|CRISPR Therapeutics Announces Oral Presentation of New Data on CTX001, a CRISPR Gene-Edited Medicine for β-Thalassemia and Sickle Cell Disease, at the ASH Annual Meeting|
Session: 112 Thalassemia and Globin Gene Regulation I Track
Time and Date:
Location: Georgia World Congress Center,
Presentation: CRISPR/Cas9 Genome Editing to Treat Sickle Cell Disease and B-Thalassemia: Re-Creating Genetic Variants to Upregulate Fetal Hemoglobin Appear Well-Tolerated, Effective and Durable
CTX001 is an investigational CRISPR gene-edited therapy for patients suffering from β-thalassemia and sickle cell disease in which a patient’s hematopoietic stem cells are engineered to produce high level of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen carrying hemoglobin that is naturally present at birth and is normally replaced by the adult form of hemoglobin. The elevation of HbF is expected to alleviate transfusion-requirements for β-thalassemia patients and painful and debilitating sickle crises for sickle cell patients.
About CRISPR Therapeutics
CRISPR Therapeutics is a leading gene-editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR / Cas9 gene-editing platform. CRISPR/Cas9 is a revolutionary technology that allows for precise, directed changes to genomic DNA. The company's multi-disciplinary team of world-class researchers and drug developers is working to translate this technology into breakthrough human therapeutics in a number of serious diseases. Additionally, CRISPR Therapeutics has established strategic collaborations with Bayer AG and Vertex Pharmaceuticals to develop CRISPR-based therapeutics in diseases with high unmet need. The foundational CRISPR / Cas9 patent estate for human therapeutic use was licensed from the company's scientific founder Emmanuelle Charpentier, Ph.D. 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,