RegenXBio (RGNX) AI Stock Analysis

• Market Cap: $431.48M
• Dividend Yield: N/A
• Average Volume (3M): 957.64K
• Price to Earnings (P/E): ―
• Beta (1Y): 2.31
• Revenue Growth: 91.30%
• EPS Growth: 30.69%
• Country: US
• Employees: 353
• Sector: Healthcare
• Sector Strength: 45
• Industry: Biotechnology
• EPS (TTM): -1.30
• Shares Outstanding: 51,612,984
• 10 Day Avg. Volume: 1,022,172
• 30 Day Avg. Volume: 957,645
• PEG Ratio: 0.21
• Price to Book (P/B): 7.23
• Price to Sales (P/S): 4.36
• P/FCF Ratio: -5.88
• Enterprise Value/Market Cap: 1.50
• Enterprise Value/Revenue: 3.80
• Enterprise Value/Gross Profit: 7.19
• Enterprise Value/Ebitda: -4.45
• 1Y Price Target: $30.60
• Price Target Upside: 266.03% Upside
• Rating Consensus: Strong Buy
• Number of Analyst Covering: 11
• EPS Forecast (FY): -2.15
• Revenue Forecast (FY): $243.39M

• Rating: 45 Neutral
• Price Target: $10.00 ▲(19.62% Upside)
• Action: Reiterated Date: 03/06/26
• Date: Action: Reiterated 03/06/26

The score is held down primarily by weak financial performance (large ongoing losses and continued cash burn) and bearish technical positioning (below key moving averages with negative MACD). Offsetting factors include constructive earnings-call catalysts and liquidity guidance into early 2027 (with a potential AbbVie milestone), but regulatory setbacks and program risk remain material.

Positive Factors

Platform Partnerships & Milestones

The NAV AAV platform generates non-dilutive cash via large partnership milestones and royalties. A validated collaboration with AbbVie and milestone-triggered payments provide durable funding optionality and third-party validation that supports long-term R&D and commercialization capacity.

Late-stage Pipeline Momentum

Pivotal enrollment completion and robust functional signals for RGX-202 materially de-risk the Duchenne program versus earlier-stage assets. If topline and subsequent regulatory interactions remain constructive, this asset can shift the company toward sustainable product revenue and reduce reliance on partner monetization.

Revenue Recovery, High Gross Margins & Runway

A strong 2025 revenue rebound and very high gross margin reflect the capital-efficient licensing/royalty model and limited COGS for platform-derived revenues. Combined with $241M cash and recent monetization transactions, the company has a definable runway to advance late-stage programs while pursuing additional milestone or royalty funding.

Negative Factors

Regulatory Safety Setbacks

A documented AAV integration event linked to a tumor and subsequent CRL/clinical holds create persistent regulatory and safety scrutiny across AAV CNS programs. This can force longer follow-up, additional studies, or altered regulatory expectations that materially extend timelines and development costs for affected indications.

Sustained Cash Burn & Negative Free Cash Flow

Ongoing negative operating and free cash flow indicate the business is not yet self-funding. Persistent burn increases reliance on upfronts, milestone payments, royalty monetizations, and capital markets — raising execution risk if financings or partner payments are delayed or reduced.

Eroding Equity & Higher Leverage

A sharply reduced equity base and higher leverage diminish financial flexibility and the company’s ability to absorb shocks. With negative returns on equity since 2022, the balance-sheet shift raises refinancing and covenant risk and limits capacity to pursue opportunistic investments without external funding.

RegenXBio Business Overview & Revenue Model

Company Description

REGENXBIO Inc., a clinical-stage biotechnology company, provides gene therapy product candidates to deliver genes to cells to address genetic defects or to enable cells in the body to produce therapeutic proteins or antibodies that are intended to impact disease. Its gene therapy product candidates are based on NAV Technology Platform, a proprietary adeno-associated virus gene delivery platform. The company's lead product candidate is RGX-314, which is in Phase III clinical trial for the treatment of wet age-related macular degeneration. It is also developing RGX-121 that is in Phase I/II clinical trial to treat mucopolysaccharidosis type II;RGX-111, which is in Phase I/II clinical trial for treating mucopolysaccharidosis type I;RGX-181 which is in pre clinic stage for the treatment of late-infantile neuronal ceroid lipofuscinosis type II disease;RGX-202, to treat Duchenne muscular dystrophy which is in phase I/II clinical trial; and RGX-381, to treat the ocular manifestations of CLN2 disease which is in preclinical stage. REGENXBIO Inc. also licenses its NAV Technology Platform to other biotechnology and pharmaceutical companies; and has a collaboration and license agreement with Neurimmune AG to develop novel gene therapies. REGENXBIO Inc. was founded in 2008 and is headquartered in Rockville, Maryland.

How the Company Makes Money

RegenXBio generates revenue primarily through strategic partnerships and collaborations with larger pharmaceutical companies, which often involve upfront payments, milestone payments, and royalties on sales of approved therapies. The company may receive funding through research grants and investments aimed at advancing its gene therapy pipeline. Additionally, potential commercialization of its therapies, once approved, will contribute to revenue through product sales. Collaborations with organizations such as Novartis and other biotech firms are significant sources of income, as they typically include financial support for research and development activities, as well as sharing of technology and expertise.

RegenXBio Financial Statement Overview

Summary

Financials are pressured by multi-year large net losses and persistently negative operating/free cash flow (2025 FCF about -$126M). Revenue rebounded strongly in 2025 (about +106% YoY) with very strong gross margin (~88%), but heavy operating costs and the erosion of equity (down sharply since 2021) keep overall fundamentals weak despite moderate absolute debt.

Income Statement

Revenue has been volatile, with a sharp decline from 2021 to 2024 and a rebound in 2025 (annual revenue up ~106% YoY in 2025 after modest declines in 2023–2024). Profitability is the key weakness: the company has posted large losses since 2022, with very negative net margins (2025 net margin ~-114% vs. 2021 net margin ~27%). A clear positive is strong gross profitability in 2025 (gross margin ~88%), but heavy operating costs keep operating and net results deeply negative.

Balance Sheet

Leverage is moderate and has risen as equity has declined: debt-to-equity increased from ~0.11 (2021) to ~0.72 (2025), reflecting erosion in the equity base. Total debt is fairly stable (~$74–95M across the period), but equity fell meaningfully from ~$764M (2021) to ~$103M (2025), which reduces financial flexibility. Returns on equity have been strongly negative since 2022, consistent with sustained net losses.

Cash Flow

Cash generation is a major concern: operating cash flow and free cash flow have been consistently negative from 2022 through 2025 (2025 operating cash flow about -$124M; free cash flow about -$126M). The burn rate improved versus 2023–2024 (less negative cash flow), but the business is still not self-funding. Cash flow is broadly tracking reported losses (free cash flow to net income ~1.0 in 2024–2025), which signals limited offset from non-cash charges and underscores ongoing cash consumption.

Breakdown	Dec 2025	Dec 2024	Dec 2023	Dec 2022	Dec 2021
Income Statement
Total Revenue	170.44M	83.33M	90.24M	112.72M	470.35M
Gross Profit	150.14M	49.76M	53.03M	58.18M	418.51M
EBITDA	-145.58M	-198.23M	-239.46M	-244.24M	169.54M
Net Income	-193.88M	-227.10M	-263.49M	-280.32M	127.84M
Balance Sheet
Total Assets	453.03M	465.99M	573.97M	833.27M	1.11B
Cash, Cash Equivalents and Short-Term Investments	230.07M	234.69M	275.26M	364.64M	457.44M
Total Debt	260.52M	82.03M	89.29M	94.80M	86.68M
Total Liabilities	350.30M	206.34M	262.23M	317.07M	349.61M
Stockholders Equity	102.73M	259.65M	311.74M	516.20M	764.30M
Cash Flow
Free Cash Flow	-126.38M	-175.56M	-228.37M	-238.21M	134.70M
Operating Cash Flow	-123.96M	-173.13M	-218.41M	-207.49M	218.88M
Investing Cash Flow	-15.87M	103.45M	190.94M	-11.93M	-406.64M
Financing Cash Flow	116.77M	92.68M	-34.97M	-28.84M	195.25M

RegenXBio Technical Analysis

• Technical Analysis Sentiment: Negative
• Last Price: 8.36
• 50DMA: Negative
• 100DMA: Negative
• 200DMA: Negative
• MACD: Negative
• RSI: Neutral
• STOCH: Positive

Evaluating momentum and price trends is crucial in stock analysis to make informed investment decisions. For RGNX, the sentiment is Negative. The current price of 8.36 is below the 20-day moving average (MA) of 8.81, below the 50-day MA of 10.70, and below the 200-day MA of 10.56, indicating a bearish trend. The MACD of -0.43 indicates Negative momentum. The RSI at 41.13 is Neutral, neither overbought nor oversold. The STOCH value of 13.38 is Positive, not indicating any strong overbought or oversold conditions. Overall, these indicators collectively point to a Negative sentiment for RGNX.

RegenXBio Peers Comparison

Name	Overall Rating	Market Cap	P/E Ratio	ROE	Dividend Yield	Revenue Growth	EPS Growth
SLDB	57 Neutral	$554.72M	-2.83	-73.63%	―	―	17.88%
GLUE	54 Neutral	$1.29B	-33.72	-15.12%	―	1112.27%	―
MNPR	53 Neutral	$392.07M	―	-25.72%	―	―	-73.48%
Sector *	51 Neutral	$7.86B	-0.30	-43.30%	2.27%	22.53%	-2.21%
RGNX	45 Neutral	$431.48M	-3.83	-103.12%	―	91.30%	30.69%
INBX	42 Neutral	$943.45M	―	-269.56%	―	―	―

RegenXBio Corporate Events

REGENXBIO Faces FDA Setback on RGX-121 Gene Therapy

On February 9, 2026, REGENXBIO Inc. reported that the U.S. Food and Drug Administration had issued a Complete Response Letter for its Biologics License Application for RGX-121, a one-time gene therapy for the ultra-rare neurodegenerative disorder Mucopolysaccharidosis II, or Hunter syndrome. The FDA’s February 7, 2026 letter cited concerns over defining the neuronopathic patient population, the comparability of the external natural history control group, and the use of CSF HS D2S6 as a surrogate endpoint for clinical benefit.

The setback halts near-term approval plans for RGX-121 despite a decade of development and positive biomarker, functional, and safety data from the CAMPSIITE I/II/III trial, and it underscores the regulatory challenges of demonstrating efficacy in ultra-rare diseases. REGENXBIO said it will seek a Type A meeting and pursue a path to resubmit the application, as clinicians and patient advocates warned that delays in access to innovative gene therapies could have severe consequences for children with rapidly progressive Hunter syndrome.

The most recent analyst rating on (RGNX) stock is a Hold with a $12.00 price target. To see the full list of analyst forecasts on RegenXBio stock, see the RGNX Stock Forecast page.

FDA Halts RegenXBio MPS Gene Therapy Trials

On January 28, 2026, REGENXBIO announced that the U.S. Food and Drug Administration placed clinical holds on its investigational gene therapies RGX-111 for MPS I (Hurler syndrome) and RGX-121 for MPS II (Hunter syndrome). The action followed detection of an intraventricular CNS tumor in a five-year-old participant who had received RGX-111 four years earlier; preliminary analysis found an AAV vector genome integration event linked to overexpression of the proto-oncogene PLAG1, though causality has not been established and the child remains asymptomatic with developmental gains. No neoplasms have been seen in nine other RGX-111 recipients or in 32 patients treated with RGX-121, and management publicly questioned the FDA’s decision to halt RGX-121 given its safety record and pivotal-trial efficacy to date. The holds temporarily stall two cornerstone ultra-rare disease programs that target critical unmet neurological needs in MPS I and II, introducing regulatory uncertainty for REGENXBIO’s CNS gene therapy platform while heightening scrutiny of AAV vector integration risks across the gene therapy field.

The most recent analyst rating on (RGNX) stock is a Buy with a $37.00 price target. To see the full list of analyst forecasts on RegenXBio stock, see the RGNX Stock Forecast page.