Page 2 - Exa Cel News Today

The FDA has approved a gene-editing therapy for sickle cell disease (SCD) for the first time.

FDA soon rules if gene-editing CRISPR drug Exa-cel can be used commercially to treat sickle cell disease, opening door to many other therapies.

With a landmark U.K. approval in hand for Casgevy (exagamglogene autotemcel [exa-cel]) to treat sickle cell disease and transfusion-dependent beta thalassemia, Crispr Therapeutics AG and partner Vertex Therapeutics Inc. are turning their attention to the PDUFA dates set by the U.S. FDA for the treatment in both conditions.

Had it been asked to, the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee would have voted Oct. 31 to recommend approval of Vertex Pharmaceutical Inc. and Crispr Therapeutics AG’s exagamglogene autotemcel, or exa-cel, as a one-time transformative treatment for severe sickle cell disease in individuals 12 and older.

FDA will soon decide approval of exa-cel and lovo-cel for sickle cell disease. These may be one-time treatment options for SCD patients burdened by unmet needs.

Sickle cell disease is a painful, debilitating, and possibly deadly inherited disorder that currently can only be remedied with a bone marrow transplant. Now, Boston's Vertex...

An advisory committee is meeting to discuss the potential approval of exagamglogene autotemcel, a CRISPR-based gene editing therapy candidate for sickle cell disease.