Gene Transfer Study in Patients With Late Onset Pompe Disease

NCTID	NCT04174105 (View at clinicaltrials.gov)
Description	This is a phase 1/2 open-label, ascending dose, multicenter clinical study to evaluate the safety and efficacy of AT845 in adult (aged ≥ 18 years) subjects, ambulatory or nonambulatory, with Late Onset Pompe Disease (LOPD). (Show More)
Indication	Glycogen Storage Disease Type 2 (Late-Onset Pompe Disease)
Compound Name	Zocaglusagene nuzaparvovec (AT845)
Sponsor	Astellas Gene Therapies
Funder Type	Industry
Status	Recruiting
Enrollment Count	18

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	AAV8
Editor Type
Dose 1	3 x 10^13 vg/kg
Dose 2	6 x 10^13 vg/kg
Dose 3	1 x 10^14 vg/kg
Dose 4
Dose 5

No.of Trial Sites	4
Locations	United States,United Kingdom

Has US IND	True
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