Vertex Crispr Agreement

Vertex Crispr Agreement

About CRISPR TherapeuticsCRISPR Therapeutics is a leading gene editing company focused on the development of genetically modified drugs for serious diseases with its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that enables precise and targeted modifications of genomic DNA. CRISPR Therapeutics has developed a portfolio of therapeutic programs in a wide range of disease areas, including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has strategic collaborations with leading companies such as Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. built. CrispR Therapeutics AG is headquartered in Zug, Switzerland, with its U.S. subsidiary CRISPR Therapeutics, Inc., and R&D activities based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, UK. For more information, see www.crisprtx.com. CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex.

Of the gene editing approaches studied/evaluated for TDT and SCD, CTX001 is the most advanced in clinical development. About CRISPR Therapeutics The mission of CRISPR Therapeutics is to develop transgenic medicines for patients with serious diseases. Our therapeutic approach aims to cure diseases at the molecular level with the revolutionary CRISPR-Cas9 gene editing technology. With our multidisciplinary team of world-renowned academics, drug developers and clinicians, we are uniquely positioned to translate CRISPR-Cas9 technology into human therapy drugs. We have obtained the basic CRISPR-Cas9 license for human therapeutic use from our scientific founder, Dr. Emmanuelle Charpentier. We are headquartered in Basel, Switzerland, our R&D activities are located in Cambridge, Massachusetts, and we have branches in London, UK. www.crisprtx.com CRISPR Therapeutics Investor Contact: Susan Kim, +1 617-307-7503susan.kim@crisprtx.com CTX001 is jointly developed by Vertex and CRISPR under a co-development and co-commercialization agreement signed in 2015.

TDT/SCD gene therapy is the first treatment from the joint research programme. Vertex and CRISPR use the gene editing approach to detect treatments for genetic diseases, including cell dremity. . Founded in 1989 in Cambridge, Massachusetts, Vertex now has research and development sites and sales offices in the United States, Europe, Canada and Australia. Five years in a row, the science magazine Vertex is one of its top employers in the life sciences. For more information and the latest company updates, please visit www.vrtx.com. . TRAIN, Switzerland and CAMBRIDGE, Mass. and BOSTON, Dec. 5, 2020 (GLOBE NEWSWIRE) — CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced new data on a total of 10 patients treated with crispr/Cas9`s CTX001, who have a consistent and sustained response to treatment.

All seven patients with transfusion beta-thalassemia (TDT), including three with severe genotype or b0/b0, were transfusion-independent at last follow-up and all three patients with dieh drecelia (CPD) were free of vaso-occlusive seizures from CTX001 infusion to final follow-up. . . .