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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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The International Council for Harmonisation has identified four new topics that can benefit from global regulatory alignment, with timelines for initiating work to be determined later.

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The first two classes of negotiated drugs under the Inflation Reduction Act would not benefit from a rare disease adjustment House Republicans included in their reconciliation package.

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Industry is concerned that Prasad may make regulatory flexibility tougher to obtain for cell and gene therapy, while vaccine and public health advocates are angry about Prasad’s criticisms of US COVID-19 policies.

More from Advanced Technologies

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• By Eliza Slawther

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EU Wants Industry To Define ‘Trade Secrets’ Under Health Data Sharing Regulation

 
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