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REGENXBIO Inc.

regenxbio.com

What Do They Do?

Develops one-time gene therapies for people with retinal, neuromuscular and neurological diseases, and licenses its gene-delivery platform to biopharmaceutical companies.

How Do They Make Money?

Biopharmaceutical partners pay upfront and annual fees for licenses, milestones when development targets are achieved, royalties when licensed products sell, and sublicense fees when licensees receive them. They pay for development and manufacturing services as the work is performed.

revenue
SegmentShareRevenue
License and royalty
Licensed

92%
FY2022: 100%
Service
Recurring
8.3%

Who Are Their Customers?

Biopharmaceutical companies, including Novartis Gene Therapies and Nippon Shinyaku, contract with REGENXBIO through license and collaboration agreements for platform rights, therapy development and related services.

Do Revenues Turn Into Profits?

No. It lost $193.9M in FY2025.

Operating income, FY2025
−95% of revenue
▼ −31% (Loss) vs FY2024 (−$233.3M, −280%)
Net income, FY2025
−114% of revenue
▼ −15% (Loss) vs FY2024 (−$227.1M, −273%)
Free cash flow, FY2025
−74% of revenue
▼ −28% (Loss) vs FY2024 (−$175.6M, −211%)

How Are They Changing?

Since the FY2025 annual report
Latest quarter: Q2 FY2026, the three months to Jun 30, 2026
Revenue
▲ +406%
vs $21.4M in Q2 FY2025
Net income
▲ Now profitable
vs −$70.9M in Q2 FY2025
License and royalty share of revenue vs 86.5% in Q2 FY2025
  • Aug 24, 2026 FDA places RGX-121 on clinical hold; no near-term BLA resubmission expected Spine MRI findings in five study participants prompted the hold; REGENXBIO and its partner are reviewing follow-up information.

Shrinking. Revenue fell 64% in 4 years, from $470.3M in FY2021 to $170.4M in FY2025, about 22% a year.

Revenue over time
License and royalty Service
$500M$250M$0

Who Do They Compete With?

Competes with 4D Molecular Therapeutics, Amicus Therapeutics, Astellas Pharma, BioMarin Pharmaceutical, Lilly, MeiraGTx, Novartis, Passage Bio, PTC Therapeutics, Roche, Sanofi, Sarepta Therapeutics, Solid Biosciences, Taysha Gene Therapies, Tenaya Therapeutics, Ultragenyx Pharmaceutical, and uniQure

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