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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)
NCTID
NCT05230459
(View at clinicaltrials.gov)
Description
The purpose of this study is to evaluate the safety and tolerability of a single intravenous infusion of AB-1003 in adults diagnosed with limb girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). Participants will be treated in sequential, dose-level cohorts. (Part 1)
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Indication
Limb Girdle Muscular Dystrophy, Limb-Girdle Muscular Dystrophy Type 2, LGMD2I, Muscular Dystrophy, LGMD2, LGMD, FKRP, FKRP Mutation, Fukutin Related Protein
Compound Name
AB-1003 (LION-101)
Sponsor
AskBio Inc
Funder Type
Industry
Status
Recruiting
Enrollment Count
10
Therapy Information
Target Gene/Variant
FKRP
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
AAV9
Editor Type
none
Dose 1
Undisclosed dose 1
Dose 2
Undisclosed dose 2
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2022-01-27
Completion Date
2028-12
Last Update
2024-11-29
Participation Criteria
Eligible Age
18 Years - 65 Years
Standard Ages
Adult, Older adult
Eligible Sex
ALL
Locations
No.of Trial Sites
6
Locations
United States
Regulatory Information
Has US IND
True
Recent Updates
First patient dosed (8/3/23)
Resources/Links
News and Press Releases
AskBio Receives FDA Rare Pediatric Disease and Orphan-Drug Designations for AB-1003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2I/R9
Preclinical Publications
Improved efficacy of FKRP AAV gene therapy by combination with ribitol treatment for LGMD2I
Metabolomics Analysis of Skeletal Muscles from FKRP-Deficient Mice Indicates Improvement After Gene Replacement Therapy
Efficacy of Gene Therapy Is Dependent on Disease Progression in Dystrophic Mice with Mutations in the FKRP Gene
Adeno-associated virus 9 mediated FKRP gene therapy restores functional glycosylation of α-dystroglycan and improves muscle functions