Gene Therapy is Fighting Rare Diseases in FDA, NIH Program

Gene Therapy is Fighting Rare Diseases in FDA, NIH Program

Season: 2
| Episode: 39
The technique aims to treat some diseases by gene replacement.
Peter Marks, Director, CBER, FDA
P.J. Brooks, Deputy Director, Office of Rare Diseases Research, NCATS, NIH

This past fall, the National Institutes of Health, the Food and Drug Administration and other partners launched the Bespoke Gene Therapy Consortium in an effort to leverage adeno-associated viruses as vectors to treat rare diseases. The initiative seeks to streamline research to clinical use, driving efficiencies into the traditional research and development process. FDA leader Peter Marks and NIH's P.J. Brooks discuss the consortium's work and how it aims to help people with rare diseases.

 
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