REGENXBIO Inc.
- Open
- 10.70
- Day high
- 10.86
- Day low
- 10.33
- Prev close
- 10.66
- Volume
- 784K
- Mkt cap
- $711M
- P/E (TTM)
- —
- EPS (TTM)
- —
- P/B
- 9.9
- P/S
- 4.1
- Yield
- —
- Per share
- —
- ▼Insiders net selling -$718K over the last 3 months (0 open-market buys, 3 sales)
- 🏛Institutions accumulating (13F)
REGENXBIO Inc. (RGNX) is a Healthcare company listed on NASDAQ. The stock is up 19% over the past year. Over the trailing 3 months, insiders filed 0 open-market buys and 3 sales (SEC Form 4).
REGENXBIO Inc. (RGNX) financials & analyst ratings
Fundamentals (TTM)
Analyst consensus · 3 analysts
Source: exchange market data + company filings. Figures are trailing-twelve-month or as most recently reported. For informational purposes only — not investment advice.
RGNX earnings date, history & EPS estimates
| Report date | EPS est | EPS actual | Surprise | Revenue | Rev. surprise |
|---|---|---|---|---|---|
| Aug 6, 2026 | $0.08 | $0.43 | +446.2% | $108M | +10.6% |
| May 14, 2026 | $-1.36 | $-1.72 | -26.5% | $6M | -75.3% |
| Mar 5, 2026 | $-1.01 | $-1.30 | -28.7% | $30M | -33.3% |
| Nov 6, 2025 | $-1.38 | $-1.20 | +13.0% | $30M | -49.8% |
| Aug 7, 2025 | $-1.13 | $-1.38 | -22.1% | $21M | -80.6% |
| Mar 13, 2025 | $-1.27 | $-1.01 | +20.5% | $21M | -10.5% |
| Aug 1, 2024 | $-1.29 | $-1.05 | +18.6% | $22M | -8.4% |
| Feb 27, 2024 | $-1.27 | $-1.43 | -12.6% | $22M | -34.7% |
| Aug 2, 2023 | $-1.26 | $-1.66 | -31.7% | $20M | -42.1% |
| May 3, 2023 | $-1.52 | $-1.53 | -0.7% | $19M | -37.5% |
| Feb 28, 2023 | $-1.48 | $-1.38 | +6.8% | $31M | -16.3% |
| Nov 3, 2022 | $-1.55 | $-1.75 | -12.9% | $27M | -14.0% |
RGNX insider trading activity (SEC Form 4)
| Date | Insider | Type | Shares | Price |
|---|---|---|---|---|
| Aug 4, 2026 | PAKOLA STEVEofficer: Chief Medical Officer | Tax | 7,468 | $9.69 |
| Aug 4, 2026 | Christmas Patrick J.officer: Chief Strategy & Legal Officer | Tax | 4,699 | $9.69 |
| Jul 6, 2026 | PAKOLA STEVEofficer: Chief Medical Officer | Option | 2,161 | $7.86 |
| Jul 6, 2026 | PAKOLA STEVEofficer: Chief Medical Officer | Sell | 2,161 | $13.64 |
| Jul 6, 2026 | Simpson Currandirector, officer: Chief Executive Officer | Grant | 1,453 | $10.18 |
| Jul 6, 2026 | Simpson Currandirector, officer: Chief Executive Officer | Tax | 6,818 | $13.09 |
| Jul 6, 2026 | Simpson Currandirector, officer: Chief Executive Officer | Grant | 951 | $7.00 |
| Jul 1, 2026 | PAKOLA STEVEofficer: Chief Medical Officer | Sell | 36,725 | $12.86 |
| Jul 1, 2026 | PAKOLA STEVEofficer: Chief Medical Officer | Option | 36,725 | $7.86 |
| Jul 1, 2026 | PAKOLA STEVEofficer: Chief Medical Officer | Sell | 19,608 | $11.04 |
| Jun 2, 2026 | KARABELAS ARGERIS Ndirector | Grant | 46,600 | $7.01 |
| Jun 2, 2026 | STUMP DAVID Cdirector | Grant | 46,600 | $7.01 |
| Jun 2, 2026 | STUMP DAVID Cdirector | Grant | 9,807 | — |
| Jun 2, 2026 | Zachary Jenniferdirector | Grant | 46,600 | $7.01 |
| Jun 2, 2026 | Mills Kenneth T.director | Grant | 9,807 | — |
Source: RGNX SEC Form 4 filings, latest Aug 4, 2026. For informational purposes only — not investment advice.
See the full RGNX insider & 13F page →REGENXBIO Inc. company profile
Overview
REGENXBIO Inc. (NASDAQ:RGNX) is a clinical-stage biotechnology company founded in 2008 and headquartered in Rockville, Maryland. The company went public in September 2015 and has established itself as a leading developer of gene therapies based on its proprietary NAV Technology Platform, which utilizes adeno-associated virus (AAV) vectors to deliver therapeutic genes to cells. REGENXBIO focuses on developing treatments for genetic diseases and conditions where cells need to produce therapeutic proteins or antibodies. The company has built a robust pipeline of gene therapy candidates targeting various rare diseases, with several programs advancing through clinical trials toward potential regulatory approval.
Business
REGENXBIO operates in the gene therapy sector of biotechnology, developing treatments that address genetic defects by delivering corrective genes directly to patients' cells. Gene therapy represents a revolutionary approach to medicine where instead of treating symptoms with traditional drugs, scientists introduce genetic material into a patient's cells to correct defective genes or provide new cellular functions. The company's core technology is the NAV Technology Platform, a proprietary system that uses adeno-associated virus (AAV) vectors as delivery vehicles. AAV is a naturally occurring virus that has been modified to be safe and effective at delivering therapeutic genes to specific tissues without causing disease. Think of AAV vectors as sophisticated molecular delivery trucks that can transport genetic cargo to precise locations in the body. REGENXBIO's pipeline spans three main therapeutic areas: 1. Retinal diseases (approximately 60-70% of development focus based on partnership scale): The lead program RGX-314 targets wet age-related macular degeneration and diabetic retinopathy, conditions that cause vision loss. This program is developed in partnership with pharmaceutical giant AbbVie and represents the company's most advanced commercial opportunity. 2. Rare genetic diseases (approximately 20-25% of focus): Including RGX-121 for Hunter syndrome (mucopolysaccharidosis type II), a rare genetic disorder where patients lack an enzyme needed to break down certain molecules, leading to progressive damage throughout the body. 3. Neuromuscular diseases (approximately 10-15% of focus): Most notably RGX-202 for Duchenne muscular dystrophy, a severe genetic disorder that causes progressive muscle weakness and degeneration in boys. The company also licenses its NAV Technology Platform to other biotechnology and pharmaceutical companies, creating an additional revenue stream from its proprietary delivery system.
Revenue model
REGENXBIO generates revenue through multiple business models. The primary revenue sources include collaboration and licensing agreements with pharmaceutical partners, particularly the global partnership with AbbVie for the RGX-314 retinal program. This partnership provides upfront payments, milestone payments upon achieving development and regulatory goals, and future royalties on commercial sales. The company also earns revenue by licensing its NAV Technology Platform to other biotechnology and pharmaceutical companies, receiving licensing fees and royalties when partners use REGENXBIO's AAV delivery technology in their own programs. Additionally, REGENXBIO has established partnerships like the one with Nippon Shinyaku for its rare disease programs, providing milestone payments and development funding. Looking toward commercialization, REGENXBIO expects to generate revenue through direct product sales of approved gene therapies, particularly for rare diseases where the company retains commercial rights. Gene therapies typically command premium pricing due to their one-time treatment approach and the high value they provide to patients with otherwise limited treatment options. Several factors could significantly impact the company's margins and profitability. Positive factors include the potential for high-margin gene therapy sales, milestone payments from partners as programs advance, and the scalability of the NAV Technology Platform for licensing. The one-time treatment nature of gene therapies allows for premium pricing, and success in rare diseases often faces limited competition. Challenging factors include the high cost of clinical development and manufacturing for gene therapies, regulatory uncertainties that could delay approvals, and the substantial manufacturing infrastructure required for AAV vector production. The company also faces competition from other gene therapy developers and traditional treatments, while healthcare payers are increasingly scrutinizing the cost-effectiveness of high-priced gene therapies.
Competitive moat
REGENXBIO's competitive moat centers on its proprietary NAV Technology Platform and extensive intellectual property portfolio around AAV vector delivery systems. The company has developed specialized expertise in AAV vector design, manufacturing, and delivery to specific tissues, which creates meaningful barriers to entry for competitors. The platform's versatility allows REGENXBIO to address multiple therapeutic areas with the same core technology, providing operational leverage and risk diversification. The company's strategic partnerships, particularly with AbbVie, provide additional competitive advantages through access to global commercial infrastructure, regulatory expertise, and substantial financial resources. These relationships also validate REGENXBIO's technology and create switching costs for partners who have invested significantly in joint development programs. However, the moat faces several challenges. The gene therapy field is rapidly evolving with numerous competitors developing alternative delivery systems and competing AAV platforms. Large pharmaceutical companies are increasingly developing internal gene therapy capabilities, potentially reducing demand for REGENXBIO's platform licensing. Additionally, the company's intellectual property will eventually expire, and regulatory pathways for gene therapies continue to evolve, potentially favoring different technological approaches. The strength of REGENXBIO's moat is moderate - sufficient to provide near-term competitive advantages but not insurmountable given the dynamic nature of biotechnology innovation. Success will largely depend on the company's ability to demonstrate superior clinical outcomes and maintain technological leadership as the field advances.
Risks & safety
REGENXBIO presents a moderate margin of safety profile typical of clinical-stage biotechnology companies, with both strengths and risks to consider. • Liquidity position: $272 million in cash and marketable securities as of Q1 2025, providing runway into second half of 2026. Current ratio of 2.93 indicates strong short-term liquidity. • Debt levels: Manageable debt-to-equity ratio of 0.29, indicating conservative capital structure without excessive leverage risk. • Cash burn: Positive free cash flow of $33 million in Q1 2025, a significant improvement from previous quarters' negative cash flows. However, this appears driven by milestone payments rather than sustainable operations. • Valuation metrics: P/E ratio of 15.1 based on recent positive earnings, though this may not reflect sustainable profitability. EV/EBITDA of 6.5 appears reasonable for a biotech with near-term commercial prospects. • Revenue visibility: Partnership milestones and potential Priority Review Voucher monetization provide near-term non-dilutive funding options. • Clinical risks: Multiple programs in late-stage development create both opportunity and execution risk. Regulatory approval timelines remain uncertain despite advanced trial stages.
Recent development
Over the past few years, REGENXBIO has executed a focused strategy to advance its most promising programs toward commercialization while building strategic partnerships to reduce development risk and enhance commercial potential. The company's most significant strategic move was establishing a global collaboration with AbbVie for the RGX-314 retinal program, which provides substantial financial support and access to AbbVie's global ophthalmology commercial infrastructure. This partnership has enabled REGENXBIO to advance pivotal trials for both wet age-related macular degeneration and diabetic retinopathy, with enrollment completion expected in 2025 and top-line data anticipated in 2026. In rare diseases, REGENXBIO achieved a major milestone by submitting its first Biologics License Application (BLA) for RGX-121 in Hunter syndrome in March 2025, positioning the company for potential FDA approval in the second half of 2025. The company also established a partnership with Nippon Shinyaku for its mucopolysaccharidosis programs, providing additional funding and access to Japanese markets. The RGX-202 program for Duchenne muscular dystrophy has progressed significantly, with the company advancing into pivotal studies and expanding the program to include younger patients aged 1-3 years. REGENXBIO is targeting a BLA submission in mid-2026 with potential commercial launch in 2027. Operationally, the company has invested heavily in manufacturing capabilities, opening a new cGMP facility in Rockville, Maryland, with capacity to produce AAV vectors at commercial scale. This vertical integration provides greater control over supply chain and manufacturing costs while supporting the company's "5x25" strategy to advance five AAV therapeutics to pivotal or commercial stage by 2025.
RGNX company profile · for informational purposes only — not investment advice.
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