A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4

NCTID	NCT06246513 (View at clinicaltrials.gov)
Description	This is a multicenter, global study of the effects of a single systemic dose of SRP-9003 on beta-sarcoglycan (β-SG) gene expression in participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4). This study will consist of both ambulatory participants (Cohort 1) and non-ambulatory participants (Cohort 2). (Show More)
Development Status	Active
Indication	Limb-Girdle Muscular Dystrophy, Type 2E/R4
Disease Ontology Term	DOID:0110279
Compound Name	SRP-9003
Compound Alias	Bidridistrogene xeboparvovec, MYO-101
Compound Description	AAVrh74-MHCK7-SGCB
Sponsor	Sarepta Therapeutics, Inc.
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	17
Results Posted	Not Available

Target Gene/Variant	SGCB
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	AAVrh74
Editor Type	none
Dose 1	1.85E13 vg/kg (n=3)
Dose 2	5E13 vg/kg
Dose 3	7.41E13 vg/kg (n=3)
Dose 4
Dose 5

No.of Trial Sites	5
Locations	United States,United Kingdom

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates	Data from EMERGENE are expected in the first half of 2025; BLA submission anticipated in 2025