Stage of Program: Clinical Trial, Phase 1/2
Phase 1/2 study for autologous human CD34+ hematopoietic stem cells ex vivo transduced with pCCL-CTNS lentiviral vector for treatment of Cystinosis.
Cystinosis is a rare disease that primarily affects children and young adults, and leads to premature death, usually in early adulthood. Patients inherit defective copies of a gene called CTNS, which results in abnormal accumulation of an amino acid called cystine in all cells of the body. This buildup of cystine can lead to multi-organ […]
A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I
Leukocyte Adhesion Deficiency-I (LAD-I) is a rare pediatric disease caused by a mutation in a specific gene that affects the body’s ability to combat infections. As a result, infants with severe LAD-I are often affected immediately after birth. During infancy, they suffer from recurrent life-threatening bacterial and fungal infections that respond poorly to antibiotics and […]
A Phase 1/2 Study to Assess the Safety, Tolerability, and Efficacy of ST-400 Autologous HSPC Transplant in Transfusion-dependent β-Thalassemia
Sangamo, Inc. is testing genetically engineered blood stem cells for the treatment of beta-thalassemia, a severe form of anemia caused by mutations in the hemoglobin gene. This genetic disorder requires life-long blood transfusions and carries a life expectancy of only 30-50 years. The Sangamo therapy takes a patient’s own blood stem cells and, using a […]
Trial of AB-205 in Adults With Lymphoma Undergoing High-Dose Therapy and Autologous Stem Cell Transplantation
Angiocrine Bioscience Inc. will use genetically engineered cells, derived from cord blood, to see if they can help alleviate or accelerate recovery from the toxic side effects of chemotherapy for people undergoing treatment for lymphoma and other aggressive cancers of the blood or lymph system.
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. […]
Pulmonary Arterial Hypertension Treated with Cardiosphere-Derived Allogeneic Stem Cells
Pulmonary arterial hypertension (PAH) is a progressive condition with no cure. Scientists at Cedars-Sinai Medical Center are using donor cells derived from the heart to reduce two hallmark symptoms of pulmonary hypertension: inflammation and high blood pressure in the blood vessels within the lungs. These conditions make the heart struggle to pump blood to the heart […]
A Phase 1b/2 Trial of the Anti-CD47 Antibody Hu5F9-G4 in Combination with Cetuximab in Patients with Solid Tumors and Advanced Colorectal Cancer
Forty Seven, Inc. has developed an antibody therapy to block a protein called CD47 that is found on the surface of cancer cells. CD47 acts as a ‘don’t eat me’ signal that tells immune cells not to eliminate the cancer cells. When this ‘don’t eat me’ signal is blocked by the antibody, the cancer cells […]
Treatment of non-traumatic osteonecrosis with endogenous Mesenchymal stem cells
Femoral head osteonecrosis (aka avascular necrosis) is a disease caused by loss of blood supply to the bone, leading to bone cell death, end stage hip arthritis and total hip replacement. There is an unmet need for treatment of this disease, that affects individuals at prime of life (peak age 35 years). This small molecule […]
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 […]