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This new economic valuation of the societal and health care benefits of gene therapy to treat sickle cell disease (SCD) concludes the highly personalized treatment has potential to be cost effective if priced below $2 million.
Excision BioTherapeutics has so far released positive safety data from the first 3 participants living with HIV-1, with no evidence of vector shedding in sexual organ tissue.
FDA approves 1st CRISPR gene therapy to treat sickle cell disease. Nine hospitals have been authorized to offer the one-time treatment by Vertex Pharmaceuticals
Both Vertex Pharmaceuticals Inc.’s Casgevy (exagamglogene autotemcel, exa-cel) and Bluebird Bio Inc.’s Lyfgenia (lovotibeglogene autotemcel, lovo-cel) received U.S. FDA approval Dec. 8, providing 16,000 American sickle cell patients who have recurring vaso-occlusive events with access to the first cell-based gene therapies.
New FDA-approved therapy treatments that use genetic modification called CRISPR may lead to a sickle cell disease cure.