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what it is
REGENXBIO tries to turn viruses into delivery trucks for gene therapy drugs targeting eye, liver, and brain diseases.
how it gets paid
Last year Regenxbio made $170M in revenue.
why it's growing
Revenue grew 104.5% last year. $140M mattered most because it equals about 82% of the full-year $170M total.
what just happened
The quarter delivered $140M of revenue, but the stock still wears a clinical-stage biotech loss profile with EPS at -$2.46.
At a glance
C++ balance sheet — some cracks in the foundation
5/100 earnings predictability — expect surprises
16.7% return on capital — nothing to write home about
-$4.59 fy2024 eps est
$83M fy2024 rev est
xvary composite: 30/100 — weak
What they do
REGENXBIO tries to turn viruses into delivery trucks for gene therapy drugs targeting eye, liver, and brain diseases.
The moat is the NAV platform, which is biotech jargon → a proprietary virus delivery toolkit → so your drug shots have a reusable engine. The company has 353 employees and a lead program in wet AMD, so you are not buying a lab sketch. You are buying a platform with multiple shots on goal.
How they make money
$170M
annual revenue · their business grew +104.5% last year
total revenue
$170M
+104.5%
The products that matter
licenses AAV delivery vectors
NAV Technology Platform
$170M revenue · 20+ licensees
this platform supported over 20 licensees and helped produce $170M in 2025 revenue. it matters because it funds the pipeline, but last year's $194M loss shows it is not self-sustaining on its own.
funding engine
gene therapy for wet AMD
RGX-314 (with AbbVie)
two pivotal trials · data expected Q4 2026
ATMOSPHERE and ASCENT are the pivotal trials that matter here, with top-line data expected in Q4 2026. if they work, the commercial story gets real. if they do not, the platform story looks a lot thinner.
lead catalyst
gene therapy for Duchenne MD
RGX-202
enrollment complete · top-line expected early Q2 2026
pivotal trial enrollment is complete, and top-line data is expected in early Q2 2026. that makes this the nearer-term test of whether RGNX can turn scientific promise into something the market will pay for.
near-term readout
Key numbers
94.6%
operating margin
Operating margin → profit after running the business → so what: REGENXBIO still loses almost a dollar for every dollar it brings in.
$217M
long-term debt
Debt → money owed → so what: a clinical-stage biotech already carrying $217M has less room for trial delays.
$170M
annual revenue
Revenue → money coming in → so what: this is not pre-revenue biotech theater anymore.
1.7
beta
Beta → how jumpy the stock is versus the market → so what: you should expect bigger swings than the S&P 500.
Financial health
C++
strength
- balance sheet grade C++ — below average — limited financial resources
- risk rank 5 — safer than 5% of stocks
- price stability 5 / 100
- long-term debt $217M (33% of capital)
C++ — below average. watch for debt servicing and cash burn.
Total return vs. market
Return history isn't available for RGNX right now.
source: institutional data · return history unavailable
What just happened
missed estimates
The quarter delivered $140M of revenue, but the stock still wears a clinical-stage biotech loss profile with EPS at -$2.46.
Revenue surged 371% vs. prior year in the latest quarter, and annual revenue reached $170M, up 104.5%. The quiet part out loud: one quarter produced 82% of the year's revenue, which makes concentration the story.
$43M
revenue
$2.46
eps
87.2%
gross margin
the number that mattered
$140M mattered most because it equals about 82% of the full-year $170M total, which tells you this revenue base is lumpy, not smooth.
source: company earnings report, 2026
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What could go wrong
the #1 risk is clinical failure or delay in RGX-202 and RGX-314.
med
Lead-program failure would break the equity story
RGX-202 and RGX-314 are doing most of the valuation work. One program is the nearer-term readout. The other carries the larger partnered commercial dream. If the data disappoints, the stock stops being a pipeline story and starts looking like a lossmaking platform business.
At a $442M market cap, the pipeline is clearly priced in. A failed readout would directly challenge the main reason investors are here.
med
Cash burn stays the clock on the wall
Last year brought $170M in revenue and a $194M loss. That is a -114% net margin. Put differently, the business spent more than $2 for every $1 it brought in.
If revenue falls toward the $83M fy2024 estimate while losses remain heavy, financing pressure rises fast. That usually means dilution, debt, or program prioritization.
med
Regulatory friction is already on the page
The FDA placed clinical holds on RGX-111 and RGX-121 in January 2026. That is not a theoretical risk memo. It already happened.
Even when a hold hits a different asset, it can slow timelines, raise study costs, and make investors less patient with the rest of the pipeline.
med
The class action adds noise when the company needs trust
A securities class action alleges fraud between February 2022 and January 2026. Lawsuits rarely decide the science, but they can complicate the story around it.
This is more overhang than existential threat, but small-cap biotech does not get extra benefit of the doubt when legal headlines pile on.
The risk stack is unusually concentrated. A -114% net margin, $217M of long-term debt, and multiple pipeline catalysts mean you are underwriting execution more than current earnings power.
source: institutional data · regulatory filings · risk analysis
Pay attention to
catalyst
RGX-202 top-line data
expected early Q2 2026. this is the nearest major readout and the most obvious sentiment reset button.
regulatory
Any update on the FDA holds for RGX-111 and RGX-121
january 2026 already introduced friction. what matters next is whether those holds get resolved cleanly or start infecting the broader pipeline narrative.
catalyst
RGX-314 pivotal data with AbbVie
Q4 2026 for ATMOSPHERE and ASCENT. this is the bigger later-stage swing, but it sits behind RGX-202 on the calendar.
financial
Whether revenue keeps collapsing after the $170M spike
the fy2024 estimate is $83M. if that kind of drop becomes the norm, the platform stops looking like a stabilizer and starts looking like a temporary bridge.
Analyst rankings
earnings predictability
5 / 100
in human-speak, analysts do not trust quarterly numbers here to be smooth or repeatable.
price stability
5 / 100
this stock does not trade like a sleepy healthcare name. it trades like binary news can hit at any time.
balance sheet grade
C++
below average balance sheet grade. enough to keep operating, not enough to make pipeline delays painless.
source: institutional data
Institutional activity
institutional ownership data for RGNX is being compiled.
source: institutional data
Price targets
3-5 year target range
n/a
n/a
$8
current price
n/a
target midpoint · n/a from current
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