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Changing the DNA of medicine with the world’s first CRISPR therapy

Friday was a monumental day for those living with sickle cell and those pursuing cell and gene therapy solutions for various diseases.

FDA FDA Approved

Regulatory Review: Unsung FDA approvals for rare disease in the 3rd quarter

In Q3 2022, several rare disease therapies became the first approved for their indications. Read more about under-the-radar FDA approvals for rare disease.

FDA FDA Approved Rare Disease