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)
Indication	Limb-girdle Muscular Dystrophy
Compound Name	Bidridistrogene xeboparvovec/SRP-9003 (AAVrh74-MHCK7-SGCB)
Sponsor	Sarepta Therapeutics, Inc.
Funder Type	Industry
Status	Recruiting
Enrollment Count	15

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	5E13 vg/kg
Dose 2
Dose 3
Dose 4
Dose 5

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

Has US IND	True
Recent Updates	Data from EMERGENE are expected in the first half of 2025; BLA submission anticipated in 2025