Product Description
RGX-202 is designed to deliver a transgene for a novel microdystrophin that includes the functional elements of the C-Terminal (CT) domain found in naturally occurring dystrophin. Presence of the CT domain has been shown in preclinical studies to recruit several key proteins to the muscle cell membrane, leading to improved muscle resistance to contraction-induced muscle damage in dystrophic mice. Additional design features, including codon optimization and reduction of CpG content, may potentially improve gene expression, increase translational efficiency and reduce immunogenicity. (Sourced from: https://www.regenxbio.com/therapeutic-programs/rgx-202/)
Mechanisms of Action: Gene Therapy,DMD
Novel Mechanism: No
Modality: Gene Therapy
Route of Administration: Injection
FDA Designation: Fast Track - Muscular Dystrophies|Muscular Dystrophy, DuchenneOrphan Drug - Muscular Dystrophies|Muscular Dystrophy, Duchenne *
Approval Status: Not Approved
Approved Countries: None
Approved Indications: None
Known Adverse Events: None
Company: Merck KGaA
Company Location: GERMANY HRB 6164 I8
Company CEO: Belen Garijo
Additonal Commercial Interests: Inspirna
Clinical Description
Countries in Clinic: United States
Active Clinical Trial Count: 1
Highest Development Phases
Phase 2: Muscular Dystrophy, Duchenne
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
|---|---|---|---|---|---|
| AFFINITY DUCHENNE | P2 |
Recruiting |
Muscular Dystrophy, Duchenne |
2025-12-01 |