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Clinical Trial Report

Gene Therapy Trial Report

Summary

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

NCTID NCT05541627 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Huntington's Disease
Disease Ontology Term DOID:12858
Compound Name AB-1001
Compound Alias BV-101
Compound Description AAVrh10.CAG.hCYP46A1
Sponsor Brainvectis, a subsidiary of Asklepios BioPharmaceutical, Inc. (AskBio)
Funder Type Industry
Recruitment Status
Active not recruiting
Enrollment Count 5 (ACTUAL)
Results Posted Not Available

Therapy Information

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 none
Dose 1 4E8 vg/uL
Dose 2 1.1E9 vg/uL
Dose 3
Dose 4
Dose 5

Study Record Dates

Current Stage Phase1, Phase2
Submit Date 2022-09-13
Completion Date 2028-04
Last Update 2025-09-18

Participation Criteria

Eligible Age 18 Years - 65 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL

Locations

No.of Trial Sites 1
Locations France

Regulatory Information

Has US IND False
FDA Designations
Recent Updates Bayer announced the discontinuation of this program November 2024

Resources/Links

Clinical Publications
News and Press Releases
Preclinical Publications
Protocol