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One Big Step Away From BLA Submission for Sangamo’s Fabry Gene Therapy

The EC’s decision comes several weeks after the FDA approved cilta-cel for a similar expanded indication.

FDA Issues CRL for Abeona's Epidermolysis Bullosa Gene Therapy Pz-Cel

The FDA is allowing a small, single-arm study to support a BLA submission of ST-290, if Sangamo can find help to continue developing the therapy.

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The FDA is requesting more CMC information, specifically with regard to validation requirements for specific manufacturing and release testing methods.

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