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

Phase 1/2 Active not recruiting 16 enrolled GeneCradle Inc

Non-randomizedSequentialOpen-labelTreatment

Summary

This study is being conducted to evaluate the safety and effectiveness of GC301 adeno-associated virus vector expressing codon-optimized human acid alpha-glucosidase (GAA) as potential gene therapy for Pompe disease. Patients diagnosed with infantile-onset Pompe disease who are younger than 6 months old will be studied.

Timeline

Start: 2023-06-02
Primary completion: 2025-12
Completion: 2026-12

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	GC301	Gene therapy (AAV / viral vector)	8e+13 vg/kg	Intravenous
Subject	GC301	Gene therapy (AAV / viral vector)	1.2e+14 vg/kg	Intravenous
Subject	GC301	Gene therapy (AAV / viral vector)	1.8e+14 vg/kg	Intravenous

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

Summary

Timeline

Drugs

Indications