VRTX - FDA Approves First Gene-Editing Cell Therapy For Sickle Cell Disease An Inherited Blood Disorder | Benzinga
The FDA has approved Casgevy and Lyfgenia, representing the first cell-based gene therapies for sickle cell disease in patients 12 years and older.
Both products are made from the patient's blood stem cells, which are modified and given back as a one-time, single-dose infusion as part of a hematopoietic (blood) stem cell transplant.
Before treatment, a patient's stem cells are collected. Then, the patient must undergo myeloablative conditioning (high-dose chemotherapy) to remove cells from the bone marrow and replace them with the modified cells.
Vertex Pharmaceuticals Inc (NASDAQ: ...