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) (Show More)
Development Status	Active
Indication	Limb-Girdle Muscular Dystrophy, Type 2I/R9
Disease Ontology Term	DOID:0110299
Compound Name	AB-1003
Compound Alias	LION-101
Sponsor	AskBio Inc
Funder Type	Industry
Recruitment Status	Recruiting
Enrollment Count	10
Results Posted	Not Available

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

No.of Trial Sites	6
Locations	United States

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates	First patient dosed (8/3/23)