Vertex Crispr Agreement

Vertex Crispr Agreement

About CRISPR TherapeuticsCRISPR Therapeutics is a leading genetic publishing company focused on the development of transgenic drugs for serious diseases with its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary genetic editing technology that enables precise and targeted modifications of genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs in a wide range of disease areas such as hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic cooperation with leading companies such as Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its U.S. subsidiaries CRISPR Therapeutics, Inc. and Research and Development in Cambridge, Massachusetts, as well as branches in San Francisco, California and London, UK. For more information, see www.crisprtx.com. About CRISPR Therapeutics CRISPR Therapeutics` mission is to develop transgenic drugs for patients with serious illnesses.

Our therapeutic approach aims to cure diseases at the molecular level with the revolutionary genetic editing technology called CRISPR-Cas9. With our multidisciplinary team of world-renowned academics, drug developers and clinicians, we are uniquely positioned to transform CRISPR-Cas9 technology into human therapeutics. We have licensed our scientific founder, Dr. Emmanuelle Charpentier, for the use of humanotherapeutics, of our scientific founder. We are headquartered in Basel, Switzerland, with research and development operations in Cambridge, Massachusetts and offices in London, UK. www.crisprtx.com. CRISPR Therapeutics Investor Contact:Susan Kim 617-307-7503susan.kim@crisprtx.com, CTX001 is being developed as part of a co-development and co-marketing agreement between CRISPR Therapeutics and Vertex. Of the genetic editing methods studied/evaluated for TDT and SCD, CTX001 is the most advanced in clinical development. CTX001 is jointly developed by Vertex and CRISPR as part of a co-development and co-marketing agreement originally signed in 2015. TDT/SCD gene therapy is the first treatment from the joint research program. Gene therapy works by collecting hematopoietic stem cells and precursors from the blood, which are then treated with CRISPR/Cas9 technology.

The treated cells – CTX001 – are then melted into the patient`s system as part of a stem cell transplant. Currently, there is no cure for both genetic diseases, as the few treatments available are limited to the management of patients` disability. Conditions are caused by a mutation in genes that encode proteins vital to muscle cells to stay healthy. Vertex aims to use CRISPR/Cas9 to treat the mutant gene and potentially cure the disease. The American giant Vertex Pharmaceuticals is developing an existing cooperation with the Swiss company CRISPR Therapeutics and pays up to 890 million euros (USD 1 billion) for the development of treatments for genetic diseases responsible for muscle weakness with the genetic editing tool CRISPR/Cas9.

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