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

EARLY_PHASE1Enrolling by invitationINTERVENTIONAL

Enrollment

Participants

Timeline

Start Date

March 10, 2025

Primary Completion Date

March 31, 2026

Study Completion Date

December 30, 2026

Conditions

Spinal Muscular Atrophy (SMA)

Interventions

BIOLOGICAL

EXG001-307 injection

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

Trial Locations (2)

Shanghai Children's Medical Center Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai

The Children's Hospital of Fudan University, Shanghai

All Listed Sponsors

lead

Hangzhou Jiayin Biotech Ltd

INDUSTRY