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AAV Vector Consortia Address Quality Assays, Endpoints

 
• By Cole Werble

Two outside groups aim to help CBER on gene therapy questions from ‘N of one’ to markers for hemophilia.

gene therapy collaboration
• Source: Alamy

After more than two years in the making, two outside gene therapy consortia are underway in the US to help broaden the consensus starting point on discussions with CBER on characterization assays and the appropriate use of surrogate endpoints.

The recent formal introductions of two AAV gene therapy consortia permit FDA’s Center for Biologics Evaluation and Research to consolidate conversations with the emerging gene therapy sector on several key...

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