REGENXBIO, Inc. (RGNX)

REGENXBIO's stock has increased by 72.24% in the past six months. The stock hit new 52-week highs in January 2026. Shares rose approximately 5% following encouraging Phase 1/2 trial results for RGX-202, its Duchenne muscular dystrophy treatment. REGENXBIO reported Q3 2025 EPS of -$1.20, beating forecasts by $0.10, with revenue of $29.7 million exceeding expectations by 20.58%, which led to a 2.76% stock increase in premarket trading. Several analysts have reiterated "Buy" or "Outperform" ratings and increased price targets for RGNX. The company has a PDUFA date set for February 8, 2026, for RGX-121, and a BLA submission for RGX-202 is anticipated in mid-2026.

REGENXBIO's products and services are not subject to traditional consumer-driven demand seasonality. Demand for its therapies is primarily driven by disease prevalence, clinical efficacy, regulatory approvals, and market access, rather than seasonal fluctuations. The company's revenue generation is currently more tied to licensing agreements, milestone payments from partnerships, and progress in its clinical pipeline.

REGENXBIO, Inc. is a clinical-stage biotechnology company focused on gene therapies for severe and rare diseases, utilizing its NAV Technology Platform. Its business model focuses on advancing a late-stage pipeline of gene therapy candidates and monetizing its NAV Technology Platform through strategic licensing and collaboration agreements. Key product candidates include ABBV-RGX-314 for retinal diseases, RGX-202 for Duchenne muscular dystrophy, RGX-121 for mucopolysaccharidosis type II, RGX-111 for mucopolysaccharidosis type I, RGX-181 for late-infantile neuronal ceroid lipofuscinosis type 2, and RGX-381 for ocular manifestations of CLN2 disease. The company also licenses its NAV Technology Platform to other companies.

REGENXBIO Inc. is headquartered in Rockville, Maryland, United States. The company's strategic partnerships extend its reach, including a collaboration with AbbVie Global Enterprises Ltd. to develop ABBV-RGX-314 outside the United States, and a partnership with Nippon Shinyaku for RGX-121 and RGX-111 in the United States and Asia.

REGENXBIO's brand reputation has been positively influenced by a "Moderate Buy" analyst rating, the FDA approval of Zolgensma validating its NAV Technology Platform, encouraging clinical trial results for RGX-202, and strategic partnerships with major biopharmaceutical companies. While the company is not yet profitable, its revenue growth and progress towards commercialization are viewed positively.

Institutional investors hold over 50% of REGENXBIO's shares, with major holders including BlackRock, Inc., Redmile Group, LLC, JPMorgan Chase & Co, Vanguard Group Inc, State Street Corp, Goldman Sachs Group Inc, and Morgan Stanley. Allan M. Fox, Chairman of the Board, is a significant individual shareholder, owning 6.91 million shares representing 13.64% of the company. The company's CEO, Kenneth Mills, directly holds 0.6% of the total shares outstanding.