FDA lifts clinical hold on CTX001 trial in sickle cell disease

11 October 2018

The US Food and Drug Administration has lifted the clinical hold placed in May this year and accepted the Investigational New Drug application (IND) for CTX001 for the treatment of sickle cell disease (SCD).

CTX001 is an investigational, autologous, gene-edited hematopoietic stem cell therapy for patients suffering from severe hemoglobinopathies, under development by Swiss gene editing start-up CRISPR Therapeutics (Nasdaq: CRSP) and USA-based Vertex Pharmaceuticals (Nasdaq: VRTX).

Shares in CRISPR, for which CTX001 is its lead candidate, jumped 14.14% to $41.82 in after-hours trading while Vertex closed Wednesday down 4.97% at $176.43.

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