Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug

PHASE1/PHASE2RecruitingINTERVENTIONAL

Enrollment

Participants

Timeline

Start Date

April 19, 2024

Primary Completion Date

December 31, 2026

Study Completion Date

December 31, 2026

Conditions

Pompe Disease (Late-onset)

Interventions

GENETIC

GC301

GC301, is an adeno-associated virus 9 (AAV9) vector delivering a functional copy of the human GAA gene

Trial Locations (1)

RECRUITING

Chinese PLA General Hospital, Beijing

All Listed Sponsors

lead

GeneCradle Inc

INDUSTRY