FDA Approves First Gene-Editing Cell Therapy For Sickle Cell Disease, An Inherited Blood Disorder

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 VRTX and CRISPR Therapeutics AG's CRSP Casgevy is the first FDA-approved therapy utilizing CRISPR/Cas9, a type of genome editing technology. Patients' hematopoietic (blood) stem cells are modified by genome editing using CRISPR/Cas9 technology.

Last month, the U.K. Medicines and Healthcare Products Regulatory Agency granted conditional marketing authorization for Casgevy (exagamglogene autotemcel [exa-cel]) for sickle cell disease and transfusion-dependent beta-thalassemia.

The FDA is also reviewing exa-cel for transfusion-dependent beta-thalassemia (TDT) with a PDUFA date of March 30, 2024.

Bluebird Bio Inc's BLUE Lyfgenia (lovotibeglogene autotemcel (lovo-cel)) is a cell-based gene therapy. It uses a lentiviral vector (gene delivery vehicle) for genetic modification. FDA approval comes ahead of the PDUFA date of December 20.

Sickle cell disease is a group of inherited blood disorders affecting approximately 100,000 people in the U.S. 

Hematologic malignancy (blood cancer) occurred in patients treated with Lyfgenia. A black box warning is included in the label for Lyfgenia.

Price Action: CRSP shares are down 4.38% at $67.20, VRTX stock is down 0.72% at $351.39, and BLUE shares are down 20.40% at $3.83 on the last check Friday.