CCRM: Cultivating a culture of cooperation to advance the global regenerative medicine industry

Written by CCRM

Launched in June 2011, CCRM is a unique, Canadian, not-for-profit group that is solely focused on developing and commercializing regenerative medicine, cell and gene therapy technologies. Its mission is to generate sustainable health and economic benefits through global collaborations and its vision is to be the preferred destination for the best people and companies, technologies, clinical trials and investments in cell and gene therapies and regenerative medicine.

CCRM has a relatively short history, but the seeds of its establishment date back more than 50 years when two young scientists, at the Ontario Cancer Institute in Toronto, published a paper with findings that would forever change the landscape of medicine — and give rise to regenerative medicine, as we know it today.

Drs. James Till and Ernest McCulloch discovered the first transplantable stem cells in 1961, but it took two years of exhaustive research, systematic testing and collaboration for them to definitively prove that red, white and platelet blood cells were produced by a single stem cell — thus laying the groundwork for bone marrow transplant as a therapy. From that point forward, Drs. Till and McCulloch alternated as lead authors on their papers as a strategy for avoiding arguments “about priority that could well sour their cordial relationship,” according to a statement made by the late Dr. McCulloch.

And it’s that same spirit that guides Michael May, President and CEO, in his work with CCRM. His philosophy is that in a world where there is so much competition and lack of resources, including money, if you’re good at collaborating and partnering, you can drive change faster. “All of a sudden, one plus one equals three. It’s synergy, and it makes all the difference. That’s the future of regenerative medicine and it’s the underlying strategy of how we work at CCRM.”

To that end, the organization has built an industry consortium of nearly 50 companies[i] to realize the incredible promise of regenerative medicine. With a mix of large multinationals and small biotechnology startups, the consortium brings together key sectors of the regenerative medicine community to address real-life bottlenecks and better utilize translational platforms to bring new products and therapies to market. It operates with the understanding that working together is better.

As a centre of excellence receiving funding from both the Government of Canada and the Province of Ontario, CCRM has a clear mandate to create jobs at home and stimulate the Canadian economy. In order to build a successful industry, Dr. May believes he must first create critical mass outward from local strengths.

Towards that goal, CCRM is establishing an international commercialization platform in cell and gene therapies and regenerative medicine. To date, it has secured partnerships with several leading institutions around the world that embrace CCRM’s model of key academics, industry and investor networks supported by expert staff and facilities, and dedicated funding to move scientific discoveries and technologies to market. This is a work in progress that is expected to benefit the global community over time.

Partnerships at home

Right now, CCRM’s ability to promote the success of the broader Canadian research community on the international stage is paying dividends. In 2015, CCRM secured $40 million in new funding — $20 million from the Government of Canada and $20 million from GE Healthcare — for a centre for advanced therapeutic cell technologies (CATCT), dedicated to developing advanced manufacturing solutions in cell therapies.[ii]

The announcement was made in January 2016 and work began shortly after to hire staff with specialized skills, acquire state-of-the-art equipment and attract partners for co-development projects. CATCT will be co-located with a GMP (Good Manufacturing Practice) facility, built in partnership with University Health Network and the Ontario government. The GMP facility will enable cell manufacturing for local academic trials and small and medium-sized enterprises attracted to Canada for its regenerative medicine ecosystem and favourable regulatory environment.

This new centre will help the cell therapy industry overcome critical manufacturing bottlenecks. Only once regenerative medicine technologies and cell and gene therapies can be commercialized will they be adopted widely.

CATCT joins other important initiatives that will change the face of regenerative medicine. For example there is Medicine by Design, a new regenerative medicine research hub at the University of Toronto, funded with a historic $114-million grant by the federal government. Medicine by Design harnesses the expertise at the University of Toronto and its affiliated hospitals at the convergence of physical and life sciences, engineering, mathematics and medicine to undertake transformative research in regenerative medicine and cell therapy. It fosters unique multidisciplinary collaborations and accelerates the use of engineering design principles and quantitative biological modelling to nurture innovative environments where breakthroughs emerge. CCRM is its commercialization partner.[iii]

Peter Zandstra is the CSO of CCRM and a Professor with the Institute of Biomaterials and Biomedical Engineering at the University of Toronto. He is also the inaugural Executive Director of Medicine by Design. He’s excited to have Medicine by Design and CCRM working together. In the past, Dr. Zandstra says, researchers generated cells designed for certain purposes. Now, they are taking cells that exist (or have been generated in vitro) and examining how they can be modified to treat degenerative diseases.

“We’re asking: Can we take advantage of the fact that many of the cells that could do the jobs we want are already inside our bodies — and then develop drugs, molecules or materials to modify them in order to treat specific diseases?” says Dr. Zandstra. That’s where initiatives like Medicine by Design will soon come into play — and organizations like CCRM will ensure that their breakthroughs make it to market.

Another important partnership exists between CCRM and the Ontario Institute for Regenerative Medicine (OIRM). CCRM and the Ontario Stem Cell Initiative lobbied the Ontario government to establish OIRM.[iv] In June 2015, OIRM was awarded $25 million to aid in the translation of stem cell research into curative therapies for major degenerative diseases like cardiac disease, macular degeneration and autoimmune diseases, originating from labs at McMaster University, University of Ottawa, University of Toronto and Western University and affiliated research hospitals. CCRM will support OIRM’s research projects through specialized platforms that focus on advancing manufacturing and industry engagement.

Working together with the Institute for Research in Immunology and Cancer — Commercialization of Research (IRICoR), CCRM has also launched its first spin-off company, ExCellThera.[v] This clinical-stage biotechnology company represents one of the newest ways to expand blood stem cells for therapeutic use in a robust and cost-effective method. It is also one more example of the power of partnerships to advance regenerative medicine-based therapies.

With the launch of new spinoffs like ExCellThera, initiatives like Medicine by Design and the creation of CATCT, CCRM is now beginning to attract interest from investors worldwide.

“Venture capital groups from across North America are coming to Toronto to do deals here,” says Greg Bonfiglio, founder and managing partner of Proteus, LLC, an investment and advisory firm solely focused on regenerative medicine. “CCRM can take a lot of credit for working to put infrastructure in place to leverage Canada’s academic research and its open, cooperative regulatory environment into clinical trials and beyond. That was something unique about the vision that Michael May and Peter Zandstra had when they created CCRM.” Mr. Bonfiglio also serves as chair of CCRM’s Board of Directors.

Dr. Zandstra says he co-founded CCRM with Dr. May because they both felt that the Canadian research community had much to contribute building upon the strong foundation established by Canada’s Stem Cell Network.

CCRM was created to act as an intermediary between academia and industry so both groups would be aware of how they could support each other to address gaps and develop research to make it commercializable.

Looking Ahead

Speaking to Janet Rossant, it’s clear she is excited about the current opportunities for stem cell scientists and the progress, not far in the future, that she envisions for patients and their families.

Dr. Rossant is a recognized leader in developmental biology. She’s a senior scientist in the Developmental and Stem Cell Biology Program at The Hospital for Sick Children Research Institute in Toronto and the former Chief of Research. Dr. Rossant is also the inaugural President of OIRM and served as its first Scientific Director, an organization she co-founded. Named last year’s 2015 Canada Gairdner Wightman Awardee — and the first woman to achieve this — Dr. Rossant now leads the Gairdner Foundation.

Optimistic about her research and the stem cell community around her, Dr. Rossant sees real-world applications of regenerative medicine on the not too distant horizon. “More so than we could even five years ago, we’re starting to see the real promise of stem cells realized through new products that target degenerative disease. We can see the pathway, and although there will be roadblocks along the way, the direction is clear.”

In the next five to 10 years, Dr. Rossant believes that more stem cell related solutions will come to market to combat retinal and cardiac disease, diabetes, Parkinson’s, multiple sclerosis and other neurological and autoimmune diseases.

But regenerative medicine faces a tipping point right now and she feels that keeping up the investment, continuing to recruit bright young minds, supporting funding for clinical trials and finding ways to manufacture widespread solutions is crucial. If all of that happens, regenerative medicine will become a standard part of medicine, according to Dr. Rossant.

Experts believe that the continuing success of regenerative medicine is contingent upon unique acts of collaboration between academia and industry and thoughtful commercialization.

Mr Bonfiglio believes the field of regenerative medicine is moving into a new phase. As he sees it, regenerative medicine is emerging from a phase predominately characterized by academic research into the effective commercialization of those underlying technologies. He points to the fact that we have more clinical activity now than we’ve had at any time in the last 25 years in the field and says that shows we are finally bridging that gap from bench to bedside.

Soon the future of regenerative medicine won’t be defined by breakthroughs, but rather by discovering ways to bring those breakthroughs to markets across the world.

Mr. Bonfiglio used to characterize the research happening in the regenerative medicine field in Ontario as one of the best-kept secrets. Not anymore.

In his view, CCRM helped let the world know what was happening in Toronto and in the regenerative medicine community in Canada. This has put a spotlight on Toronto as an important hub in the development and commercialization of regenerative medicine technologies.

That said, “Canada still needs to develop some of that swagger that Boston, San Francisco and Silicon Valley have,” says Dr. Zandstra. “We shouldn’t be ashamed to say that we have really good science, and we can contribute at the highest levels to the development of technologies which could have a big impact.”

Dr. Rossant would agree. She says that ego, competitive drive and commitment are important for research, but that shouldn’t diminish the importance of collaboration in order to move research forward. In Canada, there is a long tradition of this, going all the way back to Till & McCulloch.

Summary Points:

  • CCRM is a leader in developing and commercializing regenerative medicine technologies and cell and gene therapies.
  • The company’s mission is to generate sustainable health and economic benefits through global collaboration in cell and gene therapy, and regenerative medicine.
  • CCRM’s scientific advisors are internationally recognized regenerative medicine leaders who have a long and distinguished history of groundbreaking discoveries and scientific excellence.
  • The Ontario Institute for Regenerative Medicine and the University of Toronto’s Medicine by Design have sought CCRM’s commercialization expertise to support the needs of their funded academics and disease teams.
  • In June 2015, CCRM launched its first company, ExCellThera. It now has three companies in its investment portfolio.
  • CCRM has built an industry consortium of more than 45 companies and launched more than 10 co-development projects with industry partners to commercialize regenerative medicine and cell therapy technologies.
  • CCRM received CDN $40 million from the Government of Canada and GE Healthcare to develop advanced manufacturing solutions for cell therapies.
  • In 2017, CCRM will open a GMP facility, in partnership with University Health Network.

[i] Industry collaborations are key CCRM strategy to drive commercialization in RM field

[ii] GE Healthcare, FedDev Ontario commit CAD$40M for new CCRM-led centre to solve cell therapy manufacturing challenges

[iii] CCRM takes on commercialization role with Medicine by Design

[iv] Ontario invests in better health and economic opportunity through support for Ontario Institute for Regenerative Medicine

[v] New company will benefit patients undergoing transplants for Leukemias.

Authors: Alanna Evans and Stacey Johnson

Financial and competing interests disclosure:

The authors were both employed by CCRM at the time of this writing. They have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the article apart from those disclosed.

No writing assistance was utilized in the production of this article.