The latest installment of our Regenerative Medicine panel is a short exert related the regulatory pathway for cell and gene therapies. Below is a condensed and edited version of Part 4 of a panel discussion moderated by Kevin Hampton, Chief Commercial Officer at Kineticos.


  • Susan Nichols – CEO, Falcon Therapeutics
  • Flagg Flanagan – CEO & Chairman, DiscGenics 
  • Bob Hutchens – CEO, StemBioSys

KH: Transitioning to the regulatory component, shortly after Gottlieb resigned, he put out a statement regarding the need for our industry to modernize and do a better job of finding creative ways to advance therapies. I am curious to hear if each of you feel the FDA is being more reactive or proactive in the Regenerative Medicine space? I believe Gottlieb did a great job of creating an enabler mentality across the agency but do those themes applies here?

Susan: The FDA has had to play catch up in the cell-modified gene therapy space. They are anticipating 10-20 approvals each year by 2020. It’s reactive in that the influx is causing a backup. It is proactive in ways, too. Knowing cell and gene therapy is accelerating, the FDA created 50 new clinical review positions to support clearing the backlog as well as continued growth. Another way the FDA has been proactive is steadily releasing draft guidance which has been extremely helpful. This allows companies like Falcon to be proactive and anticipate.

Flagg: Dr. Gottlieb was really a pioneer for the FDA. He placed an emphasis on holding pre-IND meetings to inform everyone of the ground rules and expectations. It is very positive. Having a clear set of regulatory guidelines helps all players in the gene and cell therapy space while, importantly, protecting the public. Once this industry sees 3 or 4 major successes, meaning approvals and reimbursement for cell and gene therapy products, it will open up a lot of doors for all of us as developers, and for patients as well.

Susan: Flagg, don’t you think that Dr. Gottlieb really changed the thinking from ‘Here is what we need you to do’ to ‘How can we help you do this and get it to patients?’.

Flagg: Dr. Gottlieb did a great job of ensuring that providers and drug developers understand exactly what the expectations are. Hopefully, Dr. Sharpless continues that and every indication I’ve seen is that he will continue down that path. The FDA is much less of a black box than it used to be. We just have to get some successes.

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