NCT05614531View on ClinicalTrials.gov

Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients with Spinal Muscular Atrophy Type 1

PHASE1/PHASE2Enrolling by invitationINTERVENTIONAL
Enrollment

12

Participants

Timeline

Start Date

February 16, 2023

Primary Completion Date

August 31, 2025

Study Completion Date

August 31, 2025

Conditions
Spinal Muscular Atrophy Type I
Interventions
GENETIC

EXG001-307 injection

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

Trial Locations (2)

201100

The Children's Hospital of Fudan University, Shanghai

310051

The Children'S Hospital Zhejiang University of Medicine, Hangzhou

All Listed Sponsors
lead

Hangzhou Jiayin Biotech Ltd

INDUSTRY