The US giant Vertex Pharmaceuticals is expanding an existing collaboration with the Swiss company CRISPR Therapeutics, paying up to €890M ($1B) to develop treatments for genetic diseases causing muscle weakness using the gene editing tool CRISPR/Cas9. In June last year, Vertex expanded an existing four-year collaboration with CRISPR to develop new treatments for genetic causes of human diseases. This technology may offer the ability to directly modify or correct the underlying disease-associated changes in the human genome for the potential treatment of a large number of both rare and common diseases. About the CRISPR-Vertex Collaboration CRISPR 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. Vertex and CRISPR will collaborate on the research, development and commercialization of treatments for hemoglobinopathies that emerge from the collaboration. In addition to our clinical development programs focused on cystic fibrosis, Vertex has more than a dozen ongoing research programs aimed at other serious and life-threatening diseases. The collaboration also provides Vertex with an observer seat on the CRISPR Board of Directors, which will be filled by Dr. Altshuler. Careers at CRISPR, © 2021 CRISPR Therapeutics. Gene therapy took another public step forward during a virtual presentation at a scientific conference on Friday. At 15 months following a single infusion of CTX001, patient 1 still hasn't required a transfusion. Shares of CRISPR Therapeutics have rallied 87.3% in the past three months compared with the ... ("TDT") in partnership with Vertex Pharmaceuticals. X. Returns as of 02/05/2021. Vertex and CRISPR will focus their initial gene editing research on discovering treatments to address the mutations and genes known to cause and contribute to cystic fibrosis and sickle cell disease. For each of the up to six treatments in-licensed for development, Vertex will pay future development, regulatory and sales milestones of up to $420 million as well as royalty payments on future sales. Dive Brief: Vertex Pharmaceuticals Inc. has selected CTX001 as the first drug it will co-develop and commercialize with CRISPR Therapeutics AG per the companies' ongoing collaboration. Climb-121 and Climb-111 are intended to enroll around 45 patients, but CRISPR and Vertex had to pause conditioning and dosing newly enrolled patients entering both studies due to the COVID-19 pandemic. The investment will provide Vertex with an ownership stake in CRISPR. Vertex and CRISPR Therapeutics began a research collaboration in October of 2015 focused on the use of CRISPR’s gene editing technology, known as CRISPR-Cas9, to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CRISPR 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. Under the terms of this strategic collaboration and license agreement, Vertex will pay $175 million upfront for the exclusive worldwide rights to CRISPR Therapeutics’ existing and future intellectual property including foundational CRISPR/Cas9 technology, novel endonucleases, single and double cut guide RNAs, and AAV vectors for DMD and DM1 gene editing products. www.crisprtx.com. –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 four-year research collaboration; CRISPR to receive $105 million up-front payment, of which $30 million is an equity investment and $75 million is cash, with potential for additional milestones and royalty payments–. CRISPR Therapeutics (NASDAQ:CRSP) and Vertex Pharmaceuticals (NASDAQ:VRTX) presented updated results from studies with CTX001, the first CRISPR-based gene-editing therapy to reach human-stage testing. Vertex will pay CRISPR $75 million in cash as part of its up-front commitment. For additional information and the latest updates from the company, please visit www.vrtx.com. Cory is a long-term minded analyst focused on the Healthcare Sector. In December 2017, Vertex and CRISPR paired up to co-develop CTX001, an investigational ex vivo CRISPR gene-edited therapy for patients suffering from β-thalassemia or sickle cell disease. The deal gives Vertex an … In the years leading up to the trial, Patient 1 required 34 units of red blood cells annually and Patient 2 needed transfusions at a rate of 61 units per year. Stock Advisor launched in February of 2002. CRISPR Therapeutics and Vertex's findings could put pressure on Bluebird bio, which won European approval for its gene therapy Zynteglo in beta thalassemia, but has hit multiple delays in the U.S. Bluebird's gene therapies have shown promise for both diseases, although the company has had to fine tune its approach in sickle cell. The initial focus of the collaboration will be on the use of CRISPR-Cas9 to potentially correct the mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene known to result in the defective protein that causes CF and to edit other genes that contribute to the disease. Vertex will have exclusive rights to license up to six new CRISPR-Cas9-based treatments that emerge from the collaboration. Treatment with CTX001 should lead to the body producing healthy fetal hemoglobin to replace the faulty hemoglobin at the source of their blood disorders. CRISPR Therapeutics Receives Grant to Advance In Vivo CRISPR/Cas9 Gene Editing Therapies for HIV. About the CRISPR-Vertex Collaboration CRISPR 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. CRISPR Therapeutics' (CRSP) lead gene-editing candidate CTX001, being developed for treating SCD and TDT, holds potential. Vertex Pharmaceuticals is the IBD Stock Of The Day after the biotech company gained a key designation for its gene-editing drug. CRISPR Therapeutics and Vertex Present New Data for Investigational CRISPR/Cas9 Gene-Editing Therapy, CTX001™ at American Society of Hematology Annual Meeting and Exposition, Together With Publication in the New England … Copyright, Trademark and Patent Information. Cas9 is a CRISPR-associated endonuclease (an enzyme) known to act as the “molecular scissors” that cut and edit, or correct, disease-associated DNA in a cell. CRISPR Therapeutics and Vertex … It could be a long time before we know if Vertex and CRISPR have a new marketable treatment option for sickle cell disease. CTX001 represents the first potential treatment to emerge from the joint research program. It could be a long time before we know if Vertex and CRISPR have a new marketable treatment option for sickle cell disease. The collaboration will evaluate the use of CRISPR-Cas9 across multiple diseases where targets have been validated through human genetics. Market data powered by FactSet and Web Financial Group. For five years in a row, Science magazine has named Vertex one of its Top Employers in the life sciences. We have licensed the foundational CRISPR-Cas9 patent estate for human therapeutic use from our scientific founder, Dr. Emmanuelle Charpentier. 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