Duchenne Gene Therapy Phase 3 Success... Early Approval 'Green Light' for 2027

REGENXBIO Inc. (NASDAQ: RGNX) announced its financial results for the first quarter of 2026 on the 14th. The company reported that its Duchenne muscular dystrophy candidate RGX-202 met the primary endpoint in the Phase III AFFINITY DUCHENNE trial with high statistical significance and demonstrated a meaningful correlation between microdystrophin expression and functional improvement. Based on these data, REGENXBIO plans to pursue accelerated approval in 2027.

The company also provided updates on surabgene lomparvovec (ABBV-RGX-314), its gene therapy in development for wet age-related macular degeneration and diabetic retinopathy. Clinical site activation is underway, and first patient dosing in the Phase 2b/3 NAAVIGATE trial is expected to trigger a $100 million milestone payment. REGENXBIO further disclosed that the partial clinical hold on RGX-121, its Hunter syndrome therapy, has been lifted and that it has filed a formal appeal in response to the FDA’s complete response letter.

For the quarter, revenue declined sharply to $90.1 million, and the company recorded a net loss of approximately $90 million, reflecting the absence of upfront license revenue and the expiration of ZOLGENSMA royalties. At quarter end, REGENXBIO held $150.5 million in cash and marketable securities, providing funding into early 2027.

In March, the company released additional interim data from the Phase I/II AFFINITY DUCHENNE trial of RGX-202, reaffirming functional improvements and a favorable safety profile. At that time, REGENXBIO projected topline Phase III results in mid-2026 and planned a pre-BLA meeting with the FDA by year-end.

REGENXBIO is a U.S. biotech developing single-dose, adeno-associated virus–based gene therapies. Its pipeline focuses on rare genetic disorders such as Duchenne muscular dystrophy and Hunter syndrome, as well as retinal diseases including macular degeneration. Despite high development costs and regulatory complexity, limited treatment options in these areas continue to drive competitive partnerships with major pharmaceutical companies.