US FDA pauses CRISPR gene-edited therapy trial for sickle cell disease

By Flora Southey

- Last updated on GMT

The US FDA has placed CRISPR Technologies and Vertex Pharmaceuticals’ investigational stem cell treatment on clinical hold.

The US Food and Drug Administration (FDA) placed the autologous hematopoietic stem cell therapy on clinical hold after receiving CRISPR and Vertex’s investigational new drug (IND) application in April.   

The Phase I/II trial for CTX001 will not begin until certain regulatory questions are resolved, said the firms in a statement​: “CRISPR and Vertex expect to obtain additional information on the FDA’s questions in the near future and plan to work rapidly with the FDA toward a resolution.”

CTX001 – an investigational ex-vivo ​CRISPR gene-edited therapy – is the product of a co-development and co-commercialisation deal between CRISPR and Vertex.

If approved, the firms would equally share all R&D costs, and profits, worldwide.

CRISPR and Vertex said they don’t expect the FDA hold to affect the CTX001 Phase I/II trail in Europe, due to begin in the second half of 2018.

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