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Vertex to lead development of CRISPR Therapeutics-partnered gene therapy

Following the amended agreement, Vertex will now lead the global development, manufacturing and commercialisation of the gene therapy CTX001, with support from CRISPR Therapeutics.

In December, Vertex and CRISPR Therapeutics announced new, early-stage data from a total of ten patients treated with the CRISPR/Cas9 gene-editing therapy.

According to the companies, seven patients with transfusion-dependent beta thalassaemia (TDT), including three who have either a severe or b0/b0 genotype, were transfusion independent at the last follow-up.

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