Gene therapy leaders in Rome flag FDA regulatory shifts as key challenge

Gene therapy manufacturers gathered in Rome to discuss the sector’s outlook in Europe, with the U.S. Food and Drug Administration’s recent actions dominating conversation.

Speakers highlighted recent approvals of rare-disease treatments from companies such as Rocket Pharmaceuticals and Regeneron Pharmaceuticals as signs of progress for the field.

The departure of Vinay Prasad, the FDA’s top regulator for gene and cell therapies and vaccines, was also noted, underscoring ongoing uncertainty about future regulatory pathways.