- Ongoing, recruitingTherapeutic confirmatory (Phase III)
A Phase 1/2/3 Study of the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) in subjects with Transfusion-Dependent β-Thalassemia
This study looks at a new gene therapy, Casgevy, for people with severe beta-thalassemia who need regular blood transfusions. Researchers want to see if this treatment is safe and can help patients produce enough healthy red blood cells on their own, reducing or stopping the need for transfusions.
For: Transfusion-dependent β-thalassemia (TDT)Germany - Ongoing, recruitingPhase I and Phase II (Integrated)- Other
An open-label phase 1/2 study to evaluate the safety, biological response and efficacy of a single dose of Temferon (autologous CD34+-enriched hematopoietic stem and progenitors cells genetically modified with human Interferon-α2) in patients with metastatic renal cell carcinoma
This study is testing a new treatment called Temferon for advanced kidney cancer that has spread. It checks if Temferon is safe, how the body reacts to it, and how well it works in people who have already tried standard treatments.
For: Patients with locally advanced/metastatic renal cell carcinoma with evidence of disease progression following standard of care treatmentItaly - Ongoing, recruitingPhase I and Phase II (Integrated)- First administration to humans
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.
For: Severe sickle cell disease (SCD)Belgium · Italy