FDA approves CRISPR-based therapy for sickle cell disease

AXIOS December 8, 2023
Adriel Bettelheim

The Food and Drug Administration on Friday approved the first therapy based on CRISPR gene-editing technology to address the painful effects of sickle cell disease.

Why it matters: The technology could be applied beyond the estimated 100,000 Americans with sickle cell to other blood disorders like hemophilia, as well as certain cancers and infectious diseases.

• But gene therapies are costly, with million-dollar price tags for some products, presenting huge challenges to employers, public health programs and other payers throughout the health system.
• Morgan Stanley earlier this year projected the new sickle cell treatment could generate $19 billion in sales over the next decade.

Driving the news: The FDA approved Casgevy from Vertex Pharmaceuticals and CRISPR Therapeutics, a one-time...

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