Project Objective: Clinical Trial, Phase 2

Efficacy and safety of cryopreserved autologous CD34+ HSC transduced with EFS lentiviral vector encoding for human ADA gene in ADA-SCID subjects

Therapeutic Candidate or Device Autologous CD34+ hematopoietic stem cells (HSCs) transduced with a lentiviral vector encoding the human ADA gene (or "OTL-101") Indication Adenosine Deaminase – Severe Combined Immunodeficiency (or ADA-SCID) Therapeutic Mechanism This project will lead to a License Application for OTL-101 as a treatment for ADA-SCID. The patient’s own stem cells ("autologous") are […]

Allogeneic Cardiosphere-Derived Cells for Duchenne Muscular Dystrophy Cardiomyopathy

Therapeutic Candidate or Device Allogeneic Cardiosphere-Derived Cells (CDCs, CAP-1002) Indication Duchenne Muscular Dystrophy Cardiomyopathy Therapeutic Mechanism Secretion of identified bioactive elements will reduce myocardial fibrosis and improve cardiac function. Unmet Medical Need Heart failure is the leading cause of death among young men with Duchenne Muscular Dystrophy. No specific therapies exist to treat this element […]

Alpha Stem Cell Clinic for the Development of Regenerative Therapies

The proposed alpha clinic will bring together an outstanding team of physician-scientists with substantial clinical trials experience including stem cell and other cellular treatments of blood diseases and others. This team will also draw on our unique regional competitive advantages derived from our history of extensive collaboration with investigators at many nearby first-class research institutions […]

UCLA-UCI Alpha Stem Cell Clinic (ASCC) Consortium

Two world renowned research universities with their regional partners will join forces to create the CIRM Alpha Stem Cell Clinic (ASCC) Consortium. We are uniquely qualified by our respective institutional knowledge and collective experience to establish best practices for the delivery of stem cell therapies and education as we combine: 1) world class state-of-the-art medical […]

The Innovation-Alpha Clinic for Cellular Therapies (I-ACT) – A Program for the Development and Delivery of Innovative Cell-based Treatments and Cures for Life-threatening Diseases.

As the largest provider of bone marrow cell transplants in California, and the second largest in the nation, our institution has great expertise and an excellent record of safety in the delivery of stem cell treatments. We now propose to create the Alpha Clinic for Cell Therapy and Innovation (ACT-I) in which new, state-of-the-art, stem […]

Allogeneic Cardiac-Derived Stem Cells for Patients Following a Myocardial Infarction

The proposed research will demonstrate both safety and efficacy of a heart-derived stem cell product in patients who have experienced a heart attack either recently or in the past by conducting a mid-stage clinical trial. A prior early-stage trial showed that the product can repair damaged portions of the heart after a heart attack in […]