A Study to Evaluate AB-1001 Striatal Administration in Adults With Early Manifest Huntington's Disease

NCTID	NCT05541627 (View at clinicaltrials.gov)
Description	A Phase I/II Dose-Finding Study to Evaluate Striatal Administration of AB-1001 (previously BV-101) in Adults with Early Manifest Huntington's Disease (Show More)
Indication	Huntington's Disease, Early Manifest
Compound Name	AB-1001 (AAVrh10.CAG.hCYP46A1)
Sponsor	Brainvectis, a subsidiary of Asklepios BioPharmaceutical, Inc. (AskBio)
Funder Type	Industry
Status	Active not recruiting
Enrollment Count	5

Target Gene/Variant	CYP46A1
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Overexpression of protective allele/gene
Route of Administration	Intraparenchymal
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAVrh10
Editor Type
Dose 1	4 x 10^8 vg/uL
Dose 2	1.1 x 10^9 vg/uL
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	France

Has US IND	False
Recent Updates