Stage of Program: Clinical Stage Programs (2.0 and relevant 1.0 projects)
Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency Using a Lentiviral Vector to Transduce Autologous CD34 Hematopoietic Stem Cells
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 be fatal. This trial will focus on SCID patients who have mutations in a gene called Artemis, the most difficult form of SCID to treat […]
Phase 1 Study of CD19/CD22 Chimeric Antigen Receptor (CAR) T Cells in Adults with Recurrent or Refractory B Cell Malignancies
Chimeric Antigen Receptor (CAR) T Cell Therapy is an innovative cancer therapy with very encouraging response rates in patients. The therapy works by isolating a patient’s own T cells (a type of immune cell) and then genetically engineering them to recognize a protein on the surface of cancer cells, triggering their destruction. In some patients […]
A Phase 1/2 Study to Assess the Safety, Tolerability, and Efficacy of ST-400 Autologous HSPC Transplant in Transfusion-dependent β-Thalassemia
Sangamo, Inc. is testing genetically engineered blood stem cells for the treatment of beta-thalassemia, a severe form of anemia caused by mutations in the hemoglobin gene. This genetic disorder requires life-long blood transfusions and carries a life expectancy of only 30-50 years. The Sangamo therapy takes a patient’s own blood stem cells and, using a […]
Clinical Study of T stem cell memory (Tscm)-based CAR-T cells in Patients with Multiple Myeloma
Poseida Therapeutics is testing the safety of a gene modified cell therapy to treat multiple myeloma, the abnormal growth of malignant plasma cells of the immune system. The company’s technology is seeking to destroy these cancerous myeloma cells with an immunotherapy approach that uses the patient’s own engineered immune system T cells to seek and […]
Cellular Immunotherapy for Induction of Immune Tolerance in HLA Matched Living Donor Kidney Transplant Recipients
Patients who receive kidney transplants must take life-long immunosuppressive drugs to prevent their immune system from rejecting the transplant. Over time, these drugs are toxic and can increase a patient’s risk of infection, heart disease, cancer and diabetes. Medeor Therapeutics has developed a stem cell-based treatment they hope will eliminate the need for immunosuppressive drugs […]
Treatment of sickle cell disease by induction of mixed chimerism and immune tolerance using CD4+ T-depleted haploidentical blood stem cell transplant
Scientists at the City of Hope are conducting a Phase 1 clinical trial testing a stem cell-based therapy for adult patients with severe sickle cell disease (SCD) – a chronic, debilitating blood disease. The therapy involves transplanting blood-forming stem cells from a donor into a patient who has received a milder, less toxic chemotherapy treatment that removes […]
Induction of Tolerance to Combined Kidney and Hematopoietic Progenitor Cell Transplants from HLA Haplotype Matched Living Donors
Stanford scientists are working with kidney transplant patients to see if injecting blood stem cells and T cells (which play an important role in the immune system) from the kidney donor into the kidney recipient can enable the recipient to bypass the need for a life-long dependence on immunosuppressant drugs which have numerous negative side effects.
Evaluation of the Safety and Tolerability of KA34 in a Phase 1, Double-Blind, Dose Escalation Trial in Patients with Knee Osteoarthritis
Researchers at the California Institute for Biomedical Research (CALIBR) have been awarded $8.447 million to test KA34, a drug that, in preclinical tests, recruits stem cells to create new cartilage in areas damaged by osteoarthritis. CIRM funded the research that developed this technology and now this Phase 1 trial will test this stem cell directed […]
Antiviral Cellular Therapy for Enhancing T-cell Reconstitution Before or After Hematopoietic Stem Cell Transplantation (ACES)
Viral infection can lead to fatal complications in patients with weakened immune systems resulting from chemotherapy, bone marrow or cord blood transplant, and other forms of inherited or acquired disorders. A team at Children’s Hospital of Los Angeles is testing the feasibility of providing these immune suppressed patients with engineered T-cells to fight these viruses. […]
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 that maintains or restores the insulin-producing pancreatic beta cells that are destroyed by this disease. Researchers at Caladrius Biosciences will take cells, called regulatory T cells […]