Stage of Program: Clinical Stage Programs (2.0 and relevant 1.0 projects)


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 […]

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 […]

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. […]

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 […]

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 […]

Allogeneic Cardiosphere-Derived Cells for Duchenne Muscular Dystrophy Cardiomyopathy

Capricor is using donor cells derived from the heart to treat patients suffering from Duchenne Muscular Dystrophy (DMD), a genetic disorder that leads to progressive muscle degeneration, including heart muscle. One of the leading causes of death for children with DMD is heart failure and the aim of this treatment is to help improve heart […]

A Human Acellular Vessel in Patients Needing Renal Replacement Therapy: A Comparison with ePTFE Grafts as Conduits for Hemodialysis (HUMANITY)

Humacyte is using donor cells to create a bioengineered vein needed by people with end-stage kidney failure undergoing hemodialysis, the most common form of dialysis. In dialysis a person is connected to a machine that removes waste from their blood. The bioengineered vein is called a Human Acellular Vessel (HAV) and is made of extracellular matrix from […]