thalassemia
Vertex Pharmaceuticals Sues HHS, OIG Over Legality of Fertility Preservation Program
The firm is asking a court to determine that its program to pay for fertility treatments for patients on Casgevy doesn't violate anti-kickback and inducement laws.
Vertex CRISPR Therapy Casgevy Gets Conditional Marketing Authorization in EU
The EC's authorization for the sickle cell and transfusion-dependent beta thalassemia treatment follows marketing approvals in the US and UK.
FDA Approves Second Indication for Vertex Pharmaceuticals, CRISPR Therapeutics' Casgevy
The CRISPR gene-editing therapy is now approved in the US for transfusion-dependent beta thalassemia, in addition to sickle cell disease.
ASH Gene Therapy Highlights in Hemophilia, TDT: Vertex Pharmaceuticals, CSL Behring, and More
Researchers at the meeting reported on the long-term activity of multiple gene therapies, including one drug with 10 years of data.