Regenerative medicine. Harnessing the power of stem cells. Genetic modifications. Replacing unhealthy tissues and organs to cure devastating diseases. Though it may all sound “space age,” this remarkable technology is very much present and thriving, promising innovative and groundbreaking therapies that may one day eliminate illnesses such as diabetes, heart disease and cancer.
Canada, which ranks second in cost competitiveness for biomedical research and development, is home to CCRM, a not-for-profit organization funded by the Government of Canada, the Province of Ontario and numerous academic and industry partners. Founded in 2011, CCRM turns scientific discoveries into new companies and marketable products to help patients via specialized teams, funding, and infrastructure with pioneering researchers, leading organizations, strategic investors and entrepreneurs.
CCRM proudly specializes in developing and commercializing regenerative medicines — including cell and gene therapies —and bringing those new products to market. Boasting a team of expert engineers and scientists with more than 300 combined years of cell and gene therapy experience, CCRM’s customer-focused approach is customized to benefit individual clients, while providing scientific and business services for a variety of partners in the regenerative medicine field.
“Regenerative medicine includes what we call cell and gene therapy and tissue engineering. It’s really a combination of advanced therapies built around cells as products, and the promise of developing cures for diseases, not just being able to treat the symptoms,” says CCRM President and Chief Executive Officer Dr. Michael May. “When we talk about cells as therapy, everyone talks about stem cells, which are a very special type of cell that can be transformed into any type of tissue in the body. Regenerative medicine is really founded around this ability to control biology and special cells like stem cells.”
As one of the “hottest” sectors in biotechnology with record levels of investment, it’s not just a futuristic field anymore, he says. There are numerous products being approved and coming to market, including Spark Therapeutics’ Luxturna — a treatment for inherited blindness — and Kite Pharma’s Tecartus and Yescarta, both CAR-T cell therapies designed to combat lymphomas.
“These are cancer treatments where you take the white blood cells from a cancer patient and then engineer them outside the body to recognize the patient’s tumour cells,” says Dr. May. “They’re then grown up and re-infused back into the patient. For certain types of cancers, they’re getting 90 percent cure rates. That’s a great example of cell and gene therapy which is transforming medicine.”
While they sound life-changing, many of these cell and gene therapies and regenerative medicine-based treatments don’t reach patients in need due to commercialization issues. Complex science, risky clinical trials and expensive manufacturing, along with a lack of early-stage investment, all impede the progress from creation to commercialization.
CCRM is hoping to ease that process from its 40,000 square-foot Toronto-based facility in the MaRS Discovery District, located alongside world-class research hospitals and the University of Toronto. In 2019, the global market for regenerative medicine was valued at US$13.3 billion, forecasted to grow to US$73.4 billion by 2027, with approximately 100,000 patients to be treated with CAR-T immunotherapies by 2021.
“CCRM was established to accelerate the development of these types of products on the back of Canada being a leader in research, development and discovery in the field of regenerative medicine,” says Dr. May. “CCRM can accelerate or generate both health and economic benefits from that leadership in the space.”
A public-private partnership, CCRM works with researchers and academic institutions to access, assess and support the development of their intellectual property and inventions, says Dr. May.
“Industry provides the market pull to balance the technology push from academia, while industry is also the receptor for innovations.” Engagement with investors fuels development of an industry like this, he adds.
CCRM looks to do three things: first, find exciting early-stage technologies, de-risk them and bundle them together, then advance them with their inventors so they’re more viable and suitable to build sustainable Canadian companies around.
“The second thing we do is provide gap-filling contract services to academia and industry (e.g., process development and GMP manufacturing). Offering these services builds our own capabilities, which supports better and capital-efficient company creation, as well as CCRM’s reputation as a go-to destination for opportunity development,” Dr. May explains.
“The third thing CCRM does is to act as a seed investor in these opportunities to catalyze additional risk capital and grow companies faster,” he says. “That investment also gives CCRM a small piece of the pie, and that’s really key to our sustainability model.”
Although the organization receives funding from the government, it doesn’t want to depend on any one source of funding in the future. In fact, support has evolved over the years from 90 percent government funding to about 10 percent.
“CCRM has put a lot of focus in manufacturing and manufacturing capability,” says Dr. May. “If we can catalyze investment in companies domestically, and we’re masters of the manufacturing, the companies we create will stay in Canada.” Without this basic support, intellectual property and emerging companies will go elsewhere. CCRM has shown that by building manufacturing capacity, not only are companies staying in Canada, but more companies are being attracted to Canada.
The investment piece of the thesis is one that’s evolving, he says. In 2016, Cytiva (formerly GE Healthcare Life Sciences) and the Canadian government each contributed $20 million for CCRM to construct a fully-resourced, 10,000 square-foot facility to provide advanced manufacturing and process development for therapy developers. The Centre for Advanced Therapeutic Cell Technologies (CATCT) assists developers with industrial-scale manufacturing workflows and creates new technologies to help solve technical challenges. In 2019 CCRM opened, in partnership with University Health Network, a 20,000 square-foot Good Manufacturing Practices (GMP) facility, called the Centre for Cell and Vector Production (CCVP). CCVP provides quality assurance and quality control processes, clean rooms, and highly skilled personnel to companies and academics to manufacture cells and viral vectors for Phase I and II clinical trials.
“From day one, CCRM and its founders were insightful about the gaps and bottlenecks in manufacturing in cell and gene therapy, understanding that if we didn’t tackle those bottlenecks, the industry wouldn’t advance quickly and we wouldn’t take a leadership position in it,” says Dr. May.
Because many of these technologies develop in academia, they’re often small-scale, he says. CATCT brings them to a more industrial scale, using state-of-the-art know-how and equipment. A team of about 30 people at CATCT have completed dozens of projects with therapeutics companies, and worked with Cytiva and other enabling technology companies to develop cell manufacturing platforms that will support and advance the industry globally.
“CCVP is a facility where cells are manufactured for use in human clinical trials, the next stage being commercial-scale manufacturing,” Dr. May says. “CCVP is also a testing ground for the manufacturing processes and technologies that we’re developing in the CATCT, which is a very important part of our manufacturing strategy.”
By partnering with leading research institutions to launch new ventures, enabling industry by providing innovative contract development and manufacturing (CDMO) services, and scaling emerging companies by catalyzing investment, CCRM is converting promising technologies, processes and therapies into life-changing futures for patients.
CCRM is currently working with McMaster Innovation Park, investors and strategic partners to establish Canada’s first commercial-scale manufacturing site in Southern Ontario. Measuring hundreds of thousands of square feet, this industrial-scale factory will produce therapeutic cells, not just for clinical trials but for sale and distribution.
“There’s a strategy – develop foundational manufacturing processes and technologies, seamlessly connected to a clinical phase facility, both ultimately enabling commercial-scale production – that we hope to realize over the next three years in Southern Ontario,” Dr. May says.
Indeed, manufacturing infrastructure and capability is putting Canada on the map in that area. CCRM has also helped support and create a pipeline of companies that have raised hundreds of millions of dollars.
“All cell products in the future are going to be genetically modified,” says Dr. May. “The field isn’t just about making the cells; it’s about modifying the cells, and it’s also about making the tools to modify the cells. We also focus on the viral vectors used to modify cells.”
These academic, industry and investor networks, wrapped around a specially trained team with specialized funding to drive things forward in a collaborative, capital-efficient way, is creating companies that will be sustainable in Canada with growth for global markets.
“When we think about advanced manufacturing in this space, it’s not about building four walls and putting a sign on the front door inviting foreign companies to come to Ontario. We need to build an ecosystem, i.e., the therapeutics companies that will be customers and the logistics and supply chain companies that will be needed for support.” CCRM sees itself as an ecosystem builder and that means tackling many of the bottlenecks that must be addressed to make cell and gene therapy a viable industry, says Dr. May. For example, in addition to supporting manufacturing and company creation, the CCRM team has expertise in regulatory affairs and intellectual property protection.
“We believe we have an opportunity here to be global leaders, and have been working with academics, industry and investors around the world to start thinking about building a global network based on our Canadian model,” he says. “Canada can be the nexus of a global network of commercialization hubs, which will help scale our activities and solidify our leadership. The global network will attract talent, bundle IP and facilitate access to global markets for the companies created with the help of CCRM.”
With a 10-year plan to build relationships across the globe, CCRM is already well on its way to establishing itself as a world leader in therapies that can truly change the lives of people around the world suffering from a variety of deadly diseases.
The cost of cell and gene therapies can be prohibitive, but these are considered one-time cures. If you look at treatments for diseases that are chronic and require a patient to pay thousands or sometimes hundreds of thousands of dollars every year, the price tags don’t look quite as daunting by comparison. Can they be called cures, however? The people behind them want them to be cures, but companies won’t know until patients have been alive long enough to assess the durability of the treatments. These new therapies will revolutionize healthcare.
“There are very few organizations like ours in the world, public-private partnerships that are focused on this ecosystem building,” says Dr. May. “What sets us apart is our collaborative, capital-efficient mindset. Canadians are trusted around the world and have demonstrated that we can ‘punch above our weight.’ Doing more with less – that sets us apart from any organization like us in the world.”