The investigators aim to evaluate the safety and efficacy of in utero hematopoietic stem cell transplantation (IUHSCT) for the treatment of fetuses diagnosed w…

Fanconi AnemiaAnemia, Hypoplastic, CongenitalCongenital Bone Marrow Failure Syndromes+5 more

~Estimated

~$10 – $8,750

Agnieszka CzechowiczNCT07408583

Phase 3

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

This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The study will eval…

Beta-ThalassemiaThalassemiaHematologic Diseases+4 more

~Estimated

~$10 – $8,750

Vertex Pharmaceuticals IncorporatedNCT05477563