REGENXBIO Inc.
RGNX · Biological Products, (No Diagnostic Substances) · 51.7M shares · +8%/yr ↑ dilution · EDGAR filings ↗
Reference price $11.02 · est. market cap ~$570M — last insider sale, 2026-05-11 (estimated from the dated SEC Form 4 price × shares outstanding — not a live quote)
Grade
D48Gene-therapy developer with partnership-driven revenue growth but a Complete Response Letter, clinical holds, and going-concern-adjacent cash doubt.
Strengths
- Triple-digit revenue growth (+105%, 88.6 pct) from AbbVie collaboration and NAV licensee royalties (Zolgensma/Itvisma)
- Reasonable cash position ($251M, 70.9 pct) and a broad late-stage AAV pipeline (sura-vec wet AMD, RGX-202 Duchenne)
Risks
- FDA CRL on the RGX-121 BLA plus clinical holds on RGX-111/RGX-121; company flags existing cash may not fund 12 months
- Deeply unprofitable (operating margin -94.6%, ROE -1.89 at 12.6 pct); RGX-314 wet-AMD signal encouraging but HoFH program terminated with high AE rates
Notable findings
- Net lossNet loss (net margin -113.8%)
- Fund exitedNotable fund(s) exited this quarter: VANGUARD GROUP INC
- Cash burnNegative free cash flow (-126.4M)
- Strong balance sheetStrong balance sheet — $252M cash & investments, ~8 quarters of runway
- Revenue declineRevenue -78.9% YoY
- Notable holderHeld by 5 notable fund(s): STATE STREET CORP, GOLDMAN SACHS GROUP INC, FMR LLC +2 more (as of 2026-03-31)
- Trial resultTrial topline data reported — see 8-K for outcome 8-K ↗
Quarterly revenue
Last 4 quarters · from SEC filings.
Key metrics
Operating margin
-94.6%
Net margin
-113.8%
Return on equity
-188.7%
Return on assets
-42.8%
Revenue growth (QoQ)
-78.9%
Current ratio
2.4×
Free cash flow
-$126M
EBITDA
-$146M
Tangible book value
$103M
Cash & investments
$252M
Cash runway
8q
Stock comp / revenue
20.3%
Insider transactions · last 18 months
| Date | Insider | Type | Shares | Price |
|---|---|---|---|---|
| 2026-05-29 | Bennett Jean | Grant | 9.8K | — |
| 2026-05-29 | Zachary Jennifer | Grant | 9.8K | — |
| 2026-05-29 | Glucksmann Alexandra | Grant | 9.8K | — |
| 2026-05-29 | Fox Allan M. | Grant | 9.8K | — |
| 2026-05-29 | Mills Kenneth T. | Grant | 9.8K | — |
| 2026-05-29 | Tasse Daniel | Grant | 9.8K | — |
| 2026-05-29 | STUMP DAVID C | Grant | 9.8K | — |
| 2026-05-29 | KARABELAS ARGERIS N | Grant | 9.8K | — |
| 2026-05-29 | MIGAUSKY GEORGE V | Grant | 9.8K | — |
| 2026-05-11 | PAKOLA STEVE | Sell | 15K | $11.02 |
| 2026-03-10 | PAKOLA STEVE | Sell | 5.1K | $10.35 |
| 2026-02-01 | PAKOLA STEVE | Tax withheld | 5.3K | $11.16 |
| 2026-02-01 | Christmas Patrick J. | Tax withheld | 4.7K | $11.16 |
| 2026-01-27 | Mills Kenneth T. | Option exercise | 225K | $13.09 |
| 2026-01-27 | Mills Kenneth T. | Tax withheld | 222K | $13.41 |
| 2026-01-06 | Malzahn Craig | Grant | 66K | — |
| 2026-01-06 | Chan Mitchell | Grant | 74K | — |
| 2026-01-06 | PAKOLA STEVE | Grant | 66K | — |
| 2026-01-06 | Christmas Patrick J. | Grant | 74K | — |
| 2026-01-06 | Simpson Curran | Grant | 145K | — |
| 2026-01-02 | Malzahn Craig | Tax withheld | 1.2K | $14.40 |
| 2026-01-02 | PAKOLA STEVE | Tax withheld | 18K | $14.40 |
| 2026-01-02 | Christmas Patrick J. | Tax withheld | 9.8K | $14.40 |
| 2026-01-02 | Simpson Curran | Tax withheld | 19K | $14.40 |
| 2025-10-09 | Simpson Curran | Sell | 21K | $12.62 |
| 2025-09-29 | Simpson Curran | Sell | 7.6K | $10.03 |
| 2025-09-10 | Simpson Curran | Sell | 7.7K | $10.02 |
| 2025-09-01 | Malzahn Craig | Tax withheld | 234 | $8.93 |
| 2025-09-01 | Chan Mitchell | Tax withheld | 4.9K | $8.93 |
| 2025-08-01 | Christmas Patrick J. | Tax withheld | 4.7K | $8.19 |
| 2025-07-01 | Simpson Curran | Tax withheld | 4.7K | $8.23 |
| 2025-05-30 | Fox Allan M. | Grant | 7.8K | — |
| 2025-05-30 | Zachary Jennifer | Grant | 7.8K | — |
| 2025-05-30 | Tasse Daniel | Grant | 7.8K | — |
| 2025-05-30 | STUMP DAVID C | Grant | 7.8K | — |
| 2025-05-30 | Mills Kenneth T. | Grant | 7.8K | — |
| 2025-05-30 | MIGAUSKY GEORGE V | Grant | 7.8K | — |
| 2025-05-30 | Glucksmann Alexandra | Grant | 7.8K | — |
| 2025-05-30 | KARABELAS ARGERIS N | Grant | 7.8K | — |
| 2025-05-30 | Bennett Jean | Grant | 7.8K | — |
| 2025-05-12 | Mills Kenneth T. | Option exercise | 21K | $3.76 |
| 2025-05-12 | Mills Kenneth T. | Sell | 21K | $7.91 |
| 2025-03-27 | Mills Kenneth T. | Option exercise | 31K | $3.76 |
| 2025-02-01 | PAKOLA STEVE | Tax withheld | 5.3K | $8.07 |
| 2025-02-01 | Danos Olivier | Tax withheld | 5.5K | $8.07 |
| 2025-02-01 | Christmas Patrick J. | Tax withheld | 4.7K | $8.07 |
| 2025-01-02 | Danos Olivier | Grant | 59K | — |
| 2025-01-02 | Simpson Curran | Tax withheld | 12K | $7.73 |
| 2025-01-02 | Simpson Curran | Grant | 99K | — |
| 2025-01-02 | Christmas Patrick J. | Tax withheld | 11K | $7.73 |
| 2025-01-02 | PAKOLA STEVE | Tax withheld | 14K | $7.73 |
| 2025-01-02 | PAKOLA STEVE | Grant | 64K | — |
| 2025-01-02 | Danos Olivier | Tax withheld | 13K | $7.73 |
All non-derivative Form 4 activity (SEC), newest first.
Clinical trials
| Trial | Title | Phase | Status | Primary completion |
|---|---|---|---|---|
| NCT06491927 ↗ | Long Term Follow-up for RGX-202 Duchenne Muscular Dystrophy | — | Enrolling By Invitation | 2029-12-01 |
| NCT07236606 ↗ | RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome) MPS II, Hunter Syndrome (MPS II) | PHASE3 | Active Not Recruiting | 2028-04-30 |
| NCT06103487 ↗ | Long Term Follow-Up for RGX-111 Mucopolysaccharidosis I | — | Enrolling By Invitation | 2027-09-01 |
| NCT05683379 ↗ | AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Males With DMD Duchenne Muscular Dystrophy | — | Recruiting | 2026-09-01 |
| NCT05693142 ↗ | AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD) Duchenne Muscular Dystrophy | PHASE2/PHASE3 | Recruiting | 2026-02-01 |
| NCT03566043 ↗ | CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome) Mucopolysaccharidosis Type II (MPS II) | PHASE2/PHASE3 | Active Not Recruiting | 2023-11-27 |
| NCT03580083 ↗ | RGX-111 Gene Therapy in Patients With MPS I Mucopolysaccharidosis Type I (MPS I), Hurler Syndrome | PHASE1/PHASE2 | Suspended | 2027-07-07 |
| NCT04597385 ↗ | Long-term Follow-Up for RGX-121 Mucopolysaccharidosis II | — | Unknown | 2025-09-01 |
| NCT04591834 ↗ | Mucopolysaccharidosis Type II Observational Mucopolysaccharidosis II | — | Withdrawn | 2025-07-01 |
| NCT04080050 ↗ | A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501 Homozygous Familial Hypercholesterolemia (HoFH) | — | Unknown | 2024-09-29 |
| NCT04462692 ↗ | An Observational Study in Children With CLN2 Batten Disease Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) | — | Withdrawn | 2023-10-01 |
| NCT04571970 ↗ | RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome) Mucopolysaccharidosis Type II (MPS II) | PHASE1/PHASE2 | Completed | 2023-05-09 |
| NCT04480476 ↗ | A Retrospective, Natural History Study in Children With CLN2 Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) | — | Withdrawn | 2022-04-01 |
| NCT03529786 ↗ | Mucopolysaccharidosis Type II Natural History Mucopolysaccharidosis II | — | Completed | 2022-03-22 |
| NCT02651675 ↗ | A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH) Homozygous Familial Hypercholesterolemia (HoFH) | PHASE1/PHASE2 | Terminated | 2020-11-27 |
| NCT04148001 ↗ | Identifying and Genotyping Homozygous Familial Hypercholesterolemia (HoFH) Patients Homozygous Familial Hypercholesterolemia (HoFH) | — | Completed | 2020-04-08 |
| NCT03066258 ↗ | Safety and Tolerability of RGX-314 (Investigational Product) Gene Therapy for Neovascular AMD Trial Neovascular Age-related Macular Degeneration, Wet Age-related Macular Degeneration | PHASE1/PHASE2 | Completed | 2019-11-24 |
| NCT07652606 ↗ | Expanded Access to RGX-202 | — | Temporarily Not Available |
Source: ClinicalTrials.gov (U.S. NIH) · active studies first.
Notable events (8-K)
Recent filings
| Form | Filed | Period | Accession |
|---|---|---|---|
| 8-K | 2026-06-03 | 2026-05-29 | 0001193125-26-254590 ↗ |
| 10-Q | 2026-05-14 | 2026-03-31 | 0001193125-26-222804 ↗ |
| 8-K | 2026-05-14 | 2026-05-14 | 0001193125-26-222768 ↗ |
| 8-K | 2026-03-20 | 2026-03-18 | 0001193125-26-117913 ↗ |
| 10-K | 2026-03-05 | 2025-12-31 | 0001193125-26-094013 ↗ |
| 8-K | 2026-03-05 | 2026-03-05 | 0001193125-26-092566 ↗ |
| 8-K | 2026-02-10 | 2026-02-07 | 0001193125-26-043342 ↗ |
| 8-K | 2026-01-28 | 2026-01-23 | 0001193125-26-026139 ↗ |
| 10-Q | 2025-11-06 | 2025-09-30 | 0001193125-25-268095 ↗ |
| 8-K | 2025-11-06 | 2025-11-06 | 0001193125-25-268101 ↗ |
| 8-K | 2025-08-18 | 2025-08-18 | 0000950170-25-109829 ↗ |
| 10-Q | 2025-08-07 | 2025-06-30 | 0001193125-25-174953 ↗ |