WebApr 10, 2024 · Vertex fully expects to file for regulatory approvals of CTX001 before the end of 2024. ... If the experimental drug performs well in phase 3 testing, a door to yet another market could open wide ... WebJun 11, 2024 · The ongoing Phase 1/2 open-label trial, CLIMB-SCD-121, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with …
β-Thalassemia: evolving treatment options beyond transfusion and …
WebOct 30, 2024 · Enrollment is ongoing in the Phase 1/2 studies evaluating the novel gene-editing therapy CTX001 for the treatment of severe sickle cell disease and beta thalassemia. Vertex and its partner CRISPR Therapeutics plan to provide the first clinical data from these studies in the fourth quarter of 2024. WebDec 5, 2024 · CTX001 was manufactured from these CD34+ cells by editing with CRISPR-Cas9 with the use of a single-guide RNA molecule ( Figure 1B ). 30 We used DNA sequencing to evaluate the percentage of... Sickle cell disease is an increasing global health problem. Estimates suggest that … chow chow old fashioned relish
Evaluation of Safety and Efficacy of CTX001 in Pediatric …
WebJun 23, 2024 · The first patient with severe sickle cell disease (SCD) who received a single infusion of CTX001, an experimental gene-editing cell therapy, remains free of vaso-occlusive crises (VOCs) nine months after treatment, a Phase 1/2 clinical trial shows. WebExagamglogene autotemcel (exa-cel), formerly known as CTX001™, is an investigational, ... In 2024, Vertex and CRISPR Therapeutics initiated a Phase 1/2/3 study evaluating exa-cel in subjects ages 12-35 with sickle cell disease and recurrent vaso-occlusive crises (VOCs). WebApr 29, 2024 · In this case, the designation was granted following data from the Phase 1/2 CLIMB-Thal-111 clinical trial. Altogether, this trial is evaluating the safety, efficacy, and tolerability of CTX001 for patients with transfusion-dependent beta thalassemia. Enrolled patients are between ages 12-35. Overall, 45 patients will enroll. chow chow origin