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CRISPR Therapeutics Announces Completion of FDA Advisory Committee Meeting for Exagamglogene Autotemcel (exa-cel) for Severe Sickle Cell Disease
-Exa-cel PDUFA target action date is December 8, 2023 for severe sickle cell disease (SCD)- ZUG, Switzerland and BOSTON, Oct. 31, 2023 (GLOBE NEWSWIRE) --
About this update from Crispr Therapeutics Ag
[{"type":"text","content":"-Exa-cel PDUFA target action date is December 8, 2023 for severe sickle cell disease (SCD)- ZUG, Switzerland and BOSTON, Oct. 31, 2023 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP) today announced the completion of the U.S. Food and Drug Administration’s (FDA) Cellular, Tissue, and Gene Therapies Advisory Committee meeting for exagamglogene autotemcel (exa-cel) for the treatment of SCD in people ages 12 and older with recurrent vaso-occlusive crises (VOCs). Exa-cel is the first potential therapy to emerge from a strategic partnership between CRISPR Therapeutics and Vertex Pharmaceuticals. If approved, exa-cel could be the first genetic therapy available to approximately twenty thousand people with severe SCD in the U.S. The FDA granted priority review for exa-cel in the treatment of people with SCD and assigned a Prescription Drug User Fee Act (PDUFA) action date of December 8, 2023. Exa-cel’s Biologics License Application (BLA) for transfusion-dependent beta-thalassemia (TDT) was assigned a PDUFA date of March 30, 2024. About the CRISPR Therapeutics and Vertex CollaborationCRISPR Therapeutics and Vertex entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. Exa-cel represents the first potential treatment to emerge from the joint research program. Under an amended collaboration agreement, Vertex now leads global development, manufacturing and commercialization of exa-cel and splits program costs and profits worldwide 60/40 with CRISPR Therapeutics. (CRSP-GEN) About CRISPR Therapeutics CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, ...