FDA Offers Gene Therapy Advice on Hemophilia, Other Diseases

In new draft guidance documents released on July 11, the Food & Drug Administration (FDA) suggested the best ways gene therapy developers can get products to treat hemophilia, retinal disorders and rare diseases approved to go to market.

Partner Michael Werner told Bloomberg Law that the FDA's move reinforces its sustained support of the technology.

"It's another very clear statement from the agency, from the highest levels of the agency, that expresses support for gene therapy," said Mr. Werner, who is co-leader of Holland & Knight's Healthcare & Life Sciences group and co-founder and senior policy counsel of Alliance for Regenerative Medicine. "It acknowledges the role that gene therapy technologies can play and will play in the treatment of diseases that many of which currently have no treatment, other than perhaps palliative care."

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