REGENXBIO Completes Enrollment in Pivotal Duchenne Gene Therapy Trial and Produces First Commercial Batches of RGX-202

REGENXBIO Completes Enrollment in Pivotal Duchenne Gene Therapy Trial and Produces First Commercial Batches of RGX-202

By, Admin 1 November 2025

REGENXBIO Inc. has reached two major milestones in its Duchenne muscular dystrophy (DMD) program — completing enrollment in the pivotal AFFINITY DUCHENNE trial and successfully manufacturing the first batches of RGX-202 intended for commercial use.

A Step Closer to Transformative Gene Therapy for Duchenne

RGX-202 is an investigational gene therapy designed to deliver durable benefits for patients with Duchenne muscular dystrophy — a rare, progressive, and life-limiting muscle disease.

The company’s goal: to bring a best-in-class treatment that meaningfully alters the disease course and expands long-term outcomes for patients with limited options.

“Completing this pivotal trial milestone and producing our first commercial batches bring us closer to delivering RGX-202 to patients who need it most,”
— Curran Simpson, President and CEO, REGENXBIO

Inside the AFFINITY DUCHENNE Trial

  • Design: Multicenter, open-label Phase I/II/III study
  • Enrollment: 30 participants completed in October 2025
  • Primary Endpoint: Proportion of participants with ≥10% RGX-202 microdystrophin expression at Week 12
  • Secondary Endpoints: Changes in timed function tests (ages ≥4), PDMS-3 and SV95C (ages 1–4)

The trial continues enrolling ambulatory participants aged 1 year and older in the confirmatory phase. Topline data are expected in early Q2 2026.

Promising Early Results

In the Phase I/II portion:

  • Microdystrophin levels ranged from 20% to 122% of normal.
  • No serious adverse events were reported.
  • Participants showed consistent functional improvement versus external controls.

Scaling for Commercial Launch

REGENXBIO has manufactured the first commercial-ready batches of RGX-202 at its Manufacturing Innovation Center in Rockville, Maryland.

Using its proprietary NAVXpress® suspension-based process, the company achieved industry-leading product purity (>80% full capsids) — the highest among Duchenne gene therapies.

This platform enables the production of up to 2,500 doses per year, supporting a potential commercial launch in 2027.

What Makes RGX-202 Different

  • Unique microdystrophin construct encoding key regions of natural dystrophin, including the C-Terminal (CT) domain
  • Codon optimization for improved gene expression and reduced immunogenicity
  • Targeted delivery via the NAV AAV8 vector and muscle-specific Spc5-12 promoter
  • High-yield, commercial-ready manufacturing process (NAVXpress®)

Together, these features position RGX-202 as a potentially best-in-class gene therapy capable of durable, safe, and scalable impact in Duchenne.

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