AAV Vector Consortia Address Quality Assays, Endpoints

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.

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