Evaluation of the Safety, Tolerability and Efficacy of Gene Therapy Drug for Late Onset Pompe Disease (LOPD)

NCTID	NCT06178432 (View at clinicaltrials.gov)
Description	This is a single-center, single-arm, open-label, single-dose treatment clinical study to evaluate the safety, tolerability and efficacy of CRG003 injection in participants with late onset Pompe disease (LOPD), with a long-term follow-up period of 5 years. CRG003 (BBM-G102) injection is an adeno-associated virus (AAV) gene therapy product for treating Pompe disease to stably express active GAA enzyme in the liver on a long-term basis after the injection. (Show More)
Development Status	Active
Indication	Pompe Disease (Late-onset)
Disease Ontology Term	DOID:2752
Compound Name	BBM-G102
Sponsor	Huashan Hospital
Funder Type	Other
Recruitment Status	Not yet recruiting
Enrollment Count	6
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	AAV
Editor Type	none
Dose 1	Undisclosed dose 1
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	China

Has US IND	False
FDA Designations
Recent Updates	This investigator-initiated trial is funded by Belief-Delivery BioMed Co.

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