Disease Tab: Other
A Phase 2 Study Evaluating the Efficacy and Safety of IV Administered rAAV9 Vector Containing RP-A501 in Male Patients with Danon Disease
A Phase 1 Study of FT819 in Participants with Moderate to Severe Active Systemic Lupus Erythematosus
A phase 1/2 study to evaluate a bispecific CD19/CD20-directed CAR T cell, in refractory lupus nephritis and systemic lupus erythematosus
Personalized antisense oligonucleotide therapy for rare pediatric genetic disease: SCN2A
A Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Patients with Mucopolysaccharidosis Type 1
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 […]
Phase I Treatment of Urethral Strictures in Humans
When a scar, or stricture, forms along the urethra it impedes the flow of urine and causes other complications. James Yoo, M.D., Ph.D., and his team at Wake Forest University Health Sciences will use epithelial and smooth muscle cells, taken from the patient’s bladder, and layer them on to a synthetic tubular scaffold. The tube […]
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 […]
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 […]