Thoughts on FDA’s Gene Expressions


The FDA issued six guidance documents yesterday addressing gene therapy—three on specific diseases and three on manufacturing—nearly a year after approving the first, Novartis AG’s Kymriah treatment for lymphoblastic leukemia. It reflects the agency’s commitment to developing a subset of regenerative medicine that could provide treatment breakthroughs, Alliance for Regenerative Medicine co-founder and senior policy counsel Michael Werner told me for my story. He’s also a partner with Holland & Knight LLP.

I also reached out to the American Society of Gene & Cell Therapy. President and Stanford professor of genetics Michele Calos had this to offer in response to my questions:

How important is this in the advancement of gene therapy development?

While it still too early to gauge fully the effect of the six new guidance documents, we expect them to be significant. The FDA has long made known their desire to promote the innovative science embodied in gene therapy, while ensuring safety and long-term viability of these treatments—all of which is in line with our goals at ASGCT. We’re pleased that the FDA is creating tools to expedite the development of gene therapies, and we’re working to make sure that these efforts will also result in increased access for patients. 

In general, has the FDA been getting things right in its regulatory approach? Either way, why do you think that?

The FDA has largely been supportive towards gene and cell therapy. Because gene and cell therapies offer tremendous potential for effective, long-term treatments for so many diseases, the FDA has been helpful in facilitating development of the work. There’s a shared understanding between the FDA, ASGCT, and the gene therapy community of the tremendous benefit gene and cell therapies can offer to millions of people worldwide.

What do you see are the biggest areas of concern in the regulation of gene therapy?

We have to establish safety and efficacy for each and every treatment, and that’s handled on an individual basis with each therapy. Moreover, we must maximize access to gene and cell therapies for those who can benefit from them. Novel gene and cell therapies will be of little use if they’re unavailable to the patients who need them.

What do you think is next in the area of gene therapy development (or want to see next)?

After decades of careful research, gene and cell therapies are now moving from the bench to the clinic at an accelerated pace. A number of these treatments are covered in the latest FDA guidance.  Our hope is that these FDA documents will make gene and cell therapies more valuable for patients, by offering guidance to help validate their safety and long-term benefits.

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