- In December 2023, Vertex Pharmaceuticals and CRISPR Therapeutics announced the U.S. FDA approval of Casgevy (exa-cel), the first CRISPR-based gene-editing therapy for sickle cell disease. This one-time treatment is now available at authorized treatment centers across the U.S., offering a potential cure for eligible patients aged 12 and older with recurrent vaso-occlusive crises
- In September 2024, Pfizer announced the withdrawal of Oxbryta (voxelotor) from all markets due to safety concerns, including an increased risk of complications and death. This decision followed findings from clinical data indicating imbalances in vaso-occlusive crises and fatal events among patients
- In January 2025, Bluebird Bio expanded access to Zynteglo, its gene therapy for transfusion-dependent β-thalassemia (also being studied for SCD), by partnering with several leading U.S. academic hospitals. This initiative aims to make curative therapies more accessible and affordable, particularly for patients in underserved regions. The company also initiated a pilot reimbursement model to streamline insurance coverage and reduce out-of-pocket costs
- In January 2025, Beam Therapeutics announced updated data from the BEACON Phase 1/2 clinical trial of BEAM-101, a base-editing gene therapy designed to increase fetal hemoglobin in SCD patients. The data demonstrated robust and durable increases in fetal hemoglobin and reductions in sickle hemoglobin, with rapid neutrophil and platelet engraftment. No vaso-occlusive crises were reported post-engraftment
