Stage of Program: Clinical Trial, Phase 1


Evaluation of Safety and Feasibility of Cytomegalovirus-Specific, Anti-HIV Chimeric Antigen Receptor (CMV/HIV-CAR) T Cells in People with HIV

A Phase 1b, Randomized, Blinded, Placebo-Controlled Dose-Ranging Study of GNSC-001 Evaluating Safety, Pharmacodynamics, and Biomarkers in Knee OA

A Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Patients with Mucopolysaccharidosis Type 1

TACH101: A Potent, First-In-Class KDM4 Inhibitor for Advanced Cancers

Phase 1 Clinical research program for functional cure of HIV with EBT-101, in-vivo gene therapy

A Phase I Study of Multiple doses of NSC-Based Oncolytic Virotherapy Administered Intracerebrally to Patients with Recurrent High-Grade Gliomas

FIH Study of NRTX-1001 Neural Cell Therapy in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy

The current therapies for drug-resistant epilepsy are only partially effective and have serious drawbacks.   One treatment that can significantly reduce seizure frequency is the removal of the affected part of the brain using surgical or laser ablation methods.  However, not surprisingly, removal of brain tissue can cause serious, irreversible damage, such as effects on memory, […]

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 […]

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 […]