STAT+: Vertex, CRISPR Therapeutics inch ahead of rival for genetic fix to sickle cell disease
Vertex Pharmaceuticals and its partner CRISPR Therapeutics said they had met their previously announced goal of filing by the end of March a marketing application for their therapy known as…
The first CRISPR treatment for sickle cell disease has been submitted to the Food and Drug Administration, beating a rival gene therapy that’s been delayed due to a manufacturing issue.
On Monday, Vertex Pharmaceuticals and its partner CRISPR Therapeutics said they had met their previously announced goal of filing by the end of March a marketing application for their therapy known as exa-cel. In addition to sickle cell disease, the two companies are seeking approval to treat beta-thalassemia, another type of inherited blood disease, but one that’s rarer in the U.S.
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