Stage of Program: Clinical Trial, Phase 1/2


A Double-Blind Randomized Placebo-Controlled Investigation of Autologous Muscle Derived Progenitor Cells for the Treatment of Dysphagia

Patients with head and neck cancer often undergo surgery and/or radiation to remove the tumors. As a result, they may develop problems swallowing and this can lead to serious complications such as malnutrition, dehydration, social isolation, or a dependence on using a feeding tube. Patients may also inhale food or liquids into their lungs causing […]

A phase I/II study of human placental hematopoietic stem cell derived natural killer cells (CYNK-001) for the treatment of adults with COVID-19

This trial will use blood stem cells obtained from the placenta to generate natural killer (NK) cells, a type of white blood cell that is a vital part of the immune system, and administer them to patients with COVID-19.  NK cells play an important role in defense against cancer and in fighting off viral infections.  […]

Anti-HIV duoCAR-T cell therapy for HIV infection

The University of California San Francisco (UCSF) is conducting a clinical trial that modifies a patient’s own immune cells in order to treat and potentially cure HIV.  Current treatment of HIV involves the use of long-term antiretroviral therapy (ART).  However, many people are not able to access and adhere to long-term ART. The team will […]

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 […]

Pancreatic Islet and PARAthyroid Co-Transplantation for Treatment of Diabetes in IntraMuscular Site: PARADIGM

Transplantation of beta cells, contained in donor pancreatic islets, can reverse the symptoms of diabetes.  However, due to a poor islet survival rate, transplants require islets from multiple donors.  Since islet cells are transplanted directly into the vessels that enter the liver, it is extremely difficult to monitor and retrieve these cells should the need […]

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.

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 […]

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 […]