Clinical Stage Programs (2.0 and relevant 1.0 projects) – Page 21

Therapeutic Opportunities To Target Tumor Initiating Cells in Solid Tumors

Cancer is a major cause of human death worldwide. The vast majority of cancer patients suffer from solid tumors whose growth destroys vital organs. We propose to develop novel therapeutic…

A Phase 1/2a First-in-Human, Dose-Escalation Study to Evaluate TRX319 in Subjects with Progressive Multiple Sclerosis

Autologous iPSC-derived Retinal Pigment Epithelium Cell Therapy to Restore Vision in Blinding Eye Disease

Advancing ISP-001 for Mucopolysaccharidosis I: Clinical Expansion, Clinical Manufacturing and Release Testing

Phase 1/2 Study to Evaluate the Safety, Tolerability, and Efficacy of MZ-1866, a gene therapy for Pitt Hopkins Syndrome

A Phase 1 trial of RNDP-001, a dopaminergic progenitor drug product, in idiopathic Parkinson’s Disease

Phase I/II Clinical Trial for CMT4J

Clinical Development of ETX101, a Gene Regulation Therapy for SCN1A+ Dravet Syndrome

Phase 1B/2A study of the safety and tolerability of human neural stem cells for Huntington’s Disease (REGEN4HD)

Optogenetic Gene Therapy for Treatment of Retinitis Pigmentosa