“It has been remarkable to be part of this groundbreaking program,” said Stephan Grupp, M.D., Ph.D., Section Chief of the Cellular Therapy and Transplant Section and Director of the Kelly Center for Cancer Immunotherapy at the Children's Hospital of Philadelphia, and Steering Committee Chair for the CLIMB-121 clinical program. approval of the first-ever medicine using CRISPR gene editing is breathtaking, and a truly humbling moment for me personally and for the whole organization,” said Samarth Kulkarni, Ph.D., Chairman and Chief Executive Officer of CRISPR Therapeutics. “When our company was founded, we had a vision to translate CRISPR technology into multiple breakthrough therapies. “I want to convey my deepest gratitude to the patients and investigators whose trust in this program paved the way for this landmark approval.” As importantly, CASGEVY is a first-in-class treatment that offers the potential of a one-time transformative therapy for eligible patients with sickle cell disease,” said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. “CASGEVY’s approval by the FDA is momentous: it is the first CRISPR-based gene-editing therapy to be approved in the U.S. This approval means that for the first time, approximately 16,000 patients with SCD may be eligible for a durable one-time therapy that offers the potential of a functional cure for their disease by eliminating severe VOCs and hospitalizations caused by severe VOCs. Food and Drug Administration (FDA) has approved CASGEVY™ (exagamglogene autotemcel ), a CRISPR/Cas9 genome-edited cell therapy, for the treatment of sickle cell disease (SCD) in patients 12 years and older with recurrent vaso-occlusive crises (VOCs). Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) announced today that the U.S. – Multiple authorized treatment centers activated –īOSTON & ZUG, Switzerland-(BUSINESS WIRE)-Dec. ![]() – Approximately 16,000 patients 12 years of age and older with severe sickle cell disease may now be eligible for this one-time treatment – Disease model iPS cell lines: Includes affected, carrier, and controls.– First-ever approval of a CRISPR-based gene-editing therapy in the U.S.Modified hES cell lines: Genetically engineered reporters, disease models, and isogenic corrected controls.Matched research bank material is available for assessment and use in preclinical applications. A Drug Master File in place with the FDA, and access will be provided upon request as part of an Investigational New Drug (IND) Application. These lines have been banked under cGMP compliant conditions and are thoroughly tested in compliance with FDA points to consider, making them ideal for use as starting material for clinical applications. Cell lines banked under GMP conditions: Master Cell Bank vials of WA09 (H9) are available.It includes related cell lines, apparently normal and disease model lines, and both natural and engineered isogenic pairs. ![]() Induced pluripotent stem (iPS) cell lines: Our large iPSC collection has tremendous diversity across multiple factors including ethnicity, age, sex and disease state. The collection includes WA09 (H9), the most widely used and published line globally. Human embryonic stem (hES) cell lines: Includes cell lines pre-approved for use with NIH and MRC funding, and registered with hPSCreg for use in the EU.
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