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Gene Therapy Consortium to Launch Rare Diseases Pilot

Gene Therapy Consortium to Launch Rare Diseases Pilot
The FDA will soon announce a new pilot program to help developers of gene therapies for rare diseases get their treatments approved faster, said Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research (CBER), at the agency’s Rare Diseases Day 2023 virtual public meeting on Monday.

Marks said the concept for the pilot, which will be run through the 16-month-old Bespoke Gene Therapy Consortium, was born out of the way the FDA worked with drug manufacturers during the lockdown and meetings chaos of COVID-19.

“During the pandemic, we had very constant contact with many of the manufacturers where we essentially threw out the way we do meetings and had a constant back and forth on almost a day-to-day basis when issues arose,” said Marks. He said CBER wants to employ a similar workflow for select gene therapies that hold particular promise for treating rare diseases to move them forward more quickly.

Marks said the FDA will announce the new pilot program soon via the Federal Register.

March 1, 2023

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