F.D.A. Approves Sickle Cell Treatments, Including One That Uses CRISPR

The Food and Drug Administration (FDA) approved the first-ever gene editing therapy for sickle cell disease

Additionally, the FDA approved a second treatment using conventional gene therapy for sickle cell, not involving gene editing.

The approvals provide hope for the 100,000 Americans, mostly Black, suffering from sickle cell disease.

The disease causes excruciating pain, organ damage, and strokes.

Despite the positive development, accessing either therapy poses challenges, including cost and limited availability.

Vertex sets the price for editing a patient’s genes at $2.2 million, while Bluebird's therapy is priced at $3.1 million.

The high cost raises concerns about accessibility for a broader population.

– Living with sickle cell disease is financially burdensome, averaging $1.7 million for those with commercial insurance over a patient’s lifetime.

Medical centers are compiling lists of interested patients anticipating treatment availability.