Tagged as: exagamglogene autotemcel

UK Authorizes World-First Gene Therapy from Vertex and CRISPR Therapeutics for Blood Disorders

Earlier today, UK’s Medicines and Healthcare products Regulatory Agency (MHRA) granted conditional marketing authorization to Vertex Pharmaceuticals and CRISPR Therapeutics for their CRISPR/Cas9 gene therapy, CASGEVY (exagamglogene autotemcel), marking the world’s first regulatory approval for a CRISPR-based gene-edited therapy.  CASGEVY is the first medicine to be licensed that uses the…

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FDA to Review Vertex Pharmaceutical’s Application for CRISPR-based Gene Therapy Exa-cel

The Cellular, Tissue, and Gene Therapies Advisory Committee of the FDA is meeting tomorrow, Tuesday, October 31, 2023, to review a Biologics License Application by Vertex Pharmaceuticals Inc. for exagamglogene autotemcel (exa-cel).  Exa-cel is a cell-based gene therapy product composed of the patient’s own hematopoietic stem and progenitor cells (HSPCs) edited…

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