- Vertex to receive non-exclusive rights to CRISPR Therapeutics’ CRISPR/Cas9 to accelerate development of potentially curative cell therapies for T1D- - CRISPR Therapeutics to receive $100M upfront payment plus milestone and royalty payments on potential future gene-edited hypoimmune T1D products-
BOSTON and CAMBRIDGE, Mass. and ZUG, Switzerland, April 26, 2021 (GLOBE NEWSWIRE) -- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) today announced the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to CTX001, an
– First-ever approval of a CRISPR-based gene-editing therapy in the U.S. – – Approximately 16,000 patients 12 years of age and older with severe sickle cell disease may now be eligible for this one-time treatment – – Multiple authorized treatment centers activated – BOSTON & ZUG, Switzerland
-EU and UK filings completed in 2022; submissions validated by EMA and MHRA and the review procedure has begun as of January 2023 - BOSTON & ZUG, Switzerland --(BUSINESS WIRE)--Apr. 3, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) today announced
-Gene editing technology to be used to discover treatments to address the mutations and genes known to cause and contribute to cystic fibrosis- -Vertex and CRISPR to utilize gene editing approach to discover treatments for genetic diseases, including sickle cell disease- -Companies establish
- Beta thalassemia: All 15 patients were transfusion independent after CTX001 infusion - - Sickle cell disease: All seven patients were free of vaso-occlusive crises after CTX001 infusion - BOSTON & ZUG, Switzerland & CAMBRIDGE, Mass. --(BUSINESS WIRE)--Jun.
– Data from 75 patients with transfusion-dependent beta thalassemia or severe sickle cell disease with follow-up of up to 37.2 months continue to demonstrate that exa-cel has the potential to be a one-time functional cure – – Safety profile generally consistent with myeloablative conditioning and
-Vertex selects CTX001 as first gene edited treatment to be developed as part of collaboration with CRISPR Therapeutics - -Clinical Trial Application for CTX001 submitted in Europe to support initiation of Phase 1/2 clinical study in β-thalassemia in 2018- -Preclinical data for CTX001 presented
- Oral presentation of encore clinical data from Phase 3 clinical trials of exa-cel - - Oral and poster presentations on quality-of-life impacts of sickle cell disease or transfusion-dependent beta-thalassemia and burden of hematopoietic stem cell transplant - BOSTON and ZUG, Switzerland
CAMBRIDGE, Mass. and ZUG, Switzerland and BOSTON, May 12, 2021 (GLOBE NEWSWIRE) -- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) today announced two abstracts detailing updated data from the ongoing CTX001 clinical trials have been accepted for
