Stage of Program: Clinical Trial, Phase 1


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 […]

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 […]

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 […]

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.

Clinical Investigation of a Humanized Anti-CD47 Antibody in Targeting Cancer Stem Cells in Hematologic Malignancies and Solid Tumors

A team at Stanford University is using a molecule known as an antibody to target cancer stem cells. This antibody can recognize and bind to CD47, a protein the cancer stem cells carry on their cell surface. The cancer cells use that protein to evade the component of our immune system that routinely destroys tumors. By disabling […]

A Phase I dose escalation and expansion clinical trial of the novel first-in-class Polo-like Kinase 4 (PLK4) inhibitor, CFI-400945 in patients with advanced solid tumors

Solid tumors are the most prevalent form of cancer, and are a major cause of death worldwide.  A team at UCLA is testing a drug for the treatment of cancer that works by blocking PLK4, a protein that is important in regulating cell growth, division and death. This protein is important for the survival of […]

A Phase I, Open-Label Study To Assess The Safety, Feasibility and Engraftment of Zinc Finger Nucleases (ZFN) CCR5 Modified Autologous CD34+ Hematopoietic Stem/Progenitor Cells (SB-728MR-HSPC) with Escalating Doses of Busulfan In HIV-1 (R5) Infected Subjects with Suboptimal CD4 Levels on cART

A team at City of Hope and Sangamo Therapeutics is genetically modifying patients’ blood forming stem cells to functionally cure people with HIV. The team is using a technology called zinc finger nucleases – a kind of molecular scissors – to snip out the target gene that codes for the CCR5 receptor. This receptor is the gateway for […]

Therapeutic Eradication of Cancer Stem Cells with UC-961 (Cirmtuzumab)

Cancer is a leading cause of death in California. Many cancers resist current therapies due to therapy-resistant cancer stem cells (CSCs). Scientists at UCSD have discovered a protein, ROR1, that is present on the surface of CSCs, but not on normal, healthy cells. The team has developed an antibody called cirmtuzumab, that specifically targets and attaches to ROR1. […]

Clinical Trial of Stem Cell Gene Therapy for Sickle Cell Disease

Sickle cell disease (SCD) is caused by a genetic mutation in the hemoglobin gene which causes red blood cells to “sickle” under conditions of low oxygen. SCD affects 1:500 African-Americans and is also common in Hispanic-Americans. The median survival for patients with SCD is 42 years for males and 48 years for females. A team at UCLA […]