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CRISPR Therapeutics AG reports developments in gene-based medicines built around CRISPR/Cas9 gene editing, cell therapy, in vivo editing and siRNA programs. Recurring updates cover CASGEVY, developed with Vertex for severe sickle cell disease and transfusion-dependent beta thalassemia, as well as pipeline work in cardiovascular disease, autoimmune disease, oncology, rare disease and Type 1 diabetes.
Company news also includes financial results, business updates, investor conference presentations, manufacturing and platform disclosures, collaborations such as Sirius Therapeutics, and capital actions involving common shares or convertible senior notes.
CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals presented promising results for CTX001, a gene-editing therapy targeting transfusion-dependent beta thalassemia (TDT) and sickle cell disease (SCD) at the EHA virtual congress. Notable outcomes include two beta thalassemia patients achieving transfusion independence at 5 and 15 months post-infusion, and one SCD patient remaining free from vaso-occlusive crises at 9 months. All treated patients have successfully engrafted and demonstrated significant improvements in hemoglobin levels and other health metrics, showcasing the potential of CTX001 as a transformative therapeutic option.
CRISPR Therapeutics has announced its participation in two virtual investor conferences in June 2020. The Jefferies Global Healthcare Conference will take place on June 2, 2020, at 1:30 p.m. ET. The company will also join the Goldman Sachs 41st Annual Global Healthcare Conference on June 9, 2020, at 9:40 a.m. ET. Investors can access live webcasts of both events on the company's website, with a replay available for 14 days post-presentation. CRISPR Therapeutics focuses on developing gene-based medicines for serious diseases utilizing its proprietary CRISPR/Cas9 technology.
CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals (Nasdaq: VRTX) announced that new data from ongoing Phase 1/2 trials of CTX001, a gene-editing therapy, will be presented at the EHA Congress from June 11-14, 2020. The presentations will include 12 months of follow-up data from the CLIMB-111 trial for transfusion-dependent beta thalassemia and 6 months of data from the CLIMB-121 trial for severe sickle cell disease. CTX001 aims to elevate fetal hemoglobin levels, potentially alleviating treatment burdens for patients. The collaboration focuses on innovative treatments for serious diseases.
CRISPR Therapeutics (CRSP) and Vertex Pharmaceuticals (VRTX) announced that the FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to CTX001, a gene-edited therapy for sickle cell disease and beta thalassemia. This designation marks a crucial regulatory achievement and indicates CTX001's potential to address serious health conditions. RMAT designation allows for expedited development processes, enhancing the pathway to potential approval. CRISPR is set to present additional clinical data for CTX001 later this year, bolstering the collaboration's prospects in addressing unmet medical needs.
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