Stage of Program: Clinical Trial, Phase 1/2
Lentiviral Gene Therapy for Infants with X-linked Severe Combined Immunodeficiency using Autologous Bone Marrow Stem Cells and Busulfan Conditioning
Therapeutic Candidate or Device Bone marrow stem cells will be transduced with a lentiviral vector to deliver a normal copy of the gamma-chain gene to treat X-linked SCID. Indication x-linked severe combined immunodeficiency, a severe pediatric disorder in which children have multiple defects in their immune system. Therapeutic Mechanism The gene therapy will correct the […]
Pulmonary Arterial Hypertension Treated with Cardiosphere-Derived Allogeneic Stem Cells
Therapeutic Candidate or Device CAP-1002 Indication Patients with Pulmonary Arterial Hypertension (PAH) Therapeutic Mechanism CAP-1002's mechanism of action may result in reduced wall thickening of small blood vessels in the lung that are markedly narrowed/obstructed in PAH. The latter results in high resistance against which the right ventricle (RV) must pump in order to drive […]
Human Neural Progenitors Secreting Glial Cell Line-Derived Neurotrophic Factor (CNS10-NPC-GDNF) for the Treatment of Amyotrophic Lateral Sclerosis
Therapeutic Candidate or Device CNS10-NPC-GDNF – a neural progenitor cell secreting GDNF Indication ALS Therapeutic Mechanism This therapy will replace damaged astrocytes. The new astrocytes will release paracrine factors. As the cells have been modified to release GDNF they will also provide this factor to dying motor neurons. Unmet Medical Need There is no treatment […]
Stem Cell Gene Therapy for HIV Mediated by Lentivector Transduced, Pre-selected CD34+ Cells in AIDS lymphoma patients
Therapeutic Candidate or Device HSC gene modified by a lentiviral vector which encodes a triple combination of HIV-resistance genes and a pre-selective marker. Indication HIV in AIDS-lymphoma patients. Therapeutic Mechanism Bone marrow transplants are standard of care for AIDS-lymphoma patients providing a cure for the patient's lymphoma. A purified population of triple combination anti-HIV lentiviral […]
A Phase I/II, Non Randomized, Multicenter, Open-Label Study of G1XCGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease
Therapeutic Candidate or Device The therapeutic product candidate is autologous CD34+ hematopoietic stem/progenitor cells (HSPC) transduced with the G1XCGD lentiviral vector. Indication The target indication is for the transplantation of patients with severe X-linked Chronic Granulomatous Disease (XCGD) lacking matched donors. Therapeutic Mechanism Transplantation and engraftment of gene-corrected autologous HSPC after reduced intensity conditioning for […]
Clinical Development of a Cell Therapy for Diabetes
We are developing a stem cell-derived replacement cell therapy for insulin-requiring diabetes. Through a process known as directed differentiation, embryonic stem cells are turned into pancreatic cells in the laboratory. The pancreatic cells are loaded into a delivery device, which is essentially a small envelope made with a semi-permeable membrane, not unlike a flat tea […]
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 […]
A Phase I/IIa Dose Escalation Safety Study of AST-OPC1 in Patients with Cervical Sensorimotor Complete Spinal Cord Injury
The proposed project is designed to assess the safety and preliminary activity of escalating doses of human embryonic stem cell derived oligodendrocyte progenitor cells (OPCs) for the treatment of spinal cord injury. OPCs have two important functions: they produce factors which stimulate the survival and growth of nerve cells after injury, and they mature in […]