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…
Phase 1 Clinical Development of IO-202, A First-in-Class Antibody Targeting LILRB4, for the Treatment of AML with Monocytic Differentiation and CMML
Immune-Onc Therapeutics will conduct a clinical trial for patients with acute myeloid leukemia (AML) and chronic myelomonocytic leukemia (CMML), both of which are types of blood cancer. AML affects approximately 20,000…
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…
Phase 2 Study of Hematopoietic Stem Cell Gene Transfer Inducing Fetal Hemoglobin in Sickle Cell Disease
Boston Children's Hospital is conducting a gene therapy clinical trial for sickle cell disease (SCD). This project is part of an agreement between CIRM and the National Heart, Lung, and…
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…
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…
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…
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…
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…
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