AAV2/8-LSPhGAA (ACTUS-101) in Late-Onset Pompe Disease

NCTID	NCT03533673 (View at clinicaltrials.gov)
Description	Open-label, ascending dose trial of ACTUS-101 administered intravenously. (Show More)
Indication	Glycogen Storage Disease Type 2 (Late-Onset Pompe Disease)
Compound Name	AAV2/8-LSPhGAA (ACTUS-101)
Sponsor	Asklepios Biopharmaceutical, Inc.
Funder Type	Industry
Status	Active not recruiting
Enrollment Count	7

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	Hepatocyte
Delivery System	Viral transduction
Vector Type	AAV2/8
Editor Type
Dose 1	1.6 x 10^12 vg/kg
Dose 2	Undisclosed dose 2
Dose 3	Undisclosed dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	United States

Has US IND	True
Recent Updates	First patient dosed 1/22/19