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NCT06888661

An Open-label, Dose-escalation Clinical Trial to Assess the Safety and Efficacy of EXG001-307 After Intrathecal Injection in Patients With Spinal Muscular Atrophy

Study Summary

The purpose of this trial is to evaluate safety and efficacy of intrathecal delivery of EXG001-307 as a treatment of spinal muscular atrophy .

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Interventions

EXG001-307 injectionBIOLOGICAL
non-replicating, rAAV vector based on AAV9 containing cDNA encoding the human SMN protein.

Study Locations

FacilityCityStateCountry
Shanghai Children's Medical Center Affiliated to Shanghai Jiao Tong University School of MedicineShanghaiShanghai MunicipalityChina
The Children's Hospital of Fudan UniversityShanghaiShanghai MunicipalityChina

Official Trial Information

View on ClinicalTrials.gov

Data sourced from ClinicalTrials.gov. Last updated: April 14, 2026