US regulators lift clinical hold on CRISPR Therapeutics, Vertex's sickle-cell disease treatment CTX001

CRISPR Therapeutics and Vertex Pharmaceuticals announced Wednesday that the FDA lifted a clinical hold that was placed on CTX001 in May and accepted their investigational new drug application for the sickle-cell disease treatment. The companies are seeking US approval for CTX001, an autologous gene-edited haematopoietic stem cell therapy, for use in patients suffering from severe haemoglobinopathies. Shares in CRISPR jumped as much as 13.5 percent on the news. 

The drugmakers previously announced that they had obtained approvals for CTX001 clinical trial applications in several countries outside the US for both sickle-cell disease and β-thalassemia. They indicated that they remain on pace to start a Phase I/II study of CTX001 for the treatment of sickle-cell disease by the end of the year. Additionally, the companies said they have initiated enrolment of patients with transfusion-dependent β-thalassemia in a Phase I/II β-thalassemia trial in Europe. 

CRISPR and Vertex entered into a partnership in 2015 to use the former's CRISPR/Cas9 gene editing platform to discover and develop treatments targeting the underlying causes of disease. Under the deal, Vertex has the rights to licence up to six new treatments developed with the CRISPR/Cas9 gene-editing technology. The drugmakers agreed last December to jointly develop and commercialise CTX001 for sickle-cell disease and β-thalassemia as part of their existing collaboration. 

For related analysis, see ViewPoints: CRISPR/Cas9 finally off to the races, with zinc fingers waiting in the wings.

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