A dedicated Regenerative Medicine and Advanced Therapies day took place at BioPharm America™ 2015 last week in Boston, which brought together luminaries from the emerging field to discuss pressing issues and new developments.
The promise of regenerative medicine is no longer science fiction. The flow of commercial products over the next three to five years is expected to bring truly transformative treatments and significant clinical benefits to patients. Access to innovation, patient data, KOLs, and funding is critical for a product to get to the commercialization stage. However, in addition to demonstrable efficacy and safety, we need novel business models and a supportive policy environment. With demonstrated value to patients, there also needs to be fair pricing and fair reimbursement, as well as continued cultivation of an open mindset on improved scale-up manufacturing processes that can accommodate advances in science.
The opening panel discussion, “The evolution of the Regen Med ecosystem: What’s required for its growth and sustainability?” was moderated by Dolores Baksh – Innovation Leader, Cell Therapy Technologies, GE Healthcare and featured panelists Chris Gemmiti – Business Development Lead, Wyss Institute for Biologically Inspired Engineering, Harvard University; Jeff Goater – Senior VP, Finance and Business Development, Voyager Therapeutics; Doug Kerr – Senior Director, Corporate Strategy and Portfolio Management, Biogen; Gary M. Sclar – Associate Director, Dana-Farber Cancer Institute.
The panel gave a glimpse of the innovative biotech and regenerative medicine companies in the Boston-Cambridge area, but largely focused on the pressing question of what is needed to make the sector sustainable for the long-term. The answers? Infrastructure, of course, and partnerships. Partnerships are key to the success of this sector. Academia, medical centers, and patient organizations have active roles in partnering with the biopharma industry to turn these treatments into reality.
The current landscape has lots of small, narrowly defined companies. Biogen, for one, sees itself as an aggregator for these new technologies, but does not want to disrupt the culture of these small companies, noting that it is good to “stay hungry.” Since much of the technology is not mature, companies must continue to evaluate how they access innovation “real time.”
The session “Gene and cell therapy” was moderated by Brock Reeve – Executive Director, Harvard Stem Cell Institute and featured panelists Frank Borriello – VP, Head of Search and Evaluation, Global Business Development, Baxalta; Robert Millman – CEO, Semma Therapeutics, and Managing Director, MPM Capital; Stephen Potter – Chief Business Officer, AGTC; and Madhusudan Peshwa – Executive VP, Cellular Therapeutics, MaxCyte.
In particular,the panel discussed overcoming hurdles to commercialization, including manufacturing and payer issues. “We’re all chasing this Holy Grail of one-shot lifelong therapies,” said Baxalta’s Borriello, as quoted in a Boston Globe article on the event, bringing to light the ever-present debate about how one-time therapies would be billed and the overall effect on prescription drug reimbursements.
Despite this and other hurdles, there are still huge investment opportunities in the field, especially for companies in hub locations like Boston.
Another panel, “Genome editing,” looked at progress in the field and application of these advances, such as CRISPR, TALENS and ZFNs, as well as near and long-term expectations. The panel was moderated by Doug Kerr – Senior Director, Corporate Strategy and Portfolio Management, Biogen with panelists Daniel G. Anderson – Associate Professor, Department of Chemical Engineering, Institute for Medical Engineering and Science, Harvard-MIT Division of Health Sciences and Technology, David H. Koch Institute for Integrative Cancer Research, MIT; Alexandra Glucksmann – Chief Operating Officer, Editas Medicine; Edward Lanphier – President and CEO, Sangamo BioSciences; and Sven Ante (Bill) Lundberg – Chief Scientific Officer, CRISPR Therapeutics. All agreed that the field is moving forward and that progress is being made, but there is a lot of work yet ahead.
“It’s beautiful biology, but it’s still basic science,” said Glucksmann of Editas Medicine as quoted in the Boston Globe article. “We’re not there yet with the technology or the delivery.”