Stage of Program: Clinical Stage Programs (2.0 and relevant 1.0 projects)
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. […]
GENE-MODIFIED HEMATOPOIETIC STEM/PROGENITOR CELL BASED THERAPY FOR HIV DISEASE
Calimmune is genetically modifying patients’ own blood-forming stem cells (also known as bone marrow stem cells) so they can produce immune cells—the ones normally destroyed by the HIV virus—that cannot be infected by the virus. The goal of this treatment is to enable the patients to clear their systems of the virus, effectively curing the […]
Stem Cell Gene Therapy for HIV Mediated by Lentivector Transduced, Pre-selected CD34+ Cells in AIDS lymphoma patients
A team at UC Davis is taking a patient’s blood forming stem cells and inserting three anti-HIV genes into them and then returning them to the individual to help rebuild their immune system. The anti-HIV genes are then passed on to all new immune system cells, which makes them resistant to HIV. Because AIDS-related lymphoma is linked […]
Lentiviral Gene Therapy for Infants with X-linked Severe Combined Immunodeficiency using Autologous Bone Marrow Stem Cells and Busulfan Conditioning
St. Jude Children’s Research Hospital is teaming up with UC San Francisco to repair the damaged immune system of children born with SCID. They will genetically modify the patient’s own blood stem cells, with the goal of creating a new blood system and restoring the health of the immune system.
A monoclonal antibody that depletes blood stem cells and enables chemotherapy free transplants
This trial proposes to replace SCID patients’ dysfunctional immune cells with healthy ones using a safer form of bone marrow transplant (BMT). Current BMT procedures must use toxic chemotherapy to make space in the bone marrow for the healthy transplanted stem cells to engraft. The Stanford team will instead test a safe, non-toxic protein called […]
Efficacy and safety of cryopreserved autologous CD34+ HSC transduced with EFS lentiviral vector encoding for human ADA gene in ADA-SCID subjects
In ADA-SCID, allogeneic hematopoietic (blood) stem cell transplants from non-matched sibling donors are a high risk procedure. Additionally, the efficacy of chronic enzyme replacement therapy is uncertain in the long-term. A team at UCLA is using a patient’s own blood stem cells to try and rebuild the damaged immune systems of patients with ADA-SCID. They will use what’s […]
In Utero Hematopoietic Stem Cell Transplantation For The Treatment Of Fetuses With Alpha Thalassemia Major
Dr. MacKenzie and her team at UCSF are using hematopoietic stem cells (HSCs) to treat babies in the womb who have alpha thalassemia major, a blood disorder that is almost always fatal. Current treatment requires in utero blood transfusions and monthly blood transfusions for life or a bone marrow transplant if a suitable donor is identified. In this trial, HSCs […]
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
X-linked Chronic Granulomatous Disease (X-CGD) is a rare immune disorder that prevents white blood cells from killing foreign invaders. This results in severe, recurrent infections that can impact quality and length of a patient’s life. X-CGD is usually diagnosed before age 5, but without treatment, children die before age 10. A team at UCLA is using the patient’s own genetically […]
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 […]
Phase 1 Safety Assessment of CPCB-RPE1, hESC-derived RPE Cell Coated Parylene Membrane Implants, in Patients with Advanced Dry Age Related Macular Degeneration
Age-related macular degeneration is a progressive disease resulting in death of the retinal pigment epithelium (RPE) causing distortion to central vision and eventually to legal blindness. Regenerative Patch Technologies and scientists at the University of Southern California and UC Santa Barbara, are growing specialized cells of the retina (called retinal pigment epithelium) from embryonic stem […]