Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
RGX-202 · 1 trial · 1 indication
Evaluate incidences of AEs and SAEs
Proportion of participants whose RGX-202 microdystrophin protein expression determined in their muscle biopsy is ≥ 10% relative to dystrophin level in non-DMD participants
| Arm | Type | Description |
|---|---|---|
| Part 1: Cohort 1 and 1b (enrollment complete): RGX-202 Dose 1 | EXPERIMENTAL | A single IV infusion of RGX-202 at a dose of 1×10\^14 GC/kg body weight |
| Part 1: Cohort 2, 2c;, and Part 2; and Part 3 (enrollment complete for all): RGX-202 Dose 2 | EXPERIMENTAL | A single IV infusion of RGX-202 at a dose of 2x10\^14 GC/kg body weight (or a fixed dose of 1.00×10\^16 GC \[150 mL\] for participants weighing \> 50 kg) |
| Name | Type | Description |
|---|---|---|
| RGX-202 | GENETIC | RGX-202 is a recombinant AAV8 containing a transgene encoding a novel microdystrophin |
Part 1 - Key Inclusion Criteria: * The participant's legal guardian(s) is (are) willing and able to provide written, signed informed consent prior to any study-related procedures; and, where applicable, the minor participant has provided written or verbal assent according to local requirements. * I...
RGX-202 is an investigational gene therapy being developed for Duchenne Muscular Dystrophy (DMD), a genetic disorder characterized by progressive muscle degeneration. It is currently in Phase 2 clinical development and has not been approved by the FDA.
RGX-202 targets dystrophin, a protein that is deficient or absent in patients with Duchenne Muscular Dystrophy. By delivering a functional version of the dystrophin gene, the therapy aims to restore production of this essential protein in muscle cells.
RGX-202 is being developed by REGENXBIO Inc., a biotechnology company traded on the Nasdaq under the ticker symbol RGNX. The company is conducting clinical trials to evaluate the safety and efficacy of this gene therapy for Duchenne Muscular Dystrophy.
RGX-202 is currently in Phase 2 clinical development. It is an investigational therapy and has not received FDA approval. The drug has been granted several FDA designations, including accelerated approval, fast track, orphan drug, and rare pediatric disease designations.
RGX-202 is being evaluated in a Phase 2 clinical trial with the identifier NCT05693142, titled 'AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)'. This active, non-recruiting study is enrolling 65 male participants aged 1 year and older in the United States and Canada.
AFFINITY DUCHENNE is the name of the clinical trial evaluating RGX-202, not an alternative name for the drug itself. The trial, identified as NCT05693142, is a Phase 2 study assessing the safety and efficacy of RGX-202 in participants with Duchenne Muscular Dystrophy.