Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients

PHASE1/PHASE2RecruitingINTERVENTIONAL

Enrollment

Participants

Timeline

Start Date

May 10, 2024

Primary Completion Date

December 31, 2026

Study Completion Date

December 31, 2028

Conditions

Spinal Muscular Atrophy Type 3

Interventions

GENETIC

GC101

Self-complementary AAV9 carrying a codon-optimized SMN coding sequence(coSMN1) driven by CMV enhancer and chicken β-actin promoter

Trial Locations (1)

RECRUITING

Beijing Tiantan Hospital, Capital Medical University, Beijing

All Listed Sponsors

lead

GeneCradle Inc

INDUSTRY