FDA awarded record number of review designations to CRISPR drugs in 2023

In late 2023, Vertex Pharmaceuticals (Nasdaq: VRTX) and CRISPR Therapeutics’ (Nasdaq: CRSP) made history by gaining the first FDA approval for a CRISPR-based drug, Casgevy (exagamglogene autotemcel) for the treatment of sickle cell disease with vaso-occlusive crisis.

This historic achievement coincided with a record number of 14 review designations awarded by the Food and Drug Administration to CRISPR-based therapies in 2023, according to sector analysts firm GlobalData.

Casgevy is the first CRISPR-based drug to receive marketing authorization globally. CRISPR technology rectifies non-functional genes as opposed to replacing or disrupting pathogenic genes. Casgevy is developed to precisely edit the faulty gene in a patient’s bone marrow stem cell, enabling the production of functional hemoglobin. This replaces the previous permanent treatment option of a bone marrow transplant, which carries many risks, including rejection.