Penn Med responds to FDA guidance on gene therapy

Penn Medicine gave feedback this month on recent Food and Drug Administration guidance regarding gene therapy research. 

The guidance advises developers of gene editing therapies to rely more heavily on existing scientific and manufacturing information to reduce repetitive work and speed up the development of new treatments. In a Sept. 1 letter to the FDA’s acting director, Perelman School of Medicine Dean Jonathan Epstein offered support for the proposal.

A request for comment was left with Penn Med. 

Epstein wrote in support of “a clear rigorous framework for leveraging prior knowledge,” and made “modest suggestions” for clarification in two main areas.

He first asked for the document to further clarify how “this final version of the Guidance will impact previous longstanding guidance.”

Epstein suggested that the FDA consider allowing knowledge to be transferred between “appropriately qualified manufacturing facilities” with the goal of increasing the potential to “learn from and incorporate public knowledge.”

The letter also highlights Penn Medicine’s “historic achievements” in gene and cell therapy innovation, including the first FDA-approved CAR T-cell therapy, gene therapy for an inherited form of blindness, and the University’s “critical role” in developing mRNA vaccine technology that “helped transform the response to COVID-19.”

According to the FDA’s website, guidance documents do not establish legally enforceable responsibilities and are therefore not binding. Guidances describe the FDA’s current thinking on a topic and should be viewed only as recommendations, unless specific regulatory or statutory requirements are cited.

The docket states that the comment period ended at the beginning of September, although late comments have been allowed.

Penn’s support comes after Penn Medicine professor Paul Offit’s dismissal from the FDA vaccine advisory committee.

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