Evaluating Safety and Efficacy of Autologous Gene-edited Muscle Stem Cells (GenPHSats-bASKet)

Phase 1/2 Unknown 6 enrolled Simone Spuler, MD

NaSequentialOpen-labelOther

Summary

This study is an investigator initiated first-in-human interventional open label phase 1/2a clinical trial investigating an ATMP in the orphan disease LGMD to evaluate safety and efficacy.

Timeline

Start: 2024-07-01
Primary completion: 2025-07-01
Completion: 2025-07-01

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	GenPHSat	Cell therapy	—	Intramuscular

Evaluating Safety and Efficacy of Autologous Gene-edited Muscle Stem Cells (GenPHSats-bASKet)

Summary

Timeline

Drugs

Indications