Press release
Vertex and CRISPR Therapeutics to Present at the American Society of Hematology (ASH) Annual Meeting and Exposition
- Oral presentation of encore clinical data from Phase 3 clinical trials of exa-cel - - Oral and poster presentations on quality-of-life impacts of sickle
About this update from Vertex Pharmaceuticals Incorporated
[{"type":"text","content":"\n- Oral presentation of encore clinical data from Phase 3 clinical trials of exa-cel -\n\n- Oral and poster presentations on quality-of-life impacts of sickle cell disease or transfusion-dependent beta-thalassemia and burden of hematopoietic stem cell transplant -\n\n BOSTON and ZUG, Switzerland--(BUSINESS WIRE)--\nVertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) today announced an oral, encore presentation of clinical data from patients with sickle cell disease (SCD) or transfusion-dependent beta-thalassemia (TDT) treated with the investigational therapy exagamglogene autotemcel (exa-cel) in CLIMB-111 or CLIMB-121 and followed in CLIMB-131, a long-term follow-up study. Vertex will also present new health economics and outcomes research from multiple studies in patients with SCD and TDT.\n\n“We are excited to be at ASH and share data from the pivotal exa-cel trials and from multiple real-world studies examining the burden of severe SCD and TDT,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. “We remain on track to complete submission of the first ever CRISPR-based therapy in the EU and U.K. this year, and we have initiated our rolling submission in the U.S., which we plan to complete in Q1 2023. We are working with urgency to bring forward this potential therapy to patients who are waiting.”\n\nVertex will have the following presentations at ASH this year.\n\n\nOral presentation, abstract #12 combined with abstract #2137, in partnership with CRISPR Therapeutics, entitled “Efficacy and Safety of a Single Dose of Exagamglogene Autotemcel for Transfusion-Dependent Beta-Thalassemia and Severe Sickle Cell Disease,” will be presented on Saturday, December 10 at 10:45 a.m CST. The presentation will include encore data as presented at the European Hematology Association Congress in June 2022 on patients with SCD or TDT who received the investigational therapy exa-cel in the CLIMB-111 or CLIMB-121 study and were followed in CLIMB-131, a long-term follow-up study.\n\n\nOral presentation, abstract #577, entitled “Health-Related Quality of Life, Disease Impacts, and Health Equity Concerns in Adults with Sickle Cell Disease with Recurrent Vaso-Occlusive Crises: Preliminary Results from a Global Longitudinal Sur...