we can capitalize on Ontario’s life sciences opportunity

t the opening of Toronto Health Innovation Week this spring, MaRS Discovery District CEO Yung Wu had this to say about our health and life sciences ecosystem: “We have been through crawling and walking, now we are starting to run and sprint.” Indeed, 2018 represents a crossroads for our sector. A surge of momentum for our companies, along with a provincial election, has created an unprecedented opportunity to capitalize on Ontario’s life sciences economy. You don’t need to look far to see evidence that our sector is soaring: Canadian companies – two based in Ontario – were among the Top 20 Life Sciences Companies to Watch in 2018. PwC’s MoneyTree report headlined health tech as a sector primed for growth in 2018. In March, the Government of Ontario announced a $50 million dedicated life sciences venture fund; and global pharmaceutical giant Sanofi recently invested $500 million toward a Canadian vaccine facility to be based in North York, Ontario. On the ground, we’re pioneering innovations that are the stuff of science fiction: an Ottawa company, eSight, has developed eyewear that allows the legally blind to see. Toronto-based technology from Synaptive Medical enables doctors to perform complex brain and spinal surgeries that were previously thought impossible. The roster of Canadian companies at JP Morgan in January was nothing short of stellar. Clearly, we are becoming trailblazers; our challenge is to seize this momentum and run with it. And, in fact, our future economic success may depend on it. As a sector that contributes $38.5 billion to the province’s GDP and ranks among the top clusters in North America, the economic growth potential for Ontario life sciences is hefty. In the US, three biotech companies – Amgen, Gilead, and Celgene – have a combined market capitalization of US $258B, exceeding the entire Canadian mining sector listed on the TSX (over 1,200 compa-

Biotechnology Focus

June/July 2018

nies with a market cap of US $230B). Plainly put, an Ontario biotech success story has the potential to revolutionize our economy. Life sciences is also crucial to driving sustainability in our health care system, developing technologies to control climate change, and providing knowledge-based jobs for our highly-educated workforce (more than 55 per cent of Ontarians have a college or university degree, 25 per cent higher than the OECD average). So, how do we capture this massive opportunity? We need a plan. We need a coordinated life sciences strategy. This is where a newly-elected provincial government can step in to play a crucial role in securing our economic future. While the federal government is developing its own approach through its Health and Biosciences Economic Strategy Table, Ontario is still among the only major life sciences jurisdictions in North America without such a strategy in place. It’s fitting that BIO takes place in Boston this year. Provincial politicians of all stripes should take note of the ten-year, $1BUSD Massachusetts life sciences strategy launched back in 2007 and its enormous impact on this world-leading biotech cluster. The state has committed to renewing this funding for another five years. With the importance of such a strategy in mind, Life Sciences Ontario released its Blueprint document in December 2017. It lays out a comprehensive plan and recommendations to accelerate the success of life sciences, with specific action around access to capital, talent growth, support for innovation, and promotion of our sector. It is endorsed by leading provincial and national life sciences, health, and economic organizations. All that is needed now is for the next Ontario government to run with it. The provincial election will be decided on June 7th – the final day of BIO 2018. Regardless of the outcome, life sciences is non-partisan by its very nature and must remain a priority. It touches on almost every facet of our success as a province and nation: the food we eat, the health of our population and environment, our research and innovation capacity, and the jobs and wealth that fuel our economic growth. These issues transcend politics. We are starting to run and sprint in our Ontario life sciences sector, but this is only one leg of a long race. To make the leap to our full potential, we must all work together toward a coordinated strategy. This is the springboard that will propel our outstanding innovations into the global marketplace and create a healthier and wealthier future for all Canadians. Dr. Jason Field is President and CEO of Life Sciences Ontario, a member-funded, not-for-profit organization advancing Ontario life sciences through advocacy and policy work, educational and networking events, and support services for the sector. To see this story online visit https://biotechnologyfocus.ca/ seizing-the-day-how-we-can-capitalize-on-ontarios-life-sciencesopportunity/ Got something to say? Please send your comments/letters to biotechnology_focus@promotive.net

Biotechnology Focus delivers critical Canadian news and insights, trends and reports, from R&D to business.

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