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BioWorld - Monday, June 2, 2025
Home » Keywords » exa-cel

Items Tagged with 'exa-cel'

ARTICLES

Sickle cells

US FDA approves first CRISPR-based gene therapy Casgevy for sickle cell; Lyfgenia gets nod, too

Dec. 8, 2023
By Karen Carey and Mari Serebrov
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.
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Red blood cells, DNA

Historic MHRA exa-cel win bagged, Crispr/Vertex await FDA nod

Nov. 16, 2023
By Randy Osborne
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.
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Sickle cell illustration

Adcom: Don’t sacrifice progress for perfection

Nov. 1, 2023
By Mari Serebrov
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.
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Sickle cell disease 3D illustration

No red flags flying ahead of adcom on Vertex’s SCD therapy

Oct. 27, 2023
By Lee Landenberger
It’s just a discussion next week of Vertex Pharmaceuticals Inc. and Crispr Therapeutics AG’s sickle cell disease (SCD) therapy, with no vote expected from the U.S. FDA’s Cellular, Tissue and Gene Therapies Advisory Committee. The FDA isn’t questioning the drug’s efficacy as much as it is concerned about the effects of off-target editing.
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