A Gene Transfer Study to Evaluate the Safety, Tolerability and Efficacy of SRP-6004 in Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2B/R2 (LGMD2B/R2, Dysferlin [DYSF] Related)

NCTID	NCT05906251 (View at clinicaltrials.gov)
Description	The primary purpose of this study is to evaluate the safety of SRP-6004 administered by intravenous (IV) infusion in ambulatory participants with LGMD2B/R2 (DYSF related). (Show More)
Indication	Limb-Girdle Muscular Dystrophy Type 2B/R2
Compound Name	SRP-6004 (rAAVrh74-MHCK7-DYSF)
Sponsor	Sarepta Therapeutics, Inc.
Funder Type	Industry
Status	Active not recruiting
Enrollment Count	2

Target Gene/Variant	DYSF
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
Dose 1	2 x 10^12 vg
Dose 2	6 x 10^12 vg
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	United States

Has US IND	True
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