Phase 2 Study of Hematopoietic Stem Cell Gene Transfer Inducing Fetal Hemoglobin in Sickle Cell Disease
Boston Children's Hospital is conducting a gene therapy clinical trial for sickle cell disease (SCD). This project is part of an agreement between CIRM and the National Heart, Lung, and…
The CuRe Trial: Cellular Therapy for In Utero Myelomeningocele Repair and The CuRe Trial: Cellular Therapy for In Utero Repair of Myelomeningocele
UC Davis is conducting a clinical trial for in utero repair of myelomeningocele (MMC), the most severe form of spina bifida. MMC is a birth defect that occurs due to incomplete…
Mesenchymal Stromal Cells for ARDS (COVID positive and COVID negative)
Acute respiratory distress syndrome (ARDS) is a life-threatening lung injury that occurs when fluid leaks into the lungs. As a result, breathing becomes difficult and oxygen cannot get into the…
A Phase 2 Study of the Safety of Repeat Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects with Retinitis Pigmentosa
Retinitis pigmentosa is blinding eye disease that affects approximately 150,000 individuals in the US and 1.5 million people around the world. It is caused by the destruction of light-sensing cells…
Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency Using a Lentiviral Vector to Transduce Autologous CD34 Hematopoietic Stem Cells and Gene Therapy for Artemis-Deficient Severe Combined Immunodeficiency Using a Self-Inactivating Lentiviral Vector
UC San Francisco researchers aim to repair the damaged immune system of children born with severe combined immunodeficiency (SCID), a genetic blood disorder in which even a mild infection can…
Phase 2 Safety and Efficacy Study of CLBS03 Autologous T-Regulatory Cells in Adolescents with Recent Onset Type 1 Diabetes Mellitus
Children with type 1 diabetes (T1D) face lifelong struggles with controlling their blood sugar levels and, despite careful management, an increased risk of severe complications. Currently, there is no approved therapy…
