REGENXBIO announced the completion of enrollment in the AFFINITY DUCHENNE pivotal trial of RGX-202, an investigational gene therapy for the treatment of Duchenne muscular dystrophy, as well as the successful production of the first batches intended for commercial supply. Read More: NCT05693142.
“The Duchenne community urgently needs new treatment options that provide durable, safe outcomes and can meaningfully change the course of this degenerative disease. Completing this pivotal trial milestone and manufacturing in-house our first doses intended for commercial use bring us even closer to delivering RGX-202 as a potential best-in-class gene therapy for Duchenne patients with limited options,” said Curran Simpson, President and Chief Executive Officer, REGENXBIO. “The differentiated therapeutic approach behind RGX-202, including our industry-leading product purity levels and novel construct with the C-Terminal domain, has resulted in the positive safety and efficacy profile, with consistent functional benefit seen in Phase I/II. With these highly encouraging results, we are committed to expanding our commercial supply and sharing topline pivotal data in early Q2 of next year.”
Learn More: RGX-202 Gene Therapy Phase 3 Clinical Trial Begins
Key Results about RGX-202
- Patients treated with RGX-202 demonstrate consistent, robust microdystrophin expression and functional improvement compared to natural history in Phase I/II portion of AFFINITY DUCHENNE trial supporting potential approval via the accelerated approval pathway
- REGENXBIO continues to enroll patients in the confirmatory trial
- First batches intended for commercial supply manufactured at in-house Manufacturing Innovation Center
- Capacity to produce up to 2,500 RGX-202 doses per year
- Topline pivotal data now expected in early Q2 2026 and BLA submission in mid-2026
RGX-202 Mechanism of Action
Read More: Clinical Trials for Duchenne (List of All Researches)
