CRISPR Therapeutics AG, a gene editing company, focuses on developing gene-based medicines for serious diseases using its proprietary Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) platform. Its CRISPR/Cas9 is a gene editing technology that allows for precise directed changes to genomic DNA. The company has a portfolio of therapeutic programs across a range of disease areas, including hemoglobinopathies, oncology, regenerative medicine, and rare diseases. The company's lead product candidate is CTX001, an ex vivo CRISPR gene-edited therapy for treating patients suffering from transfusion-dependent beta-thalassemia or severe sickle cell disease in which a patient's hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin in red blood cells. It also develops CTX110, a donor-derived gene-edited allogeneic CAR-T investigational therapy targeting cluster of differentiation 19 positive malignancies; CTX120, a donor-derived gene-edited allogeneic CAR-T investigational therapy targeting B-cell maturation antigen for the treatment of relapsed or refractory multiple myeloma; and CTX130, a donor-derived gene-edited allogeneic CAR-T investigational therapy targeting Cluster of Differentiation 70 to treat various solid tumors and hematologic malignancies. In addition, the company develops VCTX210, a gene-edited immune-evasive stem cell-derived product candidate for the treatment of treatment of type 1 diabetes; and pursues various in vivo gene-editing programs that target the liver, lung, muscle, and central nervous system diseases. It has strategic partnerships with Bayer Healthcare LLC, Vertex Pharmaceuticals Incorporated, ViaCyte, Inc., Nkarta, Inc., and Capsida Biotherapeutics. CRISPR Therapeutics AG was incorporated in 2013 and is headquartered in Zug, Switzerland.

CRISPR Therapeutics AG

CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom.

Bill & Melinda Gates Foundation

EcoR1 Capital

SR One

Wellington Capital Management

New Leaf Venture Partners

Franklin Templeton

Clough Capital Partners

New Enterprise Associates

Abingworth

Versant Ventures

Vertex Pharmaceuticals

CRISPR Therapeutics

The European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for the conditional approval of the CRISPR/Cas9 gene-edited treatment, co-developed with Vertex Pharmaceuticals, for #SickleCellDisease and #BetaThalassemia. 

#Breaking: The U.S. FDA approves the CRISPR/Cas9 gene-edited treatment, co-developed with Vertex Pharmaceuticals, for #SickleCellDisease. Learn more: https://bit.ly/4apMw9p

Today, we provided an update on our immuno-oncology pipeline of CRISPR/Cas9 gene-edited allogeneic chimeric antigen receptor (CAR) T cell product candidates and we announced expansion into autoimmune disease. Learn more here: https://bit.ly/4a8zpsM

#Breaking: The UK Medicines and Healthcare products Regulatory Agency (MHRA) approves the treatment for #SickleCellDisease and #BetaThalassemia in Great Britain. This a historic scientific milestone arising out of our collaboration with Vertex. Learn more: crisprtx.com

Today was an exciting day as Dr. PK Morrow, CRISPR Therapeutics Chief Medical Officer, presented preclinical data on our investigational programs for the treatment of cardiovascular disease in two oral sessions at the American Heart Association Scientific Sessions. Learn more: https://bit.ly/47oCkLX #AHA23

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