By: Shyam Patel
Over 1200 business and science leaders in regenerative medicine descended upon an uncharacteristically cloudy San Diego last week for the annual Cell and Gene Meeting on the Mesa. Reflecting the general growth and enthusiasm in the industry the meeting attracts so many attendees now that it is forced to move to a larger venue next year.
According to meeting organizer, the Alliance for Regenerative Medicine (ARM), there are currently 892 companies and 1000 clinical trials in regenerative medicine worldwide. To date, the regenerative medicine industry has raised a staggering $10.3B in financing, and IPOs and venture capital raises this year are already much higher than previous year totals. These, of course, are good problems to have.
The industry is still riding the euphoria generated by recent US market launches of the first gene modified cell therapies, Kymriah and Yescarta, and the first gene therapy, Luxturna. Both chimeric antigen receptor (CAR) T cell therapies and gene therapies were the stars of the meeting with almost all the panels featuring companies in these two areas. The successful launches of these products, along with the Food and Drug Administration’s commitment to RMAT (Regenerative Medicine Advanced Therapy, a new designation that can mean accelerated review for a treatment) and the aforementioned cash infusion, have reduced the industry’s anxiety around regulatory and financing risks.
Now the focus is on reimbursement and adoption strategies. The big challenge for the industry will be proving the value of these one-time potentially curative but expensive therapies to the healthcare system. These new ways to treat and cure diseases will need new ways to pay for them.
As in previous years CIRM-funded projects were very well represented at the meeting. Company presentations from CIRM grantees such as Caladrius, Capricor, Cellerant, Nohla Therapeutics, Regenerative Patch Technologies, Sangamo and ViaCyte showcased the breadth of regenerative medicine research in California. In addition, the science research talks by CIRM-funded investigators Helen Blau, Stephanie Cherqui, Matt Porteus and Mark Tuszynski, which ranged from muscle rejuvenation compounds to CRISPR’ing sickle cell disease, made it clear that the next wave of technologies is already on the horizon.
All of these presenters noted that CIRM funding was the crucial driver in progressing the projects out of the laboratory and into the clinic.
In the end, the 1500 partnering meetings that took place over two days were all focused on a singular goal: delivering revolutionary therapies as quickly as possible to patients in need.