AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)

NCTID	NCT05693142 (View at clinicaltrials.gov)
Description	RGX-202 is a gene therapy designed to deliver a transgene for a novel microdystrophin that includes functional elements of naturally-occurring dystrophin including the C-Terminal (CT) domain. This is a multicenter, open-label dose evaluation clinical study to assess the safety, tolerability, and clinical efficacy of a one-time intravenous (IV) dose of RGX-202 in participants with Duchenne. For additional information on how to participate (or be considered for the study), please follow this link: https://mytomorro.ws/affinity-ct-gov (Show More)
Development Status	Active
Indication	Duchenne Muscular Dystrophy (DMD)
Disease Ontology Term	DOID:11723
Compound Name	RGX-202
Compound Description	AAV8-microdystrophin
Sponsor	REGENXBIO Inc.
Funder Type	Industry
Recruitment Status	Recruiting (Click here for study contact information)
Enrollment Count	65 (ESTIMATED)
Results Posted	Not Available

Target Gene/Variant	Micro-dystrophin
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Overexpression of protective allele/gene
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV8
Editor Type	none
Dose 1	1E14 GC/kg body weight
Dose 2	2E14 GC/kg body weight (pivotal dose)
Dose 3
Dose 4
Dose 5

No.of Trial Sites	16
Locations	Canada,United States

Has US IND	True
FDA Designations
Recent Updates	Pivotal trial underway; BLA expected 2026