Momentum has been building within the regenerative medicine industry with cell therapies and other technologies moving closer to commercialization every day. With regulatory changes hastening progress in Japan and research advances happening elsewhere in the Asia-Pacific region, one Canadian company is looking overseas for opportunities to create jobs and companies here at home, while always being mindful of the patient.
Accelerating cell therapy and regenerative medicine commercialization drives CCRM, a federally funded Centre of Excellence for Commercialization and Research based in Toronto. Through its unique public-private partnership, CCRM’s mission is to capitalize on the commercial potential and curative promise of cell therapies and regenerative medicine technologies.
Five years since its launch, CCRM’s focus has become more global and its relationships with countries, such as Australia, Singapore, Taiwan, Japan and China, are becoming more important as the organization sources technologies from around the world to advance cell therapies to all markets. The growth has been substantial. To put this in perspective: in 2014, the Asia-Pacific stem cell market was valued at US$7.10 billion and is projected to increase to US$18.71 billion by 2018 at a CAGR of 27.3 per cent.1 Globally, the stem cell therapy market is expected to be worth US$40 billion by 2020 and US$180 billion by 2030.1
Canada has a long history with stem cells. Scientists James Till and Ernest McCulloch first discovered stem cells over 50 years ago in Toronto. Ever since, Canadian researchers have remained at the forefront of regenerative medicine research. For example, cancer stem cells and retinal stem cells were discovered here. Enhancing this legacy of scientific excellence and discovery is the collaborative attitude that exists between industry, academia and government.
CCRM’s commercialization model combines network building – CCRM has built consortia of academic, industry and investment partners from the global community – unique facilities operated by staff dedicated solely to translation/commercialization, and targeted funding to bundle, de-risk, protect and leverage discoveries into licensable technologies or new companies. Moreover, CCRM’s collaborative and capital-efficient model integrates government support at all levels.
CCRM’s business development team conducts due diligence on academic discoveries and its scientists and engineers perform wet diligence, fee-for-service projects and product development in a 6,000 sq. ft. development facility provided by the University of Toronto. By the end of 2016, CCRM will move into 40,000 sq. ft. of new office and lab space at MaRS’ West Tower in the heart of Toronto’s discovery district. The jewel in the space will be a state-of-the-art centre for accelerating advanced manufacturing solutions for cell therapies, in partnership with GE Healthcare, with a sizeable investment from the federal government. To support the pipeline, CCRM and University Health Network will operate a Good Manufacturing Practices facility, backed by provincial funding.
As well, CCRM has a fee-paying industry consortium of more than 45 Canadian and international companies – who range from start-ups to multi-nationals – and represent the key sectors of the regenerative medicine field: therapeutics, devices, reagents, and cells as tools.
“There are compelling reasons for Canadian companies to be looking to Asian markets right now,” says Lee Buckler, president and CEO of RepliCel Life Sciences Inc. (TSX.V:RP). RepliCel is a Vancouver-based company, which focuses on developing autologous cell therapies to treat conditions that result from a lack of healthy cells in the body. The company is a member of CCRM’s industry consortium, but has also partnered with Japanese company Shiseido to treat pattern baldness.
“RepliCel is concentrating on strategic initiatives in Japan because of its existing partnership with Shiseido Company, Japan’s national commitment to regenerative medicine, and the opportunity for expedited market approval now available there for cell therapies,” he says.
“The knowledge we have gained from working with our industry consortium members has been invaluable to us,” explains Michael May, president & CEO of CCRM. “This is how we learn what the market actually wants and needs. By listening to academia, industry and investors in Asia, we will not only source novel technologies from diverse locations, but develop revolutionary products for patients in large, untapped markets,” he concludes.
A relationship-driven business strategy requires a large investment of time and travel. Not only does CCRM staff travel to Asia often for meetings with potential partners – the Agency for Science, Technology and Research (A*STAR) in Singapore being just one example – they are regular speakers at prominent conferences, like the 3rd Cell Culture & Bioprocessing Asia Congress in Singapore last October and the International Society for Cellular Therapy’s (ISCT) annual meeting this May. These trips – not an exhaustive list – serve as forums to present information and strengthen relationships with the local biotech communities
Successfully bridging differences in the academic and industry cultures are essential to the CCRM model. To explain, academia is curiosity-driven, whereas industry is milestone driven. When the powers of both are harnessed, it’s possible to unlock new ways to commercialize cell therapies. As a not-for-profit funded by government, industry, institutions and investors, CCRM is an objective, neutral vehicle for advancing the regenerative medicine industry. CCRM recognizes, however, that it is difficult to achieve “critical mass” in a single institution, or even a single country.
As a consequence, CCRM is establishing active hubs around the world to scale its IP sourcing, industry consortium and company creation to sustainable levels. Australia and Asia, and specifically Japan, are key targets for this activity because of the quality of the research, expertise and resources that exist. CCRM has already signed memorandums of understanding with partners on both continents to promote IP sharing and other initiatives.
Of course, it takes two to make relationship building successful and beneficial to all parties. This is why CCRM also supports Asia-Pacific companies looking to establish a foothold in North America.
“As a leading cell therapy product development company in Taiwan, working with CCRM has provided us with valuable resources and consortium contacts to support our initiatives to enter the North American market,” says Bill Milligan, SVP Corporate Development & International Business Development, Steminent.
The scientific expertise that exists in Canada, combined with its manufacturing experience, collaborative environment and favourable regulatory system make it an ideal location for international biotech companies to invest.
Ryan Chang, CEO, Steminent agrees: “Our relationship with CCRM has been beneficial, specifically for our manufacturing, clinical advisory and regulatory strategies, as we focus on bridging our lead Phase 2 clinical project in Taiwan toward regulatory approvals and sites for clinical development in North America.”
An essential tactic of CCRM’s business strategy is IP bundling and the company has developed several best practices around this strategy. IP bundling is the custom of taking intellectual property (IP) from multiple institutions and/or researchers and strategically combining them to create a marketable product or a fundable company. In June 2015, CCRM launched its first spin-off company, ExCellThera, which was built using this strategy.
ExCellThera offers an innovative umbilical cord expansion platform for the treatment of blood cancers and other disorders. This is the first of several companies that CCRM has launched based on the formation of technology bundles.
Another important relationship for CCRM is a partnership between Ontario stem cell researchers and their counterparts at Peking University in China. As the commercialization partner of the Ontario Institute for Regenerative Medicine (OIRM), CCRM is actively invested in this relationship and is working with OIRM and its Chinese partners to advance IP bundling and company creation.
Michael May and delegates from Ontario have traveled to Guangzhou for meetings with their Chinese hosts. The Chinese representatives were exposed to the strong Canadian research in blood development, expansion and transplantation that was identified as a focus for future collaboration between the two countries. As the partnership continues to evolve, commercialization expertise will become vital and CCRM is ready to facilitate access to North American markets and to gain the same access to Chinese markets in return.
In addition to being the commercialization partner of OIRM, CCRM was also named as the commercialization partner of Medicine by Design (MbD), which brings together leading researchers in regenerative medicine and cell therapy at the University of Toronto and affiliated hospitals to generate paradigm-shifting discoveries and accelerate translation, commercialization and clinical impacts.
Both OIRM and MbD are exciting government-funded initiatives that are funneling a total of $139 million into regenerative medicine and cell therapy research in Ontario to treat degenerative diseases. CCRM is lending its commercialization expertise to these initiatives to move these discoveries through the pipeline and into clinical trials to help patients in Canada and around the world.
None of CCRM’s international relationships would exist without the support of the Department of Foreign Affairs, Trade and Development (DFATD), which has been essential to CCRM’s continued success in collaborating and partnering with organizations outside of Canada. DFATD has provided introductions to companies abroad, set up meetings, hosted events and has been a significant advocate of CCRM and its model. Without their continued support, CCRM would not be making the headway today that it is globally.
All things considered, the industry is progressing well, but it is important to remember that bringing new treatments to market takes time. CCRM and others are working hard to establish global relationships that are a critical component to seeing the industry move forward to realize the potential of cell therapies and regenerative medicine technologies and change the way many diseases are treated – for patients all over the world.
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