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. (Show More)
Indication	Duchenne Muscular Dystrophy
Compound Name	RGX-202 (AAV8-microdystrophin)
Sponsor	REGENXBIO Inc.
Funder Type	Industry
Status	Recruiting
Enrollment Count	65

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	1 x 10^14 GC/kg body weight
Dose 2	2 x 10^14 GC/kg body weight
Dose 3
Dose 4
Dose 5

No.of Trial Sites	5
Locations	United States

Has US IND	True
Recent Updates	Pivotal trial initiation expected Q3-Q4 2024; EOP2 meeting with FDA in July 2024