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

NCTID	NCT06391736 (View at clinicaltrials.gov)
Description	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 late-onset Pompe disease （LOPD） who are ≥ 6 years old will be studied. (Show More)
Development Status	Active
Indication	Pompe Disease (Late-onset)
Disease Ontology Term	DOID:2752
Compound Name	GC301
Sponsor	GeneCradle Inc
Funder Type	Industry
Recruitment Status	Recruiting (Click here for study contact information)
Enrollment Count	33
Results Posted	Not Available

Target Gene/Variant	GAA
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV9
Editor Type
Dose 1	3.0E13 vg/kg
Dose 2	6.0E13 vg/kg
Dose 3	1.2E14 vg/kg (IIT study)
Dose 4
Dose 5

No.of Trial Sites	1
Locations	China