REGENXBIO Inc.
RGNX evidence brief
What we can confirm
- BioSniper Score:
- 5.5 / 10
- Market Cap:
- $580.3M
- Lead Asset:
- ABBV-RGX-314 (Phase 3)
What it means
What to watch
Prepared research question
What matters most for RGNX—catalysts, financial risk and supporting evidence?
Traceable citations · Unknowns marked
BioSniper AI · RGNX
REGENXBIO Inc. is a publicly traded gene therapy company developing AAV-based treatments for rare and retinal diseases, with lead programs in wet AMD (partnered with AbbVie) and Duchenne muscular dystrophy.
Key risk: Low cash reserves
BioSniper Score
Based on 4 of 6 signals
Market Cap
$580.3M
Cash Runway
—
Lead Asset
ABBV-RGX-314 (Phase 3)
Next Catalyst
—
Financial data as of Jun 30, 2026.
Tracking 1 pipeline candidate · 10 clinical trials for REGENXBIO Inc..
REGENXBIO Inc. is a publicly traded gene therapy company developing AAV-based treatments for rare and retinal diseases, with lead programs in wet AMD (partnered with AbbVie) and Duchenne muscular dystrophy.
- Cash & equivalents of $36.5M as of 2026-06-30 [sec].
- Total revenue of $108.0M and net income of $22.7M for the period ending 2026-06-30 [sec].
- Lead program ABBV-RGX-314 (surabgene lomparvovec) for wet AMD and diabetic retinopathy is in Phase 3 pivotal trials with over 1,200 participants enrolled as of 2025 [sec · filed 2026-04-14].
- RGX-202 for Duchenne muscular dystrophy entered Phase 3 in January 2023 [catalyst].
- Completed a public offering in 2026 raising approximately $107.8 million in net proceeds [sec_mda · as of 2026-08-06].
What works
- Strong partnership with AbbVie for the ABBV-RGX-314 program, providing development and commercialization support [pipeline].
- Multiple late-stage clinical programs (Phase 3) in large-market indications (wet AMD, DMD) with significant enrollment completed [sec · filed 2026-04-14][catalyst].
- Recent financing raised $107.8M net, extending cash runway into 2027 [sec_mda · as of 2026-08-06][news · published 2026-08-11].
What to weigh
- Cash position of $36.5M as of 2026-06-30 is relatively low for a company with multiple late-stage trials and no approved products [sec].
- FDA hold on one program (Hunter syndrome) has caused a sell-off and increased volatility [news · published 2026-08-25].
- Gene therapy development carries high risk of clinical setbacks and regulatory delays, as noted in the company's own risk factors [sec_mda · as of 2026-08-06].
From the filings
Quoted directly from source documents.
ABBV-RGX-314 pivotal trials for wet AMD have enrolled over 1,200 participants.
“We completed enrollment of more than 1,200 participants in the ABBV-RGX-314 ATMOSPHERE®and ASCENT®pivotal trials for the treatment of Wet AMD using subretinal delivery.”
SEC filings · April 14, 2026
Net proceeds from a public offering were approximately $107.8 million.
“The aggregate net proceeds received from the offering were approximately $107.8 million, net of underwriting discounts and commissions and estimated offering expenses.”
SEC MD&A · August 6, 2026
Cash runway projected to 2027.
“Regenxbio surged on milestone revenue and EPS beat, with a $313M cash runway to 2027.”
News · August 11, 2026
Catalyst timeline
- 2019-04-03 · Phase 2 start for RGX-111 (MPS I) — A Phase I/II study evaluating intracisternal RGX-111 in participants with Mucopolysaccharidosis Type I.
- 2023-01-04 · Phase 3 start for RGX-202 (DMD) — A Phase 1/2/3 open-label study of intravenous RGX-202 in males with Duchenne Muscular Dystrophy.
- 2025-03-18 · Phase 2 start for RGX-314 (diabetic macular edema) — A randomized, open-label, controlled Phase 2 study of subretinal RGX-314 in participants with center-involved diabetic macular edema.
- 2025-11-25 · Phase 3 start for RGX-314 (wet AMD) — Phase 3 pivotal trials (ATMOSPHERE and ASCENT) for wet AMD using subretinal delivery.
Risks & what to watch (3)›
Key risks
- Low cash reserves — Cash & equivalents of $36.5M as of 2026-06-30 may be insufficient to fund all programs to approval without additional financing [sec].
- FDA hold on Hunter program — An FDA hold on the RGX-111 (Hunter syndrome) program has triggered a sell-off and increased regulatory risk [news · published 2026-08-25].
- Clinical trial risk — Gene therapy development is inherently risky; setbacks in late-stage trials could significantly impact the company [sec_mda · as of 2026-08-06].
What to watch
- ABBV-RGX-314 pivotal data readout expected by Q4 2026 [news · published 2026-06-22].
- RGX-202 Phase 3 progress in Duchenne muscular dystrophy [catalyst].
- Resolution of the FDA hold on the RGX-111 program [news · published 2026-08-25].
- Cash runway and potential need for additional financing as 2027 approaches [news · published 2026-08-11].
Sources: Catalysts · News · Pipeline · SEC filings · SEC MD&A · Generated October 13, 2026 · How we verify sources →
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Evidence & data
Score breakdownBased on 4 of 6 signals · derived from public data
Cash runway data unavailable
D/E 4.39 · 0% dilution
Lead: Phase 3
1 candidate
No upcoming catalysts tracked
2 buys · 4 sells (180d)
Pipeline
Regulatory
Financials
Institutional Holdings
Source: SEC Form 13F · as of Mar 2026Institutional holders
178
Total value held
$350.0M
| Holder | Shares | Value |
|---|---|---|
| blackrock, inc. | 5,782,808 | $48.5M |
| redmile group, llc | 4,368,804 | $36.6M |
| jpmorgan chase & co | 3,483,800 | $27.1M |
| state street corp | 2,731,485 | $22.9M |
| morgan stanley | 2,066,157 | $17.3M |
| vanguard capital management llc | 2,007,146 | $16.8M |
| goldman sachs group inc | 1,839,463 | $15.4M |
| integral health asset management, llc | 1,500,000 | $12.6M |
| qube research & technologies ltd | 1,310,493 | $11.0M |
| geode capital management, llc | 1,137,498 | $9.5M |
Showing top 10 of 178 institutional holders of REGENXBIO Inc.. Open the full 13F history after sign-in.
Short Interest
as of May 19, 2026- % of float
- 13.9%
- Days to cover
- 11.9
- Shares short
- 6,692,556
- Daily short volume
- -
Short percent of float and days-to-cover reflect reported short positioning. Daily short volume is the exchange-reported short share of a single day's trading volume and is noisier. See the most-shorted biotech ranking →
RGNX short interestGovernance
Frequently asked questions
What is the AI investment thesis for REGENXBIO Inc.?
REGENXBIO Inc. is a publicly traded gene therapy company developing AAV-based treatments for rare and retinal diseases, with lead programs in wet AMD (partnered with AbbVie) and Duchenne muscular dystrophy.
What is REGENXBIO Inc.'s lead drug candidate?
REGENXBIO Inc.'s most advanced tracked candidate is ABBV-RGX-314 (Phase 3) for Wet age-related macular degeneration (wet AMD) and diabetic retinopathy (DR).
Who is the CEO of REGENXBIO Inc.?
Mr. Curran M. Simpson M.S. is the chief executive officer of REGENXBIO Inc. (RGNX).
Where is REGENXBIO Inc. headquartered?
REGENXBIO Inc. (RGNX) is headquartered in Rockville, MD, United States.
What therapeutic areas does REGENXBIO Inc. focus on?
REGENXBIO Inc. develops therapies across Rare Disease, Gene Therapy, Ophthalmology.
The AI overview and BioSniper Score are neutral, data-derived signals built from public SEC filings, ClinicalTrials.gov and FDA records — not investment advice.