REGENXBIO Achieves Significant Milestones in Q3 2025 Financial Performance and Gene Therapy Pipeline

REGENXBIO Reports Third Quarter 2025 Highlights

REGENXBIO Inc. has made significant strides in the third quarter of 2025, demonstrating strong progress in its financial performance and advancements in its innovative gene therapy pipeline. The company has been at the forefront of gene therapy technology since its inception in 2009, focusing on curative solutions for severe genetic disorders and neurodegenerative diseases.

Financial Performance Overview

As of September 30, 2025, REGENXBIO reported a cash position of $302.0 million, highlighting an increase attributed to critical partnerships and monetization agreements. The revenue for the third quarter reached $29.7 million, an improvement from $24.2 million during the same period last year. This growth was primarily fueled by revenue generated from the company’s collaboration with Nippon Shinyaku, which contributed $5.9 million in development service revenue during the quarter.

Despite this encouraging growth, REGENXBIO reported a net loss of $61.9 million for the third quarter. This reflects the company's continued investments in research and development which totaled $56.1 million, up from $54.4 million year-over-year. The increase was driven by heightened personnel costs and escalating manufacturing expenses.

Gene Therapy Advancements

REGENXBIO's flagship program, RGX-202, targets Duchenne muscular dystrophy and is designed to deliver a potentially transformative treatment for patients suffering from this condition. The pivotal trial enrollment for RGX-202, which is aimed at assessing the efficacy of the therapy, was completed successfully in October, with 30 participants enrolled. This trial focuses on ambulatory patients aged one year and older across the U.S. and Canada.

Results anticipated in early Q2 2026 are eagerly awaited, as they could play a crucial role in supporting REGENXBIO's submission of a Biologics License Application (BLA) by mid-2026. The promising data from RGX-202 suggest improved functional outcomes for patients, indicating that the treatment may effectively address the underlying cause of Duchenne muscular dystrophy.

In addition, REGENXBIO is also advancing clemidsogene lanparvovec (RGX-121), a potential first-in-class gene therapy for Hunter syndrome, in partnership with Nippon Shinyaku. The upcoming FDA approval decision is due on February 8, 2026, and positive outcomes could accelerate the therapy's market entry.

Furthermore, REGENXBIO is collaborating with AbbVie on surabgene lomparvovec (sura-vec), which is aimed at wet age-related macular degeneration (AMD) and diabetic retinopathy. Over 1,200 participants have been enrolled in pivotal studies for these indications, with anticipated topline results available in late 2026.

Future Outlook

Looking ahead, REGENXBIO is focused on maintaining momentum across its various programs while solidifying its financial health. The company’s in-house manufacturing capabilities are poised to support both clinical development and potential commercialization. With the successful completion of pivotal trials and anticipated regulatory milestones, REGENXBIO is on track to expand its impact in the gene therapy space dramatically.

Curran M. Simpson, President and CEO of REGENXBIO, expressed optimism about the company’s ability to leverage its differentiated capabilities. 'Our strong momentum is driven by our commitment to improving the lives of patients with Duchenne muscular dystrophy, Hunter syndrome, and other conditions. With our advanced technology platform, we remain well-positioned to transition into a commercial company in early 2026,' he stated.

As REGENXBIO navigates the complexities of biotech development, the coming months will be pivotal in determining the future success of its innovative therapies, benefiting patients and caregivers alike.