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REGENXBIO Reports Financial Results for Q3 2026 and Operational Highlights

On November 5, 2026, REGENXBIO Inc. (Nasdaq: RGNX) disclosed its financial and operational developments for the third quarter ending on September 30, 2026. During this quarter, the company continued to pave the way for innovative gene therapies that hold promise for various medical conditions.

Leadership's Outlook

Curran Simpson, the President and CEO of REGENXBIO, expressed optimism regarding the company's trajectory, stating, _“We are continuing to build momentum as we prepare to launch potential first- or best-in-class gene therapies in 2027 and 2028.”_ His remarks underline the critical nature of the pivotal data expected soon for the key product, sura-vec, anticipated to significantly enhance treatment options for patients experiencing wet age-related macular degeneration (AMD) and others.

Program Updates and Milestones

Neuromuscular Disease: RGX-202

One of REGENXBIO’s flagship projects is RGX-202, aimed at treating Duchenne muscular dystrophy (Duchenne). This innovative gene therapy seeks to correct the root cause of Duchenne by facilitating the targeted expression of a microdystrophin, closely resembling natural dystrophin. The therapy employs a novel construct and an immune suppression regimen, ensuring product purity. The ongoing clinical trials reflect REGENXBIO's commitment to providing effective treatments while maintaining safety and durability for patients.

The company plans a pre-Biologics License Application (BLA) meeting with the FDA in Q4 2026, aiming for accelerated approval by H2 2027. Additionally, the initiation of the AFFINITY® RISE trial will further support RGX-202’s global regulatory submissions in 1H 2027.

Retinal Diseases: sura-vec

In collaboration with AbbVie, REGENXBIO is also advancing sura-vec, a breakthrough gene therapy targeting wet AMD and diabetic retinopathy (DR). Results from pivotal studies, ATMOSPHERE® and ASCENT®, are expected later this quarter, marking a significant moment in the treatment landscape for retinal diseases. These studies aim to solidify sura-vec’s position as a first-in-class option as global regulatory submissions for this therapy are projected for 2027.

The long-term follow-up from the ALTITUDE® trial showed promising results, demonstrating sustained safety and efficacy in patients receiving sura-vec for DR. The NAAVIGATE trial is currently underway to establish safety and efficacy in non-proliferative DR without associated diabetic macular edema.

Neurodegenerative Disease: RGX-121

Lastly, REGENXBIO is working on RGX-121 for MPS II (Hunter syndrome). Although the FDA recently placed a clinical hold on this project due to MRI findings in participants, the company remains focused on gathering more data and ensuring patient safety before proceeding further.

Financial Overview

Financially, REGENXBIO reported a cash position of $251 million as of September 30, 2026, reflected by a positive milestone payment from AbbVie and proceeds from a public offering. Despite total revenues decreasing to $9.2 million this quarter, largely due to the expiration of patents affecting ZOLGENSMA® royalties, the company reported a net loss of $40.7 million compared to a greater loss in the same period last year. This indicates a move towards financial stabilization as the company advances its pipeline.

The upcoming conference call will provide further insights into these results and what to expect moving forward. Attendees are encouraged to connect 15 minutes early to engage in the discourse surrounding REGENXBIO’s promising future in gene therapy.

Conclusion

With its focused commitment to developing transformative gene therapies, REGENXBIO Inc. is navigating complex challenges while striving to deliver effective treatments for patients with rare diseases. As key milestones loom on the horizon, all eyes are on REGENXBIO as it both inspires hope and navigates the evolving biotechnology landscape.