Biotechnology Focus June/July 2017 by Promotive Communications

INSIGHTS FOR THE LIFE SCIENCE INDUSTRY

JUNE/JULY 2017 VOLUME 20, NUMBER 2

revolution Canadian biotech firms in the forefront of the artificial intelligence disruption

INSIDE : Life sciences and gamification Versant Venture and BlueRock Shire’s big move The Vector Institute

Publication Mail Registration Number: 40052410

BDH® HIPERSOLV CHROMANORM SOLVENTS FOR HPLC The Range Keeps GROWING & GROWING! VWR is proud to announce the continued expansion of our HPLC solvent range, plus the addition of high purity modifiers to meet the exacting requirements of all HPLC users. Manufactured to the most stringent requirements both in Europe and the USA, these solvents and modifiers provide outstanding value for the money. These solvents: • Meet demanding HPLC and UV/Vis analytical and quality control requirements • Are filtered down to 0.2μm level and bottled under nitrogen • Feature tamper-evident caps for added peace of mind • Carry Globally Harmonized System (GHS) labeling to help with compliance • Feature QR codes on labels for quick access to SDS and C of A • Feature environmentally-friendly corrugated/pulp case inserts that are fully recyclable Visit vwr.com/bdh for complete specifications, Certificates of Analysis, Safety Data Sheets, and current pricing.

contents

June/July 2017 – VOLUME 20 – NUMBER 2

FEATURES 9

A unique neurotech commercialization model proves successful

The works of the Ontario Brain Institute BY RICKY CHAN

Artificial intelligence – the Canadian connection AI is the future and these biotech firms are poised to seize it

BY NESTOR ARELLANO

CCAB – Making Toronto the epicenter for commercializing biologics and antibodies The antibodies market space is a highly competitive arena but the Centre for the Commercialization of Antibodies and Biologics has a secret weapon

COMPILED BY NESTOR ARELLANO

Anchor companies and ‘sticky capital’

BY LISA WILLEMSE

A prescription for attracting bioscience investment to Ontario

Q&A with Brian Bolzon of Versant Ventures on the risks and rewards of launching biotech startups

They’ve got game How gamification is being used in brain health research

BY NESTOR ARELLANO

BY CHRISTINE MISQUITTA

Identifying the last line of defence for innovative Canadian drugs Patents and data protection BY NOEL COURAGE AND PHIL GODBACH

BY NESTOR ARELLANO

Building a new company is not just about finding great IP

CCAB: Translating early-stage biologics into your products

Shire shines as latest addition to Toronto biotech cluster Toronto’s bustling health and biotech community attracts company which recently acquired Baxalta in a US$32-M merger

BY GAIL GARLAND

Ontario’s regenerative medicine pipeline Ontario stem cell researchers, led by the Ontario Institute for Regenerative Medicine are making gains

COMPILED BY SHAWN LAWRENCE

In Conversation with Ontario’s Minister of Research and Innovation Reza Moridi Research and Innovation Ministers Reza Moridi weighs in on AI and the Vector Institute.

BY NESTOR ARELLANO

What is BlueRock working on? A snapshot of some of the research being done at the new $225-million biotech firm created by Bayer and Versant Ventures.

It’s a good time to invest in oncology and innovation

Ontario Regional Jurisdictions BY THE BIOTECHNOLOGY FOCUS STAFF

Ontario life sciences on the rise The province’s life sciences sector is turning heads BY JASON FIELD

The Last Word We have the scent that some serious international clustering dynamics is in the air BY DR. RAPHAEL HOSTEIN

The Fight Against Cancer Innovation Trust and getting innovation to market BY JEFF COURTNEY

www.biotechnologyfocus.ca

June/July 2017 BIOTECHNOLOGY FOCUS 3

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

Terri Pavelic Nestor Arellano Shawn Lawrence Dr. Raphael Hofstein

Lisa Willemse Gail Garland Christine MIsquitta Jason Field Ricky Chan Jeff Courtney

You say you want a revolution My apologies to the Beatles but it seems a revolution is what we’re in the cusp of right now – an AI revolution. Beyond the doom and gloom prognostications about how machines are going to take over the world, there are those brave souls that see the bright future of artificial intelligence. Remember when the Internet first broke out into the public scene? Yup, it seems very hard to imagine now how we can ever do without it. This is our annual Ontario edition produced to coincide with the 2017 Bio International Convention. Our coverage once again highlights the Ontario life sciences industry and how Ontario continues to stay on top of leading science and business practices in building a robust platform for life science organizations to build and grow upon. In this edition’s In Conversation, Ontario’s Science and Innovation Minister Reza Moridi ventures to say that AI is poised to make an impact on our daily lives that is just as far reaching and as elemental as the Internet. Could Ontario be this country’s ground zero for unfolding AI disruption? Flip over to pages 30 and 31 to find out some of the exciting works scientists from the province’s various research facilities are up to. In the article Artificial Intelligence – the Canadian connection, we look at three biotech companies engaged in groundbreaking work using AI. In it, Liam Kaufmanof Winterlight Labs, Abraham Heifets of Atomwise, and Brendan Frey of Deep Genomics flesh out some of the research coming out of their labs. Also featured in this month’s edition are Shire Pharma Canada, and the Centre for Commercialization of Antibodies and Biologics. Brian Bolzon of Versant Ventures talks about his company’s winning strategy, and we find out what BlueRock is up to. In our article, They’ve got game, panelists for the 2017 Bio International Convention’s discussion on Game Theory and the Brain, talk about how gamification is helping advance brain health research. I think we’ve come up with a stellar lineup for this edition. We hope you do too.

Noel Courage Phil Goldbach GRAPHIC DESIGNER CONTROLLER MARKETING MANAGER

Elena Pankova John R. Jones Mary Malofy

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

EDITORIAL ADVISORY BOARD Christine Beyaert, Roche Canada; Pierre Bourassa, IRAP, Montréal; Murray McLaughlin, Sustainable Chemistry Alliance; Ulli Krull, UTM; John Kelly, KeliRo Company Inc.; Peter Pekos, Dalton Pharma Services; Robert Foldes, Viteava Pharmaceuticals Inc.; Gail Garland, OBIO; Barry Gee, CDRD; Bonnie Kuehl, Scientific Insights Consulting Group Inc.; Raphael Hofstein, MaRS Innovation; Roberto Bellini, Bellus Health; Peter van der Velden, Lumira Capital; Albert Friesen, Medicure Inc.; Ali Tehrani, Zymeworks Inc.; Dr. Jason Field, Life Sciences Ontario; Andrew Casey, BIOTECanada

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.

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

4 BIOTECHNOLOGY FOCUS June/July 2017

R&D news Researchers warned of CRISPR off-target effects

There’s a CRISPR genome editing boom of sorts going on with as many as 20 human trials being undertaken in several countries. However, researchers may be ignoring some serious drawback of the genome editing which made it easy and affordable for laboratories to target nearly any sequence. The capability to precisely modify the gene of any organism is a promising prospect for treating and curing diseases such as cancer, leukemia, and HIV/AIDS. However, there is

Dementia-related brain changes seen before memory problems occur Researchers at the Baycrest Rotman Research Institute (RRI) and the University of Toronto said their study indicates that there are observable changes in the brain’s structure which could predict the onset of dementia even before patients show noticeable signs of the disease such as memory loss. The team, which studied 40 adults between the ages of 59 and 81, found evidence of less brain tissue the anterolateral entorhinal cortex located in the brain’s temporal lobe. This is the sub-region of the brain where Alzheimer’s disease originates from. Early detection of alterations of the brain structure has the potential of identifying seemingly but at-risk individuals and can also foster the development of drugs and therapeutic interventions. The Alzheimer Society of Canada reports that there are more than 560,000 Canadians living with dementia. More than 1.1 Canadians are affected by the disease. As many as 25,000 new cases of dementia are diagnosed each year in Canada. The total cost of caring for people with dementia in the country is estimated at $10.4 billion. 6 BIOTECHNOLOGY FOCUS June/July 2017

growing evidence that CRISPR could alter regions of the genome which researchers are not targeting, according to Dr. J. Keith Joung of Massachusetts General Hospital, a report from StatNews.com said. At a recent American Society of Hematology’s workshop on genome-editing, Joung showed some 150 experts from industry and academia an example where CRISPR is supposed to edit the VEGFA gene on chromosome 6. Vega stimulates the production of blood vessels, including those used by cancerous tumors. Joung said studies have shown that the CRISPR can hit genes on every one of the other 22 human chromosomes. “Although each CRISPR has zero to a dozen or so ‘known’ off-target sites (where known means predicted by those web-based algorithms), Joung said, there can be as many as 150 ‘novel’ off-target sites, meaning scientists had no idea those errors were possible,” the StatNews.com report said. The issue of off-target effects is important there is the possibility that genome

editing could inadvertently disable a tumour-suppressor gene or activate a cancer-causing gene. Consider the possibility of and off-target effect where two different chromosomes are joined in a phenomenon called translocation. Translocation is the cause of chronic myeloid leukemia and other conditions. Off-target effects occur because CRISPR has two parts. The RNA part targets the site in the genome specified by the RNA’s string of nucleotides. The enzyme cuts the genome at this site. However, a genome can have more than one site where the same string of nucleotides appears. The cutting enzyme of CRISPR does not stop at one cut. The enzyme “still has the energy to bind with an off-target site, so it can still cleave those sites,” said Joung.

The joint study, published in the Neurobiology of Aging journal on May 8, looked at older adults who are living in the Toronto community without assistance and who were unaware of any major memory problems, but who scored below the normal benchmark on a dementia screening test. It is also the first study to demonstrate that performance on the Montreal Cognitive Assessment (MoCA) dementia screening test is linked to the volume (size) of this sub-region, along with other brain regions affected early in the course of Alzheimer’s disease. “This work is an important first step in determining a procedure to identify older adults living independently at home with-

out memory complaints who are at risk for dementia,” says Morgan Barense, associate professor in the department of psychology in the Faculty of Arts & Science at U of T and senior author on the study. Scientists were able to reliably measure the volume of the anterolateral entorhinal cortex by using high-resolution brain scans that were collected for each participant. The strongest volume differences were found in the exact regions of the brain in which Alzheimer’s disease originates. The researchers are planning a follow-up study to determine whether people who demonstrated poor thinking and memory abilities and smaller brain volumes indeed go on to develop dementia. “The early detection of these at-risk individuals has the potential to facilitate drug developments or other therapeutic interventions for Alzheimer’s disease,” said Rosanna Olsen, first author on the study, RRI scientist and assistant professor in U of T’s department of psychology. “This research also adds to our basic understanding of aging and the early mechanisms of Alzheimer’s disease,” she said.

To see this story online visit http://biotechnologyfocus.ca/dementia-related-brain-changes-seen-beforememory-problems-occur/

To see this story online visit http://biotechnologyfocus.ca/researchers-warned-of-crispr-off-target-effects/

R&D news MilliporeSigma proposes alternative CRISPR genome editing method

Chemical and life sciences firm MilliporeSigma has developed a new method for editing CRISPR genome which the company said provides researchers with more experimental options and rapid results that can

help in speeding up drug development. The new technique, called proxy-CRISPR, is able to access previously unreachable areas of the genome, according to the company. “With more flexible and easy-to-use genome editing technologies, there is greater potential in research, bioprocessing, and novel treatment modalities,” said Udit Batra, CEO, MilliporeSigma. “… MilliporeSigma’s new technology is just one example of our commitment to solving challenges in the genome editing field, and we will continue to make CRISPR research a priority.” CRISPR genome editing technology is advancing treatment options for some of the toughest medical conditions faced today, including chronic illnesses and cancers for which there are limited or no treatment options, according to MilliporeSigma. The applications of CRISPR are far ranging from identifying genes associated with cancer to reversing mutations that cause blindness. CRISPR enables genome editing using an enzyme called Cas9 to cut DNA, but this has limited targeting abilities.

This limitation led to MilliporeSigma’s focus on proxy-CRISPR. Most natural CRISPR systems, found in bacteria, cannot work in human cells without significant re-engineering. However, proxy-CRISPR provides a rapid and simple method to increase their usability without the laborious need to re-engineer native CRISPR proteins. MilliporeSigma has filed several patent applications on the proxy-CRISPR technology. These patent applications directed to the proxy-CRISPR technology are just some of several CRISPR patent application filings made by the company since 2012. MilliporeSigma’s research on proxy-CRISPR, “Targeted Activation of Diverse CRISPR-Cas Systems for Mammalian Genome Editing via Proximal CRISPR Targeting,” was published in the April 7, 2017, edition of Nature Communications. To see this story online visit http://biotechnologyfocus.ca/ milliporesigma-proposes-alternativecrispr-genome-editing-method/

June/July 2017 BIOTECHNOLOGY FOCUS 7

BUSINESS corner Threshold Pharmaceuticals’ TH-3424 now OBI-3424 after acquisition by OBI Pharma

Biopharmaceutical company OBI Pharma Inc. has signed an agreement with Threshold Pharmaceuticals to acquire the San Francisco-based company’s TH-3424 small-molecule protein drug. TH-3424 targets cancers overexpressing the enzyme aldo-ketoreductase 1c3 (AKR1C3). Obi Pharma is changing the name of the drug to OBI-3424. The drug is a first-in-class prodrug that selectively releases a potent DNA alkylating agent in the presence of the AKR1C3 enzyme. This selective mode of activation distinguishes OBI-3424 from traditional al-

kylating agents, such as cyclophosphamide and ifosfamide, which are non-selective. “We will continue the pre-clinical work and hope that OBI-3424 develops into a solid treatment option for patients with cancers that express AKR1C3,” said Amy Huang, general manager of OBI Pharma. “This novel cancer therapeutic enhances our pipeline and moves us another step towards becoming a global cancer biopharma company.” AKR1C3 overexpression has been documented in a number of treatment-resistant and difficult to treat cancers. For example, hepatocellular carcinomas (HCC), which highly overexpress AKR1C3 in the majority of patients.

OBI-3424 has demonstrated potent activities in preclinical models of HCC, including a model resistant to the standard of care treatment, sorafenib. “AKR1C3 is highly overexpressed in a number of cancers that represent unmet medical needs, including hepatocellular carcinoma, castrate-resistant prostate cancer, and T-cell acute lymphoblastic leukemia. OBI-3424 offers the possibility of early efficacy readouts based on objective response rates in well-defined resistant patient populations,” said Dr. Tillman Pearce, Threshold’s chief medical officer. AKR1C3 is adaptively upregulated in response to castration; therefore, castrateresistant prostate cancer is another logical unmet need population where OBI-3424 will be tested. In addition, the United States National Cancer Institute is performing preclinical evaluations of OBI-3424 for the potential treatment of T-cell acute lymphoblastic leukemia (T-ALL). To see this story online visit http://biotechnologyfocus.ca/ threshold-pharmaceuticals-th3424-now-obi-3424-after-acquisition-by-obi-pharma/

BioVectra opening new site in Nova Scotia BioVectra Inc., a contract manufacturer for pharmaceutical and biotech companies, is investing $30 million in the construction of a new microbial fermentation facility in Windsor, Nova Scotia in order to substantially bump up its capacity. The PEI-based service provider said the planned 50,000 square-foot facility will be operational by December this year. The late-stage to commercial-scale microbial fermentation and complex chemistry facility is strategically located to be near the potential clients in the northeastern part of the United States. BioVectra’s $30 million investment has equipped the facility with 40,000 liters of fermentation capacity, downstream processing equipment and new pre-clinical fermentation and potent chemistry suites. “The investment strengthens our unique position in the very competitive global 8 BIOTECHNOLOGY FOCUS June/July 2017

marketplace, enabling us to offer new and existing clients range of scale throughout the entire lifecycle of a product,” said Oliver Technow, BioVectra’s president. BioVectra provides scalable cGMP operations from 30 L to 17,000 L. The company has the capability to handle the development and cGMP scale-up of high-potency active pharmaceutical ingredients (APIs), handling potent substances such as antibody-drug conjugate warheads, with occupational

exposure limit (OEL) levels of < 20 ng/m3 (Safebridge Band 3 and 4). The new facility increases BioVectra’s ability to partner with global pharmaceutical companies by offering dual-site risk mitigation, according to Heather Delage, the company’s vice-president of business development. To see this story online visit http://biotechnologyfocus.ca/biovectraopening-new-site-in-nova-scotia/

SPECIAL REPORT By Ricky Chan

ONTARIO

A unique neurotech commercialization model proves successful The Ontario Brain Institute (OBI) boasts a healthy portfolio of 56 companies that we support as part of our strategic mandate to bring neurotech to market and establish Ontario as a world leader in brain research, commercialization, and care. We do this through the ONtrepreneurs (Ontario Neurotech Entrepreneur) and NERD (Neurotech Early Research & Development) programs, which uniquely focus on brain-related startups. Our integrated commercialization strategy is connected to a network of researchers, clinicians, and patient advocacy groups, in addition to funders and investors. This reflects OBI’s overall strategy in building convergent partnerships to ensure that technologies are validated and can improve people’s quality of life. One example is GaitTronics Inc. from Ottawa, Ontario. Through support from both ONtrepreneurs and NERD programs, they tested their robotic assistive device in children with cerebral palsy through our CP-NET research network. The neurological charity, ThreeToBe, has since purchased a unit and placed it in the Abilities Centre for people of all abilities to enjoy. Since 2012, 34 ONtrepreneurs have benefited from the $50,000 award including mentorship, business training and they are a part of OBI’s integrated network. NERD funds product

development to help bridge the funding gap to private capital by de-risking investments. The companies in these programs combined are averaging an eight-fold return on OBI’s investment. In a recent impact survey of our portfolio companies, 94 per cent of respondents indicated that we helped to improve their ability to access capital and 100 per cent of respondents experienced improved knowledge or access to business, research and healthcare information. Such successes are gaining attention and attracting companies to move to Ontario to join the rapidly growing neurotech cluster ecosystem, NeuroTech Ontario. During BIO 2017 in San Diego, OBI will announce ten new ONtrepreneurs, introducing a new wave of entrepreneurial talent and innovative neurotech. Ricky Chan is a member of the Industry Relations team at the Ontario Brain Institute and leads the NeuroTech Ontario Cluster initiative.

Ontario as a world leader in brain research, commercialization and care

RESEARCH

DATA SHARING

COMMERCIALIZATION

CARE

Access to an extensive research network with 240 researchers and 35+ institutions

Large-scale informatics platform for big data on a variety of brain disorders

Non-dilutive funding, mentorship and training opportunities

Access to patient representatives for input into technology development

WE ARE FUELING NEUROTECH COMMERCIALIZATION

Technology Development | Building Companies | Attracting Investments | Developing Talent

PORTFOLIO COMPANIES

RETURN ON

INVESTMENTS

For more information contact us at IndustryRelations@braininstitute.ca

@OntarioBrain

www.braininstitute.ca June/July 2017 BIOTECHNOLOGY FOCUS 9

Artificial intelligence By Nestor Arellano

ONTARIO

Artificial intelligence – the Canadian connection AI is the future and these biotech firms are poised to seize it

Biotechnology Focus

June/July 2017

The writing is on the wall, in papers (peer-reviewed no doubt), and all over the Internet. Artificial Intelligence is transforming our world – right before our very eyes, in live, augmented reality colour

Artificial intelligence

ONTARIO Canadians are not mere bystanders in what is being called the AI revolution. Many of our country’s scientists are leading the insurrectionists. It could be argued that Ontario is ground zero for the unfolding AI upheaval. A lot of deep learning research come out of Canada. Many of today’s world leaders in AI research can also trace their roots to Ontario universities like the University of Toronto. Three of the biotech firms engaged in some of the most exciting AI researchers have robust Canadian connections.

Winterlight Labs Can the way a person speaks be accurately used to detect dementia? Researchers at Toronto’s Winterlight Labs believe so. The company is developing a proprietary AI diagnostic platform that can objectively assess and monitor cognitive health. “Using a one-minute speech sample, our platform can analyze natural speech,” said Liam Kaufman. “It can detect and monitor dementia, aphasia and other cognitive conditions.” Kaufman holds a master’s degree in medical science, a bachelor’s degree in psychology and another in computer science – all from the U of T. He is the CEO of Winterlight, a startup he co-founded in 2015.

The Winterlight platform analyzes hundreds of linguistic cues, according to Kaufman. Peer-reviewed studies proved the platform can detect dementia of the Alzheimer type and other conditions with accuracies between 82 per cent and 100 per cent. The assessment can be administered with the use of a tablet device with which subjects are asked to view images on the screen, and speak out his or her description of the picture. The device records the subject’s voice, and sends the audio file over the cloud to Winterlight Lab’s analysis platform. The platform immediately uses 400 variables to quantify the speech, language, and cognition and then provides a mini mental state examination (MMSE) score. Dementia affects more than 47.5 million people around the world, according to World Health Organization numbers. Often, the disease is not detected until its debilitating signs begin to surface. Research has shown that certain changes in speech can signal the early onset of dementia. In an interview with U of T faculty of medicine writer Carolyn Morris, Frank Rudzicz, a scientist with the Toronto Rehabilitation Institute – an expert in acoustic processing, and co-founder Winterlight, said the technology recognizes cognitive impairment through observa-

The Winterlight platform can analyze natural speech to detect and monitor dementia, aphasia and other cognitive conditions. — Liam Kaufman

Biotechnology Focus

June/July 2017

Artificial intelligence

ONTARIO

For a drug to work, it has to stick to a target protein. Researchers need to find a molecule that would work with this protein. Today, to do this test is physically labourius. It’s slow, expensive and difficult. —Dr. Abaraham Heifets

tions of diction and syntax. One of the signs of cognitive impairment is the use of simple verbs rather than gerunds. For example, said Rudzicz, a person with cognitive impairment might say “the kid runs” instead of “there’s a kid running” or “a kid on stool” but fail to make the connection that “the son is trying to steal cookies.” Apart from the Winterlight team’s experience in cognitive neurology and speech technology, the platform is being powered by artificial intelligence. “One of the areas where AI and computers are good is in solving the needle in the haystack problem,” said Kaufman. “Deep learning is a type of AI that has been particularly good in detecting and teasing out patterns and relationships, whether it’s in the pitch of a voice or image from an X-ray or CT scan.” The platform, he said, is a huge improvement from the time-consuming, costly and subjective method of assessment which uses pencil-and-paper test. The Winterlight team is conducting a series of pilot studies with seniors in Toronto retirement homes. They hope to develop the platform into a diagnostic tool that can be used in doctors’ offices, telemedicine, and it pharma campuses.

Atomwise Researchers typically test tens of thousands of compounds as part of the process of developing new drugs. It is a task which

Biotechnology Focus

June/July 2017

could involve tests on upwards of 1,000 people. It could take up to 15 years and cost more than $1 billion. “For a drug to work, it has to stick to a target protein. Researchers need to find a molecule that would work with this protein,” said Dr. Abaraham Heifets. “Today, to do this test is physically labourius. It’s slow, expensive and difficult.” But simulation using AI can significantly cut down the time and cost, said Heifets, a former IBM software engineer and holder of a PhD in computer sciences from U of T. He is also co-founder and CEO of Atomwise Inc., a San Francisco-based tech startup that created AtomNet. AtomNet is a deep learning technology for novel small molecule discovery. It takes the legwork out of the testing process. Using deep learning, the technology predicts how molecules will react and likely they are to bind with proteins. The software can also teach itself to recognize patterns in molecular interaction. So far, the software has been used to help create new drugs for Ebola and multiple sclerosis. Whereas the physical trial-and-error method used by scientist to analyze tens of thousands of compounds could take up to 18 months, Heifets said, the Atomwise technology is “able to deliver compounds in as little as four to 12 weeks.” Atomwise can screen a million compounds in one day but “the time factor is dominated by FedEx,” he lamented.

ARTIFICIAL INTELLIGENCE

ONTARIO

Deep Genomics is using machine learning and genomic science to develop genetic medicines — Brendan Frey

DEEP GENOMICS Another company seeking to streamline what it calls the “guessand-test” approach to the molecule and protein matchmaking process is Deep Genomics. Like many tech and biotech startups in the city, it operates out of the MaRS Centre in downtown Toronto. Deep Genomics is using machine learning and genomic science to develop genetic medicines and speed up treatments that address the root causes of diseases and disorders, from cancer to autism to cystic fibrosis, according to Brendan Frey. Frey is a U of T engineering professor, as well as, co-founder and CEO of Deep Genomics. He is also co-founder of the recently launched Vector Institute. With over $200 million in funding, the Vector Institute aims to become a global hub for AI research and development. “There are many ways a protein could be causing a problem, resulting from different changes to the genome. We can see those changes at the level of individual genes,” Frey said in a recent interview with the U of T Engineering News. “Instead of focusing on proteins, we’re focusing on the genetic mutations that are the source of the problem.” Deep Genomics aims to harness the massive amount of genetic data gathered from the time gene sequencing began in 2011. The startup hopes to help pharma companies cut down their drug development. Deep Genomics’ technology was used to find ways to tackle rare Mendelian disorders, a class of genetic conditions caused by mutations in a single gene. More than 350 million people worldwide are affected by Mendelian disorders. Deep Genomics will explore disorders of the central nervous system, eye, and liver, said Frey. The company has been using machine learning to study mutations in the genome. It’s now time for Deep Genomics to use its platform to help pharmaceutical companies create genetic medicines, he said.

To see this story online visit http://biotechnologyfocus.ca/artificial-intelligencethe-canadian-connection/

www.kalgene.com “KalGene is determined to develop therapeutics to treat Alzheimer’s.” - Dr. T. Nathan Yoganathan, CEO, KalGene Pharmaceuticals Inc.

KalGene Pharmaceuticals is developing a novel therapeutic molecule to treat Alzheimer’s disease. KalGene is collabo rating with NRC, McGill, the Montreal Neurological Institute and CIMTEC to advance the therapeutic development. KalGene plans to initiate Phase 1 clinical trials in 2018.

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

Biotechnology Focus

June/July 2017

CCAB Compiled by Shawn Lawrence

ONTARIO

CCAB

MAKING TORONTO THE EPICENTER FOR COMMERCIALIZING BIOLOGICS AND ANTIBODIES

Terrence Donnelly Centre CCAB TRAC

iologics are the fastest growing biopharmaceutical segment due to their promising potential to treat a variety of diseases and the Centre for the Commercialization of Antibodies and Biologics is well positioned to help build Toronto into a cluster for antibody and biologic development. Since its inception in 2015, CCAB, a federally-funded Centre of Excellence for Commercialization and Research (CECR) based in Toronto, has already partnered with over 100 biotech and biopharmaceutical companies worldwide including the US, Europe, China, and India to develop therapeutic antibodies towards the clinic. Dr. Ivan Waissbluth, director of business development at CCAB, believes that their specialty and in-depth knowledge of the antibody and biologic market enable them to be a partner of choice.

Members of the CCAB team

“We direct all our business development resources toward understanding and keeping up with the evolving market demand for biologics and antibodies,” said Waissbluth. “By understanding what biotechnology companies, our customers, need as well as having an attractive technology offering, we aim to be a top-tier destination for antibody development and commercialization.”

ACCESS TO A GROWING PORTFOLIO OF ANTIBODIES AND BIOLOGICS CCAB offers its partners access to a growing library of antibodies and a state-ofthe-art discovery platform, both of which are powered by the Toronto Recombinant Antibody Centre (TRAC). TRAC is Canada’s leading centre for producing high-quality therapeutic grade antibodies. It was established in 2010

by Dr. Sachdev Sidhu, a world-renowned professor in protein engineering at the University of Toronto. Over the last several years, Sidhu has partnered with Dr. Jason Moffat, a molecular geneticist at the U of T. Together they have further expanded the research areas of TRAC by collaborating with leading scientists and clinicians around the world. “Other (commercialization) models – they have scouts working the halls (of university labs) trying to find where the next technology is coming from,” said Waissbluth. “We don’t spend any resources scouting for technology. We don’t need to because we have a direct connection to the discovery and development pipeline of TRAC - we’re talking about thousands of antibodies.” The CCAB team is entirely dedicated to advancing and licensing the antibodies from TRAC, said Waisbluth.

We don’t spend any resources scouting for technology. We don’t need to because we have a direct connection to the discovery and development pipeline of TRAC. 14

Biotechnology Focus

June/July 2017

CCAB

ONTARIO CCAB lab

They’re basically asking us if we have the antibody they need and if we don’t, can we make it.

Dr Jason Moffat

Dr. James Pan

Dr. Sachev Sidhu

TRAC has been very successful in attracting grant and sponsored research funding. Most recently, TRAC has partnered with the University of California San Francisco and the University of Chicago in a collaborative research and commercialization deal with Celgene, securing more than $40 million in funding for the discovery and development of new cancer therapeutics, $13 million of which will be spent on development in Toronto.

Licensing and co-developing antibodies and creating new companies Access to partners who can provide business expertise, financing, new pipeline assets and development capabilities including manufacturing and preclinical evaluation, are common problems among new biotech companies in Canada. CCAB aims to support the Canadian biotechnology ecosystem by providing their in-house business and scientific capabilities in biologic and antibody development and facilitate commercial transactions by offering flexible partnering/licensing options. CCAB commercializes antibodies in three main ways to cater to partners’ interests: • Out-licensing to established biopharmaceutical companies • Co-development/ risk-sharing partner-

ships, primarily with early-stage biotechnology companies • Forming startups in conjunction with venture capital groups Companies approach CCAB to evaluate and develop antibodies from CCAB/ TRAC’s established library or partner with the organization to develop custom solutions. “Our clients either come here to see the most advanced antibodies or they come here with a very specific target in mind,” Waissbluth said. “They’re basically asking us if we have the antibody they need and if we don’t, can we make it.” Working with industry partners, CCAB has evaluated and developed over 300 antibodies against 60 targets and has entered 13 licensing option agreements in the last 2-½ years. CAB has also supported the development of five startup companies and the list continues to grow. “Our vision is to make Toronto the epicenter for commercializing biologics and antibodies,” said Waissbluth.

To see this story online visit http://biotechnologyfocus.ca/ ccab-making-toronto-theepicenter-for-commercializingbiologics-and-antibodies/

Ivan Waissbluth Biotechnology Focus / June/July 2017

Special report By Gail Garland, CEO, Ontario Bioscience Innovation Organization

ONTARIO

Anchor companies and ‘sticky capital’ A prescription for attracting bioscience investment to Ontario Despite the presence of many emerging leading edge, health science companies in Ontario, we continue to struggle to anchor and grow these companies here at home. What’s holding them back? Why can’t Ontario become the home of worldleading health science technology companies? Those were the driving questions behind our effort to bring health science industry thought leaders together to examine ways to make Ontario a world leader in a $9 trillion global market. Yes, you’re reading that right. The global health sciences economy is a $9 trillion annual market – and to put that in perspective, the global automotive industry is a little over $1 trillion. While many of our research institutes and universities are world-class and turn out leading-edge research and innovation, and Ontario boasts 1,900 life science companies that contribute $21.6 billion in GDP and provide 60,000 well-paying jobs, we’re lagging on our potential. Despite the presence of many emerging companies, we don’t yet have a homegrown “anchor” company that is a global brand and dominant leader in its field. We need one. When looking at other jurisdictions around the world that have strong and growing health science sectors, a common key feature is an “anchor company” that attracts talent and capital and spins off other companies, research and investment that grow the local ecosystem. Look at Medtronic in the Minneapolis/St. Paul area, or Pfizer in Connecticut, or Novartis in Basel. These companies anchor a cluster of innovation and economic activity in their regions that, in turn, spins off thousands of other companies and jobs. Ontario has companies that may someday become these anchors, but we need multiple companies that can make Ontario a world leader and attract even more investment. The question becomes, how do we get there? When we brought together 50 industry leaders, investors, government leaders, academics and health system leaders together for a summit on this topic, we concluded that the key driver is access to capital – and in particular, “sticky capital”. We need investments that not only grow companies, but also reinvest returns in new ventures. We need to move from a model of selling our early stage innovation assets, to one that invests and grows innovation assets within the province to a point where they can supply a global market. In other words, Ontario needs to build a health sciences economy that will facilitate capital investment, and generate returns from novel technologies and Ontario-based companies that can export to a global market. To accomplish this, our group advised that the answers lie in close partnership and cooperation between academic institutions, hospitals and healthcare delivery agencies, the private sector and government – players that all have slightly different agendas and have not always worked together. All these players have a big role to play and a key driver of success will be coordinated action.

Biotechnology Focus / June/July 2017

More specifically, for our universities and research institutes to encourage the commercialization of the innovation residing in these academic institutions, we need a set of intellectual property commercialization terms that compare favourably to competitors around the world and are standardized across the province. Ontario’s hospitals and health care providers need to be key partners in adopting innovative technologies by shedding all barriers that might prevent them from being early adopters of technology. Being an early adopter of any innovation carries a degree of risk, but these risks can be mitigated and the rewards can be very high. Having a robust domestic market for innovative technology will provide the early market data and proving ground innovations need to thrive and attract global investors, and it means Ontario patients will have access to the very latest technologies and treatments. A true win-win. To attract capital, Ontario must continue to build an ecosystem that supports high growth health sciences business that export to a global marketplace and generate above-average returns for these innovative technologies. How do we do that? Continue to turn out the highest quality graduates in science and business from our post-secondary institutions and attract talent from around the world; continue to invest in the labs and commercialization infrastructure that is turning out innovative technologies; continue to showcase Ontario companies and innovations to markets around the world; and continue to build a business-friendly environment that is consistent, predictable and doesn’t penalize high-risk investments. On paper, it sounds like a simple prescription, but if we execute on this strategy – and stay focused on it – the potential rewards of improved patient outcomes, increased health care efficiency and greater prosperity for all are achievable. Let’s get started.

About OBIO® The Ontario Bioscience Innovation Organization (OBIO®) is a not-for-profit, membership-based organization engaged in the development of an integrated health innovation economy for Ontario and one that will become a global leader in providing health technology products and services to the international marketplace. OBIO advances this goal through advocacy, promotion and strategic leadership and via collaborative partnerships with industry, academia, the health system and government. It’s latest white paper “Tackling the Anchor Company Challenge” can be found at: www.obio.ca. Follow OBIO on Twitter @OBIOscience.

Versant Ventures By Nestor Arellano

ONTARIO

Q&A with Brian Bolzon of Versant Ventures Building a new company is not just about finding great IP Q&A – Brian Bolzon of Versant Ventures on the risk and rewards of launching biotech startups

2011. Inception Sciences is a small molecule pharmaceutical incubator. It focuses on creating high-value therapies that have the potential to address disease with significant unmet needs. We followed this will an Inception Sciences in Montreal. Then we set up our Blueline Bioscience incubator in Toronto. I think our biggest accomplishment is being able to cover the major biotech communities in Canada.

Q: What makes Versant’s Discovery Engines unique?

Founded in 1999, Versant Ventures, a venture capital firm which invests in early-stage companies across the healthcare sector, has in excess of $1.9 billion under management and offices in North America and Europe. Versant rocked Canada’s biotech industry last December with its announcement of a $295 million partnership with German pharmaceutical giant Bayer AG to launch Toronto-based stem cell company BlueRock Therapeutics. Biotechnology Focus recently had a chance to speak with Brad Bolzon. The managing director of Versant was candid in discussing his company’s Canadian operations and what he thought about the country’s biotech industry.

Q: What are the most recent investments Versant in Canada? We established our footprint in Canada by setting what we call Discovery Engines. They are incubators we build to help set up companies. Our first Discovery Engine in Canada was Inception Sciences in Vancouver in

Many early-stage biotech venture firms that do raw startups have the same system of incubation. They either look for viable startups or build them. We’re no different. The major distinction is that, unlike incubators that have two or three people sitting in a room scanning the startup landscape, we actually have wet lab facilities. These are facilities operated by scientists who not only look for opportunities but actually conduct experiments to validate the science. This gives us a real competitive advantage in that we are able to actually collaborate with academics to validate their research and take them to the next stage so that they can be ready for a startup company. Versant has up to 100 scientists in labs in San Diego, New York, Vancouver, Toronto, Montreal and Basel, Switzerland. We raised our sixth fund at the end of 2016 at $400 million. We now have a total of $2.3 billion in global capital.

Q: How many Versant companies do you have in Canada? In Vancouver, we have Inception Four, an ophthalmology startup working on macular degeneration in collaboration with Bayer Healthcare. In Toronto, we have Northern Biologic, an explosive Series A commitment out of U of T in the oncology area. Then in Montreal, we have Inception IBD, a company focus on inflammatory bowel disease that has partnered with Celgene. Then we made an investment in an existing company, Turnstone Biologics in Ottawa. Late last year, we created BlueRock through a joint venture with Bayer. All four are Versant-created companies. Turnstone was an existing company.

Q: Four years after opening shop

in Canada, what’s your current assessment of the biotech industry in the country?

I’m a Canadian born in Ontario. One of my first jobs in the industry was working for Eli Lilly Canada searching for collaboration opportunities. We are familiar with the capability of the Canadian research community. Clearly, there was not an emerging venture ecosystem to match it…I think we’ve

Q: Do you create your own companies?

Yes. I think 1/3 to 40 per cent of our joint funding is expected to be a Versant company.

Biotechnology Focus / June/July 2017

Versant Ventures

ONTARIO been very surprised we exceeded our expectations. We’ll continue to use Canada to build our portfolio…In the next quarter, we will be announcing the launch of our second Montreal startup that will be in the cancer field.

There are not that many life sciences venture capital companies in Canada, particularly in the early stage. So we have to partner with large pharmaceutical firms as we did with BlueRock – and we have been successful at this.

vestors…at the end of the day, we succeed through collaboration. Historically the shortcoming in Canadian bio has been a shortage of capital. Most projects require $100 million to get past liquidity.

Q: In what areas is Canada leading?

Q: What are the challenges faced by venture cap firms in Canada?

Q: What do you think of the federal

When one builds a company, it’s not just about finding a great technology and IP. It’s about building a great team, a great financing strategy, great operating business model. In Canada, when it comes to building a great team, it’s difficult. There are not that many experienced entrepreneurs that have been there and done it before. We often have to bring in a first-time CEO and mentor them. Mentoring takes time. The second challenge is finding co-in-

I think Canada has one of the most supportive governments when it comes to helping the venture capital industry. In fact, very little public funding goes to ventures in Europe and the U.S. The Canadian government is very impressive.

Work going on in the U of T network in CCRM is world class. John Bell’s work in oncolytic in Ottawa is world class. Oncology and regenerative medicine.

Q: Where is the country lagging? Scientifically, Canada has always been strong. I think it’s in the translation piece that there is some challenge. We’re off to a great start, but at the end of the day, we have to take these companies to the finish line.

CCAB:

government?

To see this story online visit http://biotechnologyfocus.ca/ qa-with-brian-bolzon-of-versantventures/

By: Christine Misquitta

Translating Early Stage Biologics Into Your Products The CCAB translates early stage biologics into high-value assets and products. We have an extensive portfolio of fullyhuman antibodies, discovered by the University of Toronto’s antibody R&D engine: the Toronto Recombinant Antibody Centre (TRAC). Work with us to access world-class expertise in synthetic antibody discovery, state-of-the-art phage displayed libraries, and proprietary screening technologies. The CCAB is strengthening the Canadian drug development landscape by accelerating academic research and, together with investors or partners, further developing select therapeutic programs toward the clinic. In addition, we also offer a number of antibody reagents, tools and technologies for licensing. Talk to our Business Development team to see how CCAB can find a custom solution to your company’s antibody needs.

Biotechnology Focus / June/July 2017

Christine Misquitta leads the general administration and communications activities for CCAB. This includes coordinating outreach events and media on behalf of CCAB for both the scientific community and general public as well as the writing of large scale funding proposals and their management once awarded. In this role, she is also the primary liaison with government, industry, partners, and other academic organizations. Christine has a background in molecular biology and obtained her PhD at McMaster University in Hamilton.

Special report By Noel Courage and Phil Goldbach

ONTARIO

Identifying the Last Line of Defence for Innovative Canadian Drugs Patents and Data Protection We reviewed innovator drugs approved by Health Canada in 2015-2016 and found that there was no clear winner between the legal protections offered by Canadian patents and data protection. Both are important. In some cases, a key patent expires after data protection. Other times, data protection outlasts the initial patent. We found that patent protection offers longer-lasting exclusivity than data protection for the majority of new, innovative drugs approved in this two year time period. However, data protection is the primary line of defence against generic market entry for about ¼ of the new drugs because there are no patents listed with Health Canada.

Overview of Exclusivities

Patent and regulatory exclusivities are crucial to protect innovative drug companies from generic competition. The government created these time periods of exclusive rights to recognize the massive amounts of time, money and effort required to arrive at any single new drug approval. Generic drug companies are later able to provide the same drugs at lower cost, by, among other things, relying on the brand name drug company’s clinical trials. Thus, innovator drug exclusivities are necessary to ensure that innovative companies can profit from their inventions, and re-invest in future R&D. In Canada, patent exclusivities typically provide a 20-year monopoly from the filing date (no patent term extension yet). The monopoly provides the exclusive right to make, use and sell an invention. The patent that covers an approved new drug compound (or the initial approved indication) is often the most commercially important. Later patents covering new uses or formulations are often easily designed around by generic companies. Quality matters more than quantity when it comes to patents. Regulatory exclusivities, such as data protection provide an eight-year basic exclusivity term in Canada where a generic drug company cannot rely on an innovator company’s approval to get its generic marketing approval. This effectively bars the generic company from the market, since it is not likely to conduct its own clinical trials. We reviewed new drugs approved in 2015 and 2016 to determine whether innovative companies were more likely to rely on patents or data protection as the last defence against generic competition. Patents might initially appear to be more advantageous due to the 20-year term. However, much of the patent term gets chewed up while the drug is still on the long road of drug development and approval.

Exclusivities by the Numbers

Almost all new active substances approved by Health Canada, with only a few exceptions, qualified for data protection (the exceptions also did not have patents listed with Health Canada). In 2015, eight of 36 new substances had data exclusivity but no patent on Health Canada’s Patent Register. Similarly, in 2016, it was nine of 35. Therefore, data protection fills an important gap in a significant number of cases where there is no patent (or a time lag before listing the patents). Where there was at least one patent on the Health Canada Register, patent exclusivities tended to last longer: • data protection was the last-expiring exclusivity only in about 20 per cent of cases in 2015 and 35 per cent in 2016; • the first patent on the Register outlasts data protection almost 40 per cent of the time for drugs approved in 2015 and 2016. The first-listed patent is often significant because it typically provides the broadest protection, for example, covering the compound or the approved indication; • in the remaining cases, data protection outlasts the first patent but not one or more later-filed patents. These later patents may be improvement patents, which are typically narrower and easier for generic companies to circumvent. The effectiveness of improvement patents varies case-bycase. We also note that patents, particularly improvement patents, are sometimes invalidated in litigation, whereas data protection is infrequently challenged. This analysis supports the conventional wisdom that innovative drug companies should continue to pursue patents and data protection, whenever both are available. They provide complimentary protections. Noel Courage is a partner with Bereskin & Parr LLP and member of the Life Sciences practice group. Noel’s practice focuses on the patenting and licensing of biotechnological, chemical and mechanical inventions. He provides opinions on patent validity and infringement issues. He also has experience in regulatory, trademark and copyright law, and provides advice on IP portfolio management strategy. His regulatory work includes advising on data exclusivity, Patent Medicine Prices Review Board and Patented Medicines (NOC) Regulations strategy. Phil Goldbach is an intellectual property manager at MaRS Innovation (MI) in Toronto, Canada. Federally supported through the Networks of Centres of Excellence, MI is a not-for-profit organization and commercialization agent for 15 leading Ontario academic institutions.

Biotechnology Focus

June/July 2017

Special report

It’s a good time to invest in oncology innovation

ONTARIO

By Jeff Courtney, President, Fight Against Cancer Innovation Trust (FACIT)

an additional $286 million to drive the commercialization of promising oncology innovations.

Dollars alone, however, won’t drive innovation to market. A whole array of capabilities is needed to convert great ideas into saleable solutions: business leadership, intellectual property management, product development, planning and more. We surround early stage technologies with commercial teams that have the required skills and experience to build and grow sustainable companies and deliver results. In many cases, we have and will continue to create start-up companies to guide and advance specific technologies.

nnovation is being called for in virtually every corner of the public sphere. Federal Budget 2017 outlined a national Skills and Innovation Plan. Ontario singled out health innovation as a priority in its 2017 budget. In February, the Advisory Council on Economic Growth identified healthcare and life sciences as one of eight key sectors for Canada to focus on. This is good news for investors because it means there are going to be new opportunities — and more of them — to back new solutions and technologies, especially in the health sector. But opportunities on their own are not enough. The right elements need to be in place to realize them successfully. In Ontario, where health technologies are concerned, those right elements include focused support for innovations in the “awkward phase” of having outgrown the academic granting system but not yet matured to the point of producing robust evidence of effectiveness. This is a crucial time when investment is sorely needed, but for investors represents high risk. FACIT, the Fight Against Cancer Innovation Trust, was created to mitigate that risk so that cancer-fighting innovations can get out of the lab and into the marketplace — where they can reap returns for investors while delivering health benefits to Canadians and benefitting Ontario’s economy.

That was the case with Triphase Accelerator Corporation, which we founded with our strategic partner, the Ontario Institute for Cancer Research (OICR), as well as MaRS Innovation and MaRS Discovery District. Triphase developed a proteasome inhibitor to treat glioblastoma — a type of brain cancer — as well as forms of multiple myeloma. Last November, the company proved its capability to serve as a clinical proof-ofconcept accelerator when it sold assets for that innovation to Celgene Corporation. That’s just one example. With Turnstone Biologics, we brought multiple organizations together into one corporate body and drew more than $65 million in venture capital from Versant Ventures, OrbiMed Advisors and F-Prime Capital. We helped to launch Fusion Pharmaceuticals and facilitate its attraction of financing from J&J Innovations-JJDC, HealthCap, TPG Biotech and Genesys Capital.

Working as we do creates a pipeline of projects, giving investors access to a greater range of technologies to consider investing in while increasing the capital pool. It’s a model that works for all stakeholders: investors, innovators, governments and patients. The proceeds we earn as a shareholder in commercially successful ventures like Triphase will be reinvested in other oncology innovations in the province, fuelling the cycle. Since its inception, FACIT has actively helped bridge The Advisory Council on Economic the oncology innovation gap in Ontario. Growth has called for a “reimagining” of government as a convenor, catalyst and investor to drive innovation. As an arm’slength instrument, we perform all three Investments Companies roles, recognizing that commercializing formed innovation is a complex and necessarily cooperative endeavour. Our invitation is FACIT Returns External investment for investors to join us, leverage our process and model, and seize the opportunities that the current focus on innovaadditional total funding and value returned tion is likely going to make increasingly ﬁnancing attracted equity investments to shareholders abundant in the years to come.

That de-risking entails many things. First, we are a funder, committing capital to thoroughly vetted projects with high potential. Since inception, we have made $30 million in funding and equity investments. That stake has helped attract

$30M

$286M

$353M

277

Jobs created

metrics as of March 31, 2017, jobs created as of March 31, 2016

Biotechnology Focus / June/July 2017

Looking to invest in oncology innovation? Contact FACIT today: facit.ca/contact

BlueRock

ONTARIO

What is BlueRock working on? Described as the country’s second-largest biotech underwriting, the US$225-million deal between Versant Ventures and Bayer to create BlueRock Therapeutics could be taken as signal that Canada’s regenerative medicine space is ready for the big time. “Canada was chosen in the first place because the relevant experts for cardiology / stem cell approaches are in Toronto,” said Dr. Juergen Eckhardt, head of Bayer Venture Investment, Bayer Lifesciences Centre. “The people around the top scientists Dr. Gordon Keller and Dr. Michael Laflamme are key for BlueRock.” “The University Health Network, Centre for Commercialization of Regenerative Medicine, and MaRS, stand for the perfect scientific set-up and an inspiring environment for this ‘moonshot’ project,” he added. Accessing stem cells technologies is part of Bayer LifeScience Centre’s strategy. Stem cell technologies have a unique potential to address diseases that do not have a cure today as multifactorial issues are damaging tissues irreversibly. “One can see that in particular, in neurodegeneration and tissue damage following a heart attack,” said Eckhardt. Bayer believes there are two novel technologies that have the potential to treat disease at the roots: one is DNA editing and gene therapy that addresses inherited mutations in our genes that can cause disease. The other one is stem cell technology that allows regenerating tissues that have been damaged because of the normal aging process or disease. BlueRock was created “because we want to develop transformative, stem cell-based medicines for patients with serious cardiovascular and neurological diseases based on the latest stem cell technology,” said Eckhardt. He noted the advent of induced pluripotent stem cells (iPSC) has been among the major progresses in the past year. iPSCs

Bayer AG and Versant Ventures are joined by Federal Minister of Innovation, Science and Economic Development Navdeep Bains and Ontario Premier Kathleen Wynne, during the launch of BlueRock Therapeutics, a cutting-edge stem cell therapeutics company. (L to R: Dr. Jerel Davis, Dr. Axel Bouchon, Hon. Navdeep Bains, Hon. Kathleen Wynne, Dr. Bradly Wouters, Dr. Gordon Keller, Alok Kanti) (CNW Group/Bayer Canada)

are stem cells that are not derived from human embryos - and novel gene editing technologies provide a unique opportunity to enter the field. iPSC technology shows significant advantages over other less-developed stem cell technologies. A team led by Laflamme is also conducting research into the use of ventricular cardiac muscle cells to treat scar tissues that form on the heart after a heart attack. BlueRock is also collaborating with

Dr. Lorenz Struder at Memorial Sloan Kettering Institute’s Center for Stem Cell Biology in New York. The work will focus on the use of pluripotent stem dell-derived neurons to treat Parkinson’s disease.

To see this story online visit http://biotechnologyfocus.ca/ what-is-bluerock-working-on

Biotechnology Focus / June/July 2017

Ministry of Research and Innovation Compiled by Nestor Arellano

ONTARIO

In Conversation with

Ontario’s Minister of Research and Innovation Reza Moridi Beyond the media hype of artificial intelligence, large companies are beginning to take notice of AI more seriously. AI’s impact is certainly being felt in Ontario’s biotech sector and recently the Toronto’s Vector Institute grabbed headlines with reports of it being one of the chief reasons why AI researchers around the world want to migrate to the city. Ontario Minister of Research and Innovation Reza Moridi weighs in on AI and the Vector Institute.

Q: Artificial intelligence has become a buzzword of sorts lately. What are your thoughts on AI? The concept of AI is not really new. Researchers have been talking about it back in the 1970’s, 1960’s or even earlier. There has always been an interest in the possibility of replicating the functions of the brain…providing machines with some semblance of human intelligence. I think AI will revolutionize the biotechnology industry. AI and deep learning advances will have a very profound impact. Look back to when the Internet was invented. It was invented primarily by scientists in concert with the Department

of National Defence to get computers to talk to each other. Today, many people cannot imagine life without the Internet. Everything is connected. I think AI will have the same impact on our daily lives.

Q: Where do you see the potential for AI in the life sciences and biotech field? I see AI assisting human researchers… making them conduct more accurate, efficient, and faster research. AI is being applied in stem cell research, gene sequencing, and regenerative medicine. There are a lot of potentials for us to be pioneers in AI research.

U of T Associate Professor Raquel Urtasun (second from right) with (from left) Minister of Finance Bill Morneau, Ontario Minister of Research, Innovation and Science Reza Moridi, Premier Kathleen Wynne, Mayor John Tory and U of T President Meric Gertler (Image courtesy of U of T)

Biotechnology Focus

June/July 2017

Q: What is the Vector Institute and what are its goals? The Vector Institute was just launched in March 2017 with support from the Government of Canada, Government of Ontario, the University of Toronto and private industry. As an independent, non-profit research institution it is focused on the field of artificial intelligence, deep learning and machine learning. It collaborates with Ontario’s academic institutions and other organizations and companies across the country in every sector. The Vector Institute will conduct research to drive adoption and bring to market AI technologies not only in the biotech sector but also in finance, environment, cleantech, transportation, healthcare, and advanced manufacturing. The Vector Institute has three main goals: become a world-leading centre for AI research; develop the machine learning PhDs and masters students in deep learning and machine learning; and spur the creation of AI super clusters to drive the economy of Toronto, Ontario, and Canada.

Q: How much money is being invested into the Vector Institute? So far, Ottawa is putting in up to $50 million, the province is investing $50 million. More than 30 private sector firms are investing $80 million.

Ministry of Research and Innovation

ONTARIO University of Toronto Researchers at the core of Vector Institute. Front, from left: Roger Grosse, Richard Zemel, Brendan Frey, Raquel Urtasun and David Duvenaud. Back, from left: Jordan Jacobs, Ed Clark, Geoffrey Hinton, Sanja Fidler and Tomi Poutanen (photo by Johnny Guatto)

Investment in research Q: Why is it important for Ontario to have something like the Vector and science is important Institute? for our economic Our economy is in a critical transition development, particularly point. We’ve started a knowledge-based economy. Investment in research and in the life sciences and science is important for our economic development, particularly in the life sciences health sector. and health sector.

Ontario needs to establish a foundation for developing talent in the AI field. Students seeking to learn about AI and researchers seeking to build careers in the field can turn to and place like the Vector Institute. It will eventually provide companies with the talent they need to build their AI labs or grow their business.

Q: Why should Canada invest deeply in AI? We have to invest today in AI research in order to be ahead of the game. We either go ahead innovate, design and build AI technology to sell to the world or else we will be the once buying it from others. We have to put more scientists in the field. We have to give them the means to innovate.

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

CELEBRATING

YEARS

Of Delivering High Impact Results

Ontario Bioscience Innovation Organization Advocacy Action Access

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

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

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

www.obio.ca

@OBIOscience Biotechnology Focus

June/July 2017

Special report By Lisa Willemse

ONTARIO

Ontario’s Regenerative Medicine Pipeline Ontario stem cell researchers, led by the Ontario Institute for Regenerative Medicine are making gains to ensure the promise of regenerative medicine is not lost in translation. As the developmental pipeline for cell-based therapies rapidly accelerates worldwide, Ontario-based researchers continue to push the envelope with home-grown clinical trials. Of the $3.6 million in research funding awarded in May, 2017 by the Ontario Institute of Regenerative Medicine (OIRM) and supported through funding from the Ontario Ministry of Research, Innovation and Science, $3 million went to five large OIRM Disease Team grants. These projects focus on delivering new curative therapies for conditions that drive large costs to the healthcare system, as well as forging global opportunities for economic development based on stem cell based therapies. “Currently in Ontario there are at least 13 clinical trials under way or about to begin that are assessing stem-cell based treatments for debilitating illnesses and injuries such as heart disease, septic shock, knee osteoarthritis, malignant brain tumours and more,” says Dr. Duncan Stewart, OIRM president and scientific director. “Twelve of these trials are led by OIRM member researchers, which is a testament to the depth and breadth of expertise we have in this province, and the level of support that exists for regenerative medicine research here.” Three years ago, there were only five trials in the province – an increase of more than 150 per cent. Launched in 2014, OIRM is dedicated to the translation of stem cell research into curative therapies for major degenera-

Biotechnology Focus / June/July 2017

“We need to emphasize high-quality translational research so we can identify which therapies work in which patients, and how the healthcare system can fully adopt their use.” — Dr. Duncan Stewart, OIRM president and scientific director

tive diseases. More than 210 research programs at universities and institutions across the province are involved with OIRM, with additional contributions from key clinical and health charity partners and from OIRM’s commercialization partner, CCRM. “The potential to address some of these very complex diseases, some of which are common and some rare, is enormous. However, we also make these treatments as costeffective as possible, and therefore widely accessible through Ontario’s health system,” Dr. Stewart explains. “Therefore, we need to emphasize high-quality translational research so we

ONTARIO can identify which therapies work in which patients, and how the healthcare system can fully adopt their use.” At The Ottawa Hospital, a research team led by neonatologist Dr. Bernard Thébaud is working on a stem cell therapy that may mitigate the damage caused by bronchopulmonary dysplasia (BPD), a chronic lung disease that often develops following ventilator and oxygen treatment in extremely premature babies. Dr. Thébaud was awarded an OIRM Disease Team grant in May to conduct preclinical work, that, if successful, will lead to a human clinical trial as soon as 2018. The appropriately named “INCuBATOR” project uses mesenchymal stem cells (MSCs) from discarded umbilical cord tissue – a relatively untapped source of stem cells – to stimulate growth and repair in the lungs. Dr. Thébaud’s team has worked extensively with these cells, having both proven their effectiveness in rats while developing a system to produce large quantities of the cells that would meet the stringent requirements of human treatment. Before a single stem cell is given as a therapy, Dr. Thébaud says it is essential to answer as many questions as possible. Poor design or a lack of comprehensive answers is a common problem that can halt trials in their tracks. But for developing infants in the first months of life, whose vision, cognitive and motor development, and digestive systems are all dependent on the ability to get enough oxygen, time is of the essence. “The idea is to accelerate the translation from the bench to oirm-Biotech-ad-7x4-F.pdf

2017-05-23

the bedside, in an evidence-based fashion. We want to show proof-of-concept that a cell product may work for babies with BPD, then streamline everything so that we can move quickly from the bench into patients.” Fragile lives require a good deal of care, which is why the project includes both the necessary pre-clinical work (in an animal model that very closely mimics the human condition of BPD) as well as a survey of the infants’ parents and health care professionals to assess the level of risk they are willing to accept. “The goal is to find obstacles or facilitators for clinical trials. In this way, we’ll be able to design a trial that makes sense, but that will also have buy-in from the parents and clinicians,” Dr. Thébaud explains. For Dr. Stewart, this work is representative of the burgeoning stem cell research and commercialization community in Ontario, led by OIRM and its partners. “Ontario has a deep history of excellence in regenerative medicine – stem cells were first identified at Princess Margaret Hospital in Toronto, and today we are ranked third in the world in terms of the impact factor of discoveries made here,” he says. “I think we are building toward a critical mass, where we’ll see frequent, significant outcomes that improve human health. But, to get there we need to ensure we have the resources to conduct trials in a meaningful way.”

9:42 AM

CELL THERAPY CLINICAL TRIALS IN ONTARIO IN 2017

BRAIN AND/OR NERVES

HEART

Phase II using stem cells for Multiple Sclerosis: Ottawa*

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

Phase II using diabetes drug for brain repair in children with cerebral palsy: Toronto* (Starting Q3 2017) 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*

CMY

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

JOINTS

2017 13 trials 9 partners 4 Ontario cities

Phase II using enhanced stem cells at the time of coronary artery bypass grafting: Toronto* (Starting Q4 2017)

LUNG Phase II using genetically modified stem cells for pulmonary arterial hypertension: London, Ottawa and Toronto* (Starting Q1 2017)

200+ patients Billions of stem cells

CRITICAL CARE

Phase I using stem cells for septic shock: Ottawa* Phase II using stem cells for septic shock: Hamilton, Ottawa, Toronto*

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*

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

(Starting Q2 2017) *OIRM researchers leading trial

@oirmnews

oirm.ca

Biotechnology Focus

June/July 2017

Shire By Nestor Arellano

ONTARIO

Shire shines

as latest addition to TORONTO’S biotech cluster Toronto’s bustling health and biotech community attracts the company which recently acquired Baxalta in a US$32-M merger

o say that Eric Tse is very busy these days would be an understatement. The general manager of Shire Pharma Canada ULC is still in the thick of consolidating staff and operations with Baxalta which Shire acquired in June last year for US$32 million. He’s also winding down a recent hiring blitz. And he is preparing the new Shire digs in downtown Toronto. By the time the dust settles on the big move some 100 personnel from Baxalta’s facility in Mississauga and the Montreal location of Shire will have been moved to a gleaming new glass-faced building near the intersection of Toronto’s Bay and Adelaide streets. Canadian lab work will continue to be done in the facilities of organizations that Shire collaborates with. The new office will focus on commercial marketing, sales, regulatory, quality assurance and tech operations, according to Tse.

Biotechnology Focus

June/July 2017

The newly-merged companies’ office will be at the heart of the city’s financial action but it’s the proximity to Toronto’s bustling health and biotech prime players that was a major draw for Shire. “Arduous assessments were done to find where to locate the new office,” said Tse. The criteria included proximity to stakeholders, customers, peers, the ecosystem, and access to multiple and convenient transportation routes. “Toronto was a real neat place to relocate. The university health network is near…you can walk to many locations. External stakeholders in the biotech and healthcare fields are nearby, and there’s

a high density of customers and government agencies,” said Tse. Moving to Toronto, he added, would also help Shire in its long-term talent search because the city is currently attracting a lot of promising and wellestablished researchers. Toronto is the most populous city in Canada and it has the country’s largest biotech cluster. More than 160 biotech firms have offices in the Greater Toronto Area. More than 50 global pharmaceutical companies have their Canadian headquarters in the Toronto region. The location provides a rich life sciences

“Toronto was a real neat place to relocate. The university health network is near… you can walk to many locations. External stakeholders in the biotech and healthcare fields are nearby, and there’s a high density of customers and government agencies.” — Eric Tse

Shire

“…Shire is now the global leader in rare diseases, with the number one rare diseases platform based on both revenue and pipeline programs.” — Dr. Flemming Ornskov talent pool with more than 11,000 principal researchers and technicians operating out of the University of Toronto Faculty of Medicine. There are eight other universities and colleges, nine teaching hospitals and numerous private sector research facilities in the area. These are heady times for Shire. Last year’s acquisition of Baxalta has catapulted Shire into global leader status in the rare diseases and specialized disorder fields. Shire brought into the merger its portfolio of products in gastrointestinal/endocrine diseases, hereditary angioedema (HAE), neuroscience, and lysosomal storage diseases. Baxalta contributed its

hematology, immunology, and oncology products. The resulting “synergies” are expected to create more than $500 million in savings. Shire projects that the combined companies will generate somewhere around 65 per cent of its annual revenues from rare disease products. Revenue is forecast to reach over $20 billion by 2020. The combined company has more than 22,000 employees across more than 100 countries. It now has more than 50 programs in clinical projects in either early, mid, and late-stage development. “…Shire is now the global leader in rare diseases, with the number one rare diseases platform based on both revenue

ONTARIO and pipeline programs,” Dr. Flemming Ornskov, Shire’s CEO, had said last year. In late May, this year, Royal Bank reiterated its outperform rating on shares of Shire PLC. BNP Paribas also reissued an outperform rating and set a GBX 5,900 ($76.16) price target on shares of Shire PLC. Having a presence in Toronto will support the company’s profile as a leading biotech company in Canada, said Tse. “Our model is a mix of internal knowledge, capabilities, and research, combined with collaborations with external partners,” he said. “Partnerships are key. We need to work with stakeholders across the health care ecosystem to be better equipped to bring new treatment to patients.”

To see this story online visit http://biotechnologyfocus.ca/ shire-shines-as-latest-additionto-torontos-biotech-cluster/

Advancing the Success of Ontario’s Life Sciences Sector Ontario has the innovations, research, and talent to become a global leader in life sciences: • Critical mass of innovation: Ontario ranks among the Top 3 North American jurisdictions by number of life sciences establishments • Intensifying investment: JLABS chose Toronto, Ontario for its ﬁrst incubator outside the US. Bayer and Versant recently established its $225 million BlueRock Therapeutics here. • Skilled workforce: Our 44 universities and colleges – including 6 aﬃliated medical schools – produce +40,000 skilled STEM graduates annually. • Rapid growth: Our sector’s job growth outpaced provincial averages by nearly 10 per cent from 2001-2013

• An ecosystem like no other: Toronto’s core research district brings together world leading research institutes, innovation centre MaRS Discovery District, hospitals, and a private sector corridor with connections to tech hubs in Waterloo & Ottawa.

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

Read the report at: bit.ly/LSOSectorReport Follow us on Medium at: Medium.com/@LifeSciencesON

Become a member at www.lifesciencesontario.ca Biotechnology Focus / June/July 2017

Gamification By Nestor Arellano

ONTARIO

They’ve got game How gamification is being used in brain health research Can elements of game be used in addressing some of the challenges of brain health and the world’s aging population? Panelists for the discussion of the topic of Game Theory and the Brain in the 2017 International Bio Convention in San Diego, Calif., talk about what online quizzes, and video games have to do with promoting better brain and mental health.

amification can be described as the method of integrating the mechanics of game into an existing process or system in order to motivate participation or engagement. In the digital age, the method has been used in various applications from online learning, to military training, fitness tracking, and customer loyalty programs. Brain health is often referred to as our generation’s big healthcare challenge. Many healthcare practitioners, researchers and engineers believe gamification can significantly help address that challenge “We are in a very exciting moment in which games and gamification can be used in new ways,” according to Dr. Sarah Diamond, president of the Ontario College of Arts and Design (OCAD) University, and moderator of the discussion panel. “We can use technology to study the ways that our brains work when we are immersed in game play through data analysis.” For instance, she said, technology can be used to produce biofeedback, and to

Biotechnology Focus

There are opportunities to apply gamification to address the brain health needs of seniors, and impact the cost of care as well. — Dr. Sarah Diamond provide feedback that further engages people’s activities. “Given the links between physical exercise and brain health, tools can enhance engagement in physical play. Or, feedback can enhance relaxation activities,” said Diamond. “We can gamify survey and other tools related to brain health information collection to encourage users’ ongoing use of tools.” Diamond noted that recent StatsCan figures indicate that for the first time in history there are now more people in Canada aged 65 and older that there are 15 years and younger. Healthcare costs for Canada’s seniors looms like tsunami in the horizon, she said.

“I am on the advisory board for the Canadian Centre for Aging & Brain Health Innovation, a consortium led by Baycrest to find solutions to improve the lives of seniors and build effective interventions in relation to dementia,” said Diamond. “Gamification and games can help in areas such as diagnosis and engagement. On the other side of the spectrum, being able to use brain games to reduced stress or anxiety for youth and young adults may lead to better brain health as we age.” The following are examples of the brain and gamification research that the panelists are involved in:

An early way to test for dementia could help transform Alzheimer from an epidemic without hope to a disease that can be managed. — Michael Meagher

Are you finding it more difficult to remember names and faces? Have you ever wondered if your failing memory is something normal or a sign of something more serious? Cogniciti has developed a free, online science-based brain health assessment tool.

June/July 2017

Cogniciti

GAMIFICATION

ONTARIO

Muse operates like an electroencephalogram (EEG), measuring brain activity and sending the data to an app so users can get feedback and improve their practice. — Dr. Graeme Moffat

Cogniciti is a private sector digital health firm founded in 2010 which serves as the commercialization arm for the Baycrest research and education hospital in Toronto. Baycrest is a global leader in cognitive neuroscience research, geriatric long-term care and community support programs for older adults. Nearly 50,000 people have already taken the assessment which has been research validated at Baycrest,” said Michael Meagher, president and CEO of Cogniciti. “Our goal is to help identify healthy adults at risk of dementia,” he said. “We want to be able to identify those people before the disease sets in.” If individuals as risk of dementia can be identified even before the debilitating signs of the disease surface, that persons and healthcare practitioners will have more time to develop treatment options that will help minimize the impact of the condition. “Just as blood pressure monitors and cholesterol tests help heart condition patients, an early way to test for dementia could help transform Alzheimer from an epidemic without hope to a disease that can be managed,” said Meagher. One of the key challenges though is how to get people to sit and concentrate on the assessment. By building in elements of game into to the system, Cogniciti was able to develop a self-administered assessment which currently 85 per cent of takers have taken the time to finish. But rather that incorporate elements reminiscent of video games, Cogniciti

opted for a more sedate feel that would appeal to people aged 50 to 80 – the company’s target audience. It’s about context, Meagher said. Other things that Cogniciti did with the assessment tool were: • Keeping the process clean and simple • Taking out the reds and green (some subjects are likely to be colour blind) • Eliminating rewards and buttons • Keep the process tied to purpose and make sure it delivers on its promise

lenges and rewards, encouraging users to build a more regular and effective practice. Muse operates like an electroencephalogram (EEG), measuring brain activity and sending the data to an app so users can get feedback and improve their practice. Moffat said technologies such as Muse that leverage data and data analytics and open up new possibilities to combine health and gaming to support brain health.

“Gamification is not a game. It’s about science,” he added. “Elements such as points and rewards that might work for a 20 or 30 something would not resonate with older people.”

Most traditional assessment methods can only be used in clinical settings, and are not engaging, said Prateek Dwivedi, chief executive officer of Ehave. Ehave is Toronto-based company that focuses on developing data-rich tools designed to improve patient management, diagnosis, and treatment. Ehave is developing Mega Team, a colourful game platform targeted at children. “Our goal is to build video games that are engaging as a starting point,” he said. “Then we add the technology that helps heal the brain.” Dwivedi believes games are uniquely positioned to help brain health because when properly designed, they can invoke cognitive activity. When tools fail to capture a subject’s attention, they pose several problems: expenses and infrequency of testing, and results that clinicians might not be able to rely on since patients are challenged to pay attention to the test. “Games have to be designed in a way that they engage and interest the target population,” Dwivedi said. “Failure to engage the patient will result in poor use, and thus inaccurate results and no remediation.”

MUSE Can a headband help enhance your meditation? Dr. Graeme Moffat leads neuroscience research and health and wellness applications for Muse, the maker of a brain sensing headband that elevates the meditation experience by gently guiding your meditation through changing sounds of weather based on the real-time state of your brain. Moffat holds a PhD in neuroscience and has more than a decade of research experience in psychology and neuroscience. He is a TalentEdge Fellow of the Ontario Centres of Excellence and a member of the Centre for Responsible Brainwave Technology (CeReB). The Muse app gives users access to personalized tracking, motivational chal-

Games have to be designed in a way that they engage and interest the target population — Prateek Dwivedi

EHAVE

To see this story online visit http://biotechnologyfocus.ca/ theyve-got-game/ Biotechnology Focus / June/July 2017

ONTARIO REGIONAL JURISDICTIONS

ONTARIO Ottawa

Sudbury

6 2

7 4

Waterloo

6 3

Life sciences Hamilton

Internationally recognized for its biotechnology strengths in the health sector, including drug discovery and validation, gene therapy and clinical trials, Hamilton offers life sciences companies the convergence of research, development and business collaboration in a unique and vibrant business setting. Commercialization is the cornerstone of bringing research to market. Hamilton is a solid return-on-investment choice for market success — with world-renowned research institutes and centres of excellence. Researchers at McMaster University, Hamilton Health Sciences, and St. Joseph’s Healthcare are consistently making groundbreaking discoveries, from life-saving drugs and vaccines to medical devices and solutions to current health issues. Hamilton’s Economic Development Office is the central point of contact for business assistance. Its services are geared to serve new start-up companies, corporate relocations, and the expansion and retention of existing business. Its mission is to serve as the catalyst for continued economic growth, job creation, and revitalization in Hamilton.

Life sciences Kingston

Healthcare is Kingston’s economic anchor. The city’s hospitals are teaching facilities and doctors are jointly appointed with Queen’s University. This partnership enables cutting edge medical research. For instance, the Human Mobility Research Centre serves as a point of collaboration between the disciplines of medicine, engineering, health sciences, and computer science; Kingston General Hospital is southeastern Ontario’s leading centre for complex-acute and specialty care; and Providence Care’s new state-of-the-art $800M facility is one of the region’s leading provider of specialized mental health care, rehabilitation, geriatric services, and complex continuing care among others.

Biotechnology Focus

June/July 2017

Kingston Toronto Missisauga Hamilton London

The Innovation Park at Queen’s University is the hub of Kingston’s innovation ecosystem. It is a community of innovators where academia, industry, government and not-for-profits work together to propel technological discoveries into the marketplace. PARTEQ Innovations provide Queen’s and partner institution researchers with the intellectual property and commercialization expertise that are needed to advance their discoveries to the public.

Life sciences London

With a rich history of achievements and medical firsts, the sector employs more than 21,000 in private sector industry, hospitals and research facilities including more than 2,000 researchers. The city’s breakthroughs date back to the 1920’s when Dr. Frederick Banting wrote his hypothesis which led to the discovery of insulin, and includes other firsts like the 2012 research by Western University’s Dr. Chi Yong Kang which resulted in the first ever preventative HIV vaccine. London is home to the Robarts Research Institute, CSTAR – The Canadian Surgical Technologies and Advanced Robotics Centre. It is one of only eight training centres in the world for da Vinci Surgical Systems, which trains and certifies surgeons to perform minimally invasive procedures. The Trudell Medical International is a world leader in aerosol drug delivery devices. Recently, the company unveiled its latest product – Aerobika – that sends vibrations into the patient’s lungs to help clear congestion in conditions like Chronic Obstructive Pulmonary Disease.

Life sciences Mississauga

Mississauga’s life sciences cluster is more diverse than ever. With over 23,000 in employment, the city’s life sciences sector is the second-largest in Canada. Mississauga has also launched a life

ONTARIO REGIONAL JURISDICTIONS

ONTARIO sciences strategy that will transform the cluster over the next five years. Mississauga’s life sciences cluster has over 230 companies. The cluster continues to expand beyond a pharmaceutical hub, with noticeable growth in medical devices, biopharma/rare diseases, diagnostics, and health IT. Mississauga continues to be a primary choice for business investment and is a proven destination where diverse companies and exceptional talent grow, succeed and prosper. With a strategic location amidst some of Canada’s leading education, research and healthcare centres, Mississauga is a well-connected ecosystem that cultivates and nurtures commercialization of life sciences innovations. www.winthehumanrace.ca

Life sciences Northern Ontario

Activated White Ltd. is a Northern Ontario company with a polymer resin called ActivatedWhite which adsorbs 80 times its weight in hydrocarbons. Among many other applications, ActivatedWhite can adsorb ultra-fine particles from the atmosphere or from combustion exhaust. Global smog issues continue to plague the environment and studies are continually highlighting the detrimental effects of ultra-fine particulates on human health including heart disease, stroke, dementia, Alzheimer’s and many other ailments. ActivatedWhite can remove close to 100 per cent of all elemental particles including those in the nucleation mode or nanometer size. ActivatedWhite is particularly relevant in personal masks for smog diversion and can be cost effectively scaled to solve some of the world’s largest persistent smog problems via ambient filtration or on power generation facility exhaust filtration. Activated White can help alleviate health ailments in many countries by scalable, inexpensive air and mask filtering. Verv Biotechnology is working on the development of a smartphone-based, consumer-focused blood biochemistry analyzer. A key component to the device is the Sudbury-based company’s microfluidic plasma separation technology. When ready for commercialization this device will enable people to monitor their health through standard blood tests such as allergies, vitamins, and cholesterol.” KE2 Technologies is a medical device company based in Sudbury that focuses on developing non-invasive sensors to improve the management of critically ill patients. KE2 Technologies’ flagship smart-bandage product, the FloPatch, addresses unmet clinical needs of critically ill patients and the physicians, nurses, and paramedics who care for them in the ambulance, emergency department, operating room, and intensive care unit.

Life sciences Ottawa

Ottawa has emerged as a centre for discovery, development, and commercialization for health technologies and medical innovation. With the highest concentration of scientist and engineers in North America, one in five students enrolled in STEM, and home to 68,000 ICT talent, Canada’s national capital region provides an innovative ecosystem for e-health solutions. Ottawa boasts of having more 20 organizations and research

institutes, including the Canadian Stroke Network, Stem Cell Network, and Biotherapeutics for Cancer Treatment. Five out of 10 Ottawa hospitals are found in Canada’s Top 40 Research Hospitals. They are home to more than 2,107 researchers, and account for over 1,500 clinical researches and active studies. Their combined research spending reached over $185million in 2015. Ottawa researchers are involved in five world leading clinical trials including; stem cell therapy for septic shock and doublevirus therapy cancer. The first nerve transfer surgery in Canada was carried out in Ottawa.

Life sciences Toronto

Toronto is where life sciences businesses come to thrive. Whether you’re looking for a home for your company or products and services to solve your problems, Toronto can probably deliver. Toronto is the fourth-largest city in North America and growing quickly, welcoming over 20,000 new people annually. It routinely wins livability awards and is considered one of the world’s most diverse, safe and cosmopolitan places. In addition to generous business supports, life in Toronto is one of the best perks you could offer your employees. In the life sciences sector, Toronto does important things extremely well. It is a global centre of excellence for regenerative medicine, biologics, oncology, artificial intelligence, chronic disease and other fields in the future of healthcare. Its downtown research park fosters collaboration in discovery and treatment, while manufacturing sites are connected via public transit. For more information, please contact Toronto’s Sector Development Office: www.toronto.ca/sectors

Life sciences Waterloo Region

Waterloo boasts an impressive life sciences ecosystem. The region’s areas of strength include health informatics, pharmaceuticals, agricultural biotechnology and medical technology. Unitron, a global innovator of advanced hearing solutions, announced a significant new win in 2017. The company boasts its Moxi Now is the world’s smallest wireless receiver-in-canal (RIC) hearing instrument. The device was awarded top prize for product design this year by the internationally esteemed Red Dot organization. Nicoya Lifesciences is a team of engineers and scientists with extensive experience working at the forefront of nanotechnology, biochemistry, and optical sensors. In 2016, Nicoya released the OpenSRP-XT – a device which provides affordable and fully automated binding kinetics for life science researchers from academia to the life science industry. Intellijoint Surgical develops and commercializes surgical navigation solutions. In March 2017, the company received clearance for its HIP Anterior, a 3D mini-optical navigation solution for Direct Anterior Approach (DAA) total hip replacement.

To see this story online visit http://biotechnologyfocus.ca/ottawa-network-ofexcellence/

Biotechnology Focus

June/July 2017 31

SPECIAL REPORT By Jason Field, President & CEO, Life Sciences Ontario

ONTARIO

Ontario life sciences on the rise The province’s life sciences sector is turning heads with its unique ecosystem – but can it make the leap to global economic powerhouse? Life Sciences Ontario’s Jason Field tackles the question. The Atlantic recently featured Toronto innovation hub MaRS Discovery District in a piece that asked the question – is Canada ready to compete for global talent? The answer is yes. And the buzz that’s been building around our tech companies is now intensifying to Ontario’s life sciences sector. Indeed, in 2016, Johnson & Johnson chose Toronto for its first JLabs life sciences incubator outside the United States, joining the ranks of outposts in major hot spots like Boston, San Francisco, and New York. “Here, you have the possibility of that whole spectrum of the healthcare system that doesn’t exist anywhere else in a square block like this,” JLabs’ Melinda Richter told the Financial Post. It was a major vote of confidence – and it set the stage for an incredible year for our life sciences ecosystem. In late August 2016, Sunovion Pharmaceuticals announced it was acquiring Toronto’s Cynapsus Therapeutics to the tune of $624 million. By December, Bayer AG and Versant Ventures had inked a US $225-million investment to establish a Toronto-based stem cell research company, BlueRock Therapeutics – one of the largest series A financings of a biotech company to date. According to Bayer, the investment was made due to innovation and infrastructure in Toronto, particularly stemming from the MaRS hub, and the region’s emerging profile for excellence in science. Hot on the heels of the BlueRock announcement came a number of key wins for Ontario companies: Toronto-based Synaptive Medical inked a key growth equity deal with General Atlantic. Ottawa’s Turnstone Biologics secured $41.4 million in financing led by OrbiMed, with participation from existing investors including FACIT and Versant Ventures. These are just a handful of examples among the many Ontario-based companies turning heads and attracting foreign and domestic investment and talent to match. So why is Ontario on the rise? Looking at the stats, it’s easy to see why we’re winning.

Biotechnology Focus

June/July 2017

Data from Life Sciences Ontario’s 2015 Sector Report indicates Ontario ranks among the Top 3 North American jurisdictions by number of life sciences establishments and the among the Top 10 by employment. Conservative estimates put the industry’s annual revenues at $40.5B, which directly contributes $21.6B to Ontario’s GDP. Six of Ontario’s universities have associated medical schools, including the University of Toronto, one of North America’s largest medical faculties. Our 44 universities and colleges produce more than 40,000 skilled graduates in science, technology, engineering and mathematics (STEM) each year. Between 2001 and 2013, the sector’s job growth outpaced the provincial average by nearly 10 per cent and demonstrated resilience during the 2008 economic downturn. It’s clear our sector has the research, innovations, talent, and partnerships to become a key driver of our knowledge economy. Foreign investors and global multinationals are seeing the value-add of Ontario’s ecosystem – including its scientific and research prowess and highly-educated talent base. However, there are challenges within our own walls. Our sector is in dire need of access to capital – particularly risk capital that will further incentivize investment. And, we urgently require the guidance of a unified life sciences strategy. This will create crucial alignment on policy across our diverse life sciences sector and all levels of government. This is the strategy that will shift Ontario life sciences from a “hot commodity” into a sustained economic powerhouse for years to come. The opportunity is in front of us: to truly seize it and capitalize on the rich resources we possess, we need a robust policy environment, a strong plan for the future, and a government willing to act on it. Dr. Jason Field is President and CEO of Life Sciences Ontario, a not-for-profit, memberfunded organization dedicated to advancing the success of Ontario’s life sciences sector.

CALENDAR

June 2017 June 19

Web: www.immuno-oncologysummit.com/ RESI (Redefining Early Stage Investments) Tel: 781-972-5400 Venue: The Westin San Diego Gaslamp Quarter, Email: chi@healthtech.com San Diego, Calif. Phone: +1 (617) 600-0668 September 2017 Email: RESI@lifesciencenation.com new card:Layout 1 1/31/2013 9:09 AM Page 1 September 11-14 Web: http://www.resiconference.com/

June 19 - 22 2017 BIO International Convention Venue: San Diego Convention Center; 111 W Harbor Dr., San Diego, Calif. Web: http://convention.bio.org/2017/ Registration: http://convention.bio.org/register/

49th Annual Canadian Mineral Analysts (CMA) Conference and Exhibition Venue: Coast Kamloops Hotel 1250 Rogers Way Kamloops, BC V1S 1N5 Contact: John Andrew New Gold Inc. New Afton Mine Tel. (250) 377-2885 E-mail: John.Andrew@newgold.com

September 21-22 Banff Venture Forum 2017 Venue: Fairmont Banff Springs, Banff, AB Web: http://www.banffventureforum.com/ Tel: (587) 350-3308

September 26 -27 Biopharm America Venue: Sheraton Boston Hotel, Boston, MA Web: https://ebdgroup.knect365.com/biopharm-america/ Email: conferences@ebdgroup.com Tel: +1 760 930 0500

June 20 - 23 2017 CSM Conference Venue: University of Waterloo, Waterloo, Ont. Contact: CSM 2017 Conference Organizer: Mrs. Wafaa H. Antonious Tel: 613 421 7229

SRC101

June 26 - 28 Epigenetic regulatory pathways Seoul 2017 Venue: Seoul, South Korea Web: http://www.abcam.com/events/ epigenetic-regulatory-pathways Tel: 617-577-4200 Email: events@abcam.com

july 2017 July 9 – 14 FASEB SRC — The TGF- Superfamily: Signaling in Development and Disease Venue: Lisbon, Portugal Web: http://www.faseb.org/SRC-Microsite/TGFB Tel: 301-634-7010 Email: src@faseb.org

July 9 -14 FASEB SRC — NAD + Metabolism & Signaling 2017 Venue: New Orleans, La Web: http://www.faseb.org/src/micro/Site/NAD Tel: 301-634-7010 Email: src@faseb.org

August 2017 August 19 -26 Neuroepigenetics and Epitranscriptomics Venue: Siena, Italy Web: http://www.nsas.it/courses-workshops/ advanced-courses/neuroepigenetics/ Contact: Francesca Martini Tel: +39.0577.146.0003 Email: info@nsas.it

August 28 – September 1 IMX — Immuno-Oncology Summit 2017 Venue: Boston, Mass

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

Yes

BIOTECHNOLOGY FOCUS

Format Preference:

digital

both

Signature:____________________________________Date: ___________________________

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

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

55 58 59 60 62 63 64 65 99

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

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

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

C86 Buying Influence A Authorize

B Recommend

A B C D E F G

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

H I J K L M Y

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

C88 Product Development Stage (check all that apply)

COMPANYs PRIMARY BUSINESS ACTIVITY 50 50a 50b 50c

C87 Which products are used in your lab?

C Specify

A Research/Development B Pilot/Scaleup

C Production/Manufacturing D Tech Transfer E Not applicable

C89 Are you building a new lab?

Yes

C90 We have the following enewsletters: 1 2 3 4

Biotechnology Focus eBulletin Laboratory focus eBulletin BioPharma BioMedical

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

For a quick response please fax: 905-727-4428 or e-mail: circulation@promotive.net Biotechnology Focus / June/July 2017

LAST word

| By Dr. Raphael Hofstein President & CEO, MaRS Innovation

The big guys are here to stay

get asked quite often “what is the biggest change you have seen during your time in Toronto?”. Recently, my answer is “the big guys have landed and they are here to stay”. Last year alone, Toronto welcomed J&J as it launched JLABS outside the United States for the first time, and Celgene established a strong strategic presence, also in Toronto. Add to it Bayer’s recent decision to include Ontario’s Centre for Commercialization of Regenerative Medicine and University Health Network as partners in their strategic commitment to cell-based therapy (via a new company called BlueRock) and EVOTEC’s establishment of a Canadian presence via a new partnership with MaRS Innovation, and we have the scent of some serious international clustering dynamics in the air. The discussion still ensues around how challenging it is to establish interactions between the public and the private sector, i.e. between governments and the health care industry. However, the long list of partnerships entered in the past year suggests that this relationship has taken a very strong turn in a positive direction. Part of the reason is the need to establish a paradigm shift from a strictly blockbuster approach to a more precision medicine driven attitude by industry leaders. An exemplary demonstration of addressing unmet medical needs is the recent decision made by Celgene to commit to the clinical development of Merizomib, a drug being developed by Toronto-based Triphase for the treatment of glioblastoma, a deadly malignancy with very few options for cure. At the time of acquisition, Celgene’s executives stated that the decision is consistent with the deep commitment and passion for the patients and their urgent needs. The drug is now in fast track development in Canada, the US and Europe. This is just one of growing number of examples illustrating that industry is rapidly adjusting to patients’ needs regardless of market size. Interactions with IBM’s Watson group as well as extensive dialogue with big pharma (Genentech/Roche, Medimmune/Astra Zeneca, and Merck, to mention a few) indicate that Canada is viewed as offering a fertile landscape in this area of precision medicine. The Canadian approach, which is essentially a longitudinal electronic medical record-based approach, offers one of the most comprehensive and user-friendly infrastructures on a global scale. Recently, the federal government and several provinces announced on a new initiative of data mining and processing by virtue of artificial intelligence with the expectation that AI will revolutionize the handling of Big Data in healthcare, among other sectors. The Vector Institute at the University of Toronto, with support from the private sector (Google, Telus and Shopify among others) as well as the AI institute at University of Alberta and the MILA (University of Montreal) are already reviewing ways of addressing the prospects of precision medicine. The opportunity for Canada is expected to grow significantly

Biotechnology Focus

over the next few years due to unprecedented pressures on health care systems world-wide. Global industry partners are recognizing Canada’s advantages in the academic sector and will continue to look to us for the development of new and disruptive technologies. In an era where information/communication technologies afre becoming so impactful on all aspects of life, healthcare strategies have to be adjusted as well; drug discovery, molecular diagnostics as well as medical technologies are already influenced by it. Canada has already established strong leadership in certain areas and the commitment of the federal government as well as the Ontario provincial government as reflected in the recently announced budgets suggest that there is a strong commitment to healthcare innovation but a lot more needs to be done so that our leadership position is preserved and we continue to be an attractive partnering opportunity for global leaders. As an example, the dialogue between global corporations and the regulators at all government levels requires streamlining so that innovative technologies get the right level of attention. There are useful models in the US and the UK which can be easily assessed in terms of the benefits that accrue to both the private and public sectors when a more modern approach is utilized. Such a dialogue paid off over the last four decades in the Bay area and in the Boston area where the interaction between the public and the private sector has always been one of win-win. Canada could do even better!

“We need approaches to the solutions that aren’t just arithmetic and additive, but are in some sense logarithmic. This will require us to reach across historic boundaries and unlock the potential of collaboration across the usual disciplines.” — Jeffrey S. Flier, Dean of the Faculty of Medicine at Harvard University in a lecture delivered at the 2013 Healthcare Innovation Forum

Got something to say? Please send your comments/letters to press@promotivemedia.ca

June/July 2017

QZA-O

The human brain has been called the most complex object in the known universe. It can be astonishingly resilient and pliable, yet remarkably delicate and perplexing. The health of this most vital of organs is a major issue facing modern society – an issue that requires innovative and intricate solutions.

Ontario, Canada is where digital media and game theory intersect with brain health to improve outcomes. It’s also where Artiﬁcial Intelligence like IBM Watson has joined forces with the Ontario Brain Institute and University Health Network in Toronto to take on Parkinson’s disease. There are brilliant minds here, solving complex problems using technology in imaginative ways. Hospitals, research centres, universities, technology incubators, start-ups, scientists and multinationals act as fully functioning neurons ﬁring through synapses within an interconnected network of innovation. If you want to be at the forefront of brain health research and commercialization, join us for a meeting of the minds in Ontario. Visit Ontario at the Canada Place Pavilion, Booth #4415

www.InvestInOntario.com/BIO2017 Paid for by the Government of Ontario

QZA-OIB-002_BioTechnologyFocus_050217.indd 1

2017-05-02 3:32 PM

Keep Growing The BioFlo® 120—easy to use, ﬂexible, dependable The straightforward setup and process control of the BioFlo 120 let you focus on what is important, your work. Designed to seamlessly integrate into your day-to-day operation, the BioFlo 120 bioreactor/fermentor emphasizes simplicity and ease of use, with no sacriﬁce on capability.

> Push button Auto Culture modes for microbial and cell culture applications > Flexible operation with glass or BioBLU® Single-Use Vessels > Scalable expanded working volume range (250 mL–40 L) > Universal connections for digital and analog sensors

131.A1.0144.A.CA-BFO.indd 1

Full page journal ad Biotechnology Focus - BFO

5/4/2017 9:19:16 AM

Biotechnology Focus June/July 2017

Articles inside

Artificial intelligence – the Canadian connection

Anchor companies and ‘sticky capital’

Q&A with Brian Bolzon of Versant Ventures on the risks and rewards of launching biotech startups

CCAB: Translating early-stage biologics into your products