Severe sickle cell disease (SCD) research hub

A Phase 1/2/3 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Severe Sickle Cell Disease

This study is testing a new gene therapy called CTX001 for severe sickle cell disease. It aims to see if it's safe and helps improve symptoms by looking at things like how well the treatment works and if it reduces painful crises.