Stage of Program: Clinical Trial, Phase 1


A Phase I, Pilot Study of Human Embryonic Stem Cell-Derived Cardiomyocytes in PaTients with ChrOnic Ischemic Left VentRicular Dysfunction (HECTOR)

Dr. Wu and his team at Stanford University will use hESCs to generate cardiomyocytes (CM), a type of cell that makes up the heart muscle.  The newly created hESC-CMs will then be administered to patients at the site of the heart muscle damage in a first-in-human trial.  This initial trial will evaluate the safety and feasibility of […]

Phase 1 Study of Autologous CD4LVFOXP3 in Participants with IPEX Syndrome

IPEX syndrome is a rare condition where the body can’t control or restrain an immune response, so the person’s immune cells attack their own healthy tissue. The syndrome mostly affects boys, is diagnosed in the first year of life and is often fatal. Children born with IPEX syndrome have abnormalities in the FOXP3 gene. This […]

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 loss of limb function, difficulty breathing, paralysis, and eventually death.  There are medications that can slow down the progression of ALS, but unfortunately there is […]

Transplantation of CRISPR-CAS9 Corrected Hematopoietic Stem Cells (CRISPR_SCD001) in Patients with Severe Sickle Cell Disease

Safety and Tolerability Study of Neural Stem Cells (NR1) in Subjects with Chronic Ischemic Subcortical Stroke

Gary Steinberg, M.D., Ph.D., and his team at Stanford University are conducting a clinical trial to test a therapy for motor disabilities caused by chronic ischemic stroke.  While “clot busting” therapies can treat strokes immediately after they occur (acute strokes), these treatments can only be given within a few hours of the initial injury.  There […]

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 […]

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 people in the United States each year and has a 5-year survival rate of about 25 percent. Anywhere from 15-30 percent of CMML cases eventually […]

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.