Stage of Program: Clinical Stage Programs (2.0 and relevant 1.0 projects)


Genetically Engineered PBMC and PBSC Expressing NY-ESO-1 TCR After a Myeloablative Conditioning Regimen to Treat Patients With Advanced Cancer (NYESO SCT)

This is a treatment for patients with sarcomas and other advanced solid tumors. In 2018 alone, an estimated 13,040 people were diagnosed with soft tissue sarcoma (STS) in the United States, with approximately 5,150 deaths.  Standard of care treatment for sarcomas typically consists of surgery, radiation, and chemotherapy, but patients with late stage or recurring […]

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 […]

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 Phase I Study of Chimeric Antigen Receptor Engineered Stem/Memory T Cells for the Treatment of HER2-Positive Brain Metastases

Breast cancer is the second-most common cancer in women, both in the United States (US) and worldwide.  Some types of breast cancer have a high likelihood of metastasizing to the brain, meaning there are tumors in the brain that have spread from the original site of the breast cancer  When that happens, there are few treatment […]

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 […]

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.

Induction of Tolerance by Combinatorial Therapy w/ Donor Stem Cells and Expanded Recipient Treg cells in HLA-mismatched Kidney Transplant Recipients

Dr. Meyer, and his team at Stanford University, will use a combination of healthy donor stem cells and the patient’s own regulatory T cells (Tregs), to train the patient’s immune system to accept the transplanted kidney and eliminate the need for immunosuppressive drugs. The initial group targeted in this clinical trial are people with what […]

Phase 1 Study of CD19/CD22 Chimeric Antigen Receptor (CAR) T Cells in Adults with Recurrent or Refractory B Cell Malignancies

Chimeric Antigen Receptor (CAR) T Cell Therapy is an innovative cancer therapy with very encouraging response rates in patients. The therapy works by isolating a patient’s own T cells (a type of immune cell) and then genetically engineering them to recognize a protein on the surface of cancer cells, triggering their destruction. In some patients […]

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 […]

A phase I trial of intratumoral administration of CCL21-gene modified dendritic cell (DC) combined with intravenous pembrolizumab for advanced NSCLC

The five-year survival rate for people diagnosed with the most advanced stage of non-small cell lung cancer (NSCLC) is between one and 10 percent. To address this devastating condition, UCLA researchers are genetically modifying a patient’s own dendritic cells – key cells of the immune system – to boost their ability to stimulate native T cells – a type […]