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 the treatment of Duchenne Muscular Dystrophy (DMD), a genetic disorder characterized by progressive muscle degeneration and weakness. It is designed to deliver a functional version of the dystrophin gene to muscle cells. RGX-202 is currently in Phase 2 clinical development.
RGX-202 targets the dystrophin protein, which is deficient or defective in patients with Duchenne Muscular Dystrophy. By delivering a functional dystrophin gene, the therapy aims to restore dystrophin production in muscle cells, potentially slowing or halting disease progression. This gene therapy approach is designed to address the underlying genetic cause of DMD.
RGX-202 is being developed by REGENXBIO Inc., a biotechnology company focused on gene therapies. REGENXBIO is publicly traded on the Nasdaq under the ticker symbol RGNX. The company is conducting clinical trials to evaluate the safety and efficacy of RGX-202 in patients with Duchenne Muscular Dystrophy.
RGX-202 is currently in Phase 2 clinical development. It is an investigational gene therapy and has not yet been approved by the FDA. The drug has received several FDA designations, including Accelerated Approval, Fast Track, Orphan Drug, and Rare Pediatric Disease designations, reflecting its potential to address a serious unmet medical need.
RGX-202 is being evaluated in the AFFINITY DUCHENNE trial (NCT05693142), a Phase 2 study in participants with Duchenne Muscular Dystrophy. This active, non-recruiting trial has an enrollment of 65 participants and is being conducted in the United States and Canada. The study includes male participants aged 1 year and older.
No, RGX-202 is the investigational gene therapy, while AFFINITY DUCHENNE is the name of the clinical trial evaluating it. The trial, identified as NCT05693142, is a Phase 2 study assessing the safety and efficacy of RGX-202 in participants with Duchenne Muscular Dystrophy. The trial is active but not currently recruiting participants.