CNS10-NPC-GDNF delivered into the motor cortex for the treatment of ALS
ALS is a neurodegenerative disease that results in the death of nerve cells in the brain and spinal cord, causing the muscles in the body to gradually weaken, leading to…
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…
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…
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…
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,…
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…
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…
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…
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…
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…
