Stage of Program: Clinical Trial, Phase 1
The CuRe Trial: Cellular Therapy for In Utero Myelomeningocele Repair and The CuRe Trial: Cellular Therapy for In Utero Repair of Myelomeningocele
UC Davis is conducting a clinical trial for in utero repair of myelomeningocele (MMC), the most severe form of spina bifida. MMC is a birth defect that occurs due to incomplete closure of the developing spinal cord, resulting in neurological damage to the exposed cord. This damage leads to lifelong lower body paralysis, and bladder and […]
Phase I Study of IL13Rα2-Targeting CAR T Cells After Lymphodepletion for Children with Refractory or Recurrent Malignant Brain Tumors
City of Hope will conduct a clinical trial for children with malignant brain tumors. Brain tumors are the most common solid tumor of childhood, with roughly 5,000 new diagnoses per year in the United States. The team will treat pediatric patients with aggressive brain tumors using chimeric antigen receptor (CAR) T cell therapy. The CAR […]
A Phase I Clinical Trial for a Lentiviral Gene Therapy Targeting the TCIRG1 Gene for Infantile Malignant Osteopetrosis (IMO)
Rocket Pharmaceuticals is conducting a clinical trial using a gene therapy for infantile malignant osteopetrosis (IMO), a rare and life-threatening disorder that develops in infancy. IMO is caused by defective bone cell function, which results in blindness, deafness, bone marrow failure, and death very early in life. The trial will use a gene therapy that targets IMO caused […]
Evaluation and Characterization of SARS-CoV-2 Antibody in Convalescent Volunteer Plasma Donors for Potential Therapeutic Use
Plasma is a component of blood that carries proteins called antibodies that are usually involved in defending our bodies against viral infections. Blood plasma from patients that have recovered from COVID-19, referred to as convalescent plasma, contain antibodies against the virus that can be used as a potential treatment for COVID-19.
A Phase 1 Study of ECT-001 Expanded Cord Blood and Myeloablative Regimen with Reduced Toxicity in Patients with Severe Sickle Cell Disease
Sickle Cell Disease (SCD) is an inherited blood disorder caused by a single gene mutation that results in the production of “sickle” shaped red blood cells. It affects an estimated 100,000 people, mostly African American, in the US and can lead to multiple organ damage as well as reduced quality of life and life expectancy. […]
A Phase 1b Safety Study for MRI guided delivery of AAV2-GDNF for the treatment of Parkinson’s disease
Parkinson’s Disease is a neurodegenerative disease that is thought to be the outcome of the gradual breakdown of dopaminergic neurons in the brain, which are a type of cell that produces a chemical in ones’ brain known as dopamine. This decrease in the brain dopamine content can result in symptoms such as uncontrollable shaking of […]
Safety and Feasibility of Cultivated Autologous Limbal Stem Cells for Limbal Stem Cell Deficiency
Limbal stem cell deficiency (LSCD) is a blinding corneal disease. LSCD is caused by a decrease in the number and/or function of limbal stem cells (LSCs), a type of stem cell that is needed to continuously regenerate tissue of the cornea, the clear front surface of the eye that refracts light entering the eye and is […]
Clinical Study to Assess Safety and Efficacy of Subretinal Injection of Human Neural Progenitor Cells for Treatment of Retinitis Pigmentosa
Retinitis pigmentosa is a blinding eye disease that affects approximately 150,000 individuals in the US and 1.5 million people around the world. It is caused by the destruction of light-sensing cells in the back of the eye known as photoreceptors. This leads to gradual vision loss and eventually blindness. For this trial, human neural progenitor […]
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 […]
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 […]