Vertex’s CRISPR Gene Therapy Lands Another FDA Nod in a Rare Blood Disease

MedCity News January 16, 2024
Frank Vinluan

The FDA approved Vertex Pharmaceuticals’ gene therapy Casgevy for treating beta thalassemia, an inherited blood disease that leads to low levels of functioning hemoglobin. Last month, the one-time treatment won its first FDA nod for treating sickle cell disease.

A one-time CRISPR-based gene therapy developed by Vertex Pharmaceuticals and CRISPR Therapeutics now has an additional FDA approval as a treatment for the rare blood disease beta thalassemia.

The affirmative decision announced Tuesday for the therapy, Casgevy, was widely expected following its December approval in sickle cell disease, but it comes more than two months early. The target date for an FDA decision in the beta thalassemia indication was March 30. Casgevy won its first ever regulatory approval in the United...

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