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US FDA CBER Chief Calls For Industry-Academia Collaboration In Developing Treatments For Ultra-Rare Diseases

 
• By Joanne S. Eglovitch

The head of the US FDA’s Center for Biologics Evaluation and Research said that new approaches are needed to facilitate product development for ultra-rare diseases. The FDA also announces the creation of a new CBER Advanced Technologies Team to promote the adoption of innovative manufacturing technologies for cell and gene therapy products.

Genetic Medicine with dna isolated on white.3d rendering
US FDA's Marks: Industry and academia could collaborate to treat the rarest of diseases

The top official at the US Food and Drug Association’s Center for Biologics Evaluation and Research has proposed that the biopharmaceutical industry partner with academia to facilitate the development of vectors for gene therapies for ultra-rare diseases. Although the plan is still in the preliminary phase, the idea is these vectors could be developed by academic investigators and then transferred to manufacturers once they show promise in early clinical trials.

The idea was floated by CBER director Peter Marks at two recent industry meetings, at the Drug Information Association annual meeting in San Diego on 26 June and at the...

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