FDA to Use New Review Tool on Sarepta’s Gene Therapy Work

FDA Hosts Cell and Gene Therapy Roundtable

The Food and Drug Administration’s Center for Biologics Evaluation and Research (CBER) hosts a roundtable on cell and gene therapy from 9 a.m. to 12 p.m. on June 5. Roundtable Participants: Jeff Allen, PhD, President and CEO Friends of Cancer Research Ron Bartek, Co-founder and President Friedreich’s Ataxia Research Alliance Allyson Berent, DVM, DACVIM, Chief Scientific Officer Foundation for Angelman Syndrome Therapeutics Co-Director Angelman S…

FDA to use new review tool on Sarepta’s gene therapy work

The company is one of the first to receive a “platform technology designation,” which could speed the review of certain gene therapy applications it later brings to the regulator.

FDA Grants Platform Technology Tag to Sarepta's Gene Therapy Vector

FDA Grants Sarepta Therapeutics Platform Technology Designation to Expedite Gene Therapy Reviews

The U.S. Food and Drug Administration (FDA) has granted […] The post FDA Grants Sarepta Therapeutics Platform Technology Designation to Expedite Gene Therapy Reviews first appeared on GeneOnline News. The post FDA Grants Sarepta Therapeutics Platform Technology Designation to Expedite Gene Therapy Reviews appeared first on GeneOnline News.

In a first, Sarepta announces platform technology designation for gene therapy vector

Sarepta Therapeutics said Wednesday it won “platform technology” status for one of its gene therapy components, marking the first public disclosure from a company to receive the long-awaited FDA designation. ...

#U.S. FDA Grants Platform Technology Designation to Sarepta Therapeutics Viral Vector for Investigational Gene Therap...

CAMBRIDGE, Mass. Sarepta Therapeutics, Inc. (NASDAQ: SRPT), renowned for its pioneering work in precision genetic medicine for rare diseases, recently announced that the rAAVrh74 viral vector utilized in its investigational gene therapy SRP-9003 (bidridistrogene xeboparvovec) has received a platform technology designation from the U.S. Food and Drug Administration (FDA). This designation marks a significant milestone, positioning Sarepta at the …