Vertex Pharmaceuticals Inc. ((VRTX)) announced an update on their ongoing clinical study.
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Vertex Pharmaceuticals Inc. is conducting a Phase 3b study titled ‘A Phase 3b Study to Evaluate Efficacy and Safety of a Single Dose of Autologous CRISPR Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Transfusion-Dependent β-Thalassemia or Severe Sickle Cell Disease.’ The study aims to assess the safety and efficacy of CTX001, a CRISPR-Cas9 modified stem cell treatment, in patients with transfusion-dependent β-thalassemia (TDT) and severe sickle cell disease (SCD).
The intervention being tested is CTX001, a biological treatment involving autologous CD34+ hematopoietic stem and progenitor cells modified using CRISPR-Cas9 technology. The treatment is designed to address genetic disorders by modifying cells to potentially eliminate the need for regular blood transfusions in patients.
This interventional study follows a single-group assignment model without masking, focusing on treatment as its primary purpose. Participants receive a single infusion of CTX001 through a central venous catheter after undergoing myeloablative conditioning with busulfan.
The study began on July 26, 2022, with primary completion and estimated completion dates yet to be announced. The latest update was submitted on March 28, 2025, indicating the study is actively recruiting participants.
The study’s progress could significantly impact Vertex Pharmaceuticals’ stock performance and investor sentiment, as successful results may position the company as a leader in gene-editing therapies for hematologic diseases. Competitors in the gene-editing space may also feel pressure to advance their own research and development efforts.
The study is ongoing, with further details available on the ClinicalTrials portal.