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Minimal debt; primarily equity-financed with ~$400M cash runway; 20% rate rise has negligible direct debt service impact

Low direct sensitivity; higher rates increase opportunity cost of cash holdings and compress biotech valuations via discount rate expansion

Low intensity; China and India supply >60% of global API inputs, creating concentration risk.

AAV vector manufacturing reagents concentrated in US/EU suppliers; plasmid DNA upstream inputs dependent on limited CMO capacity globally

Minimal international commercial revenue; primarily US-focused; currency devaluation exposure near zero in current pre-commercial stage

Overwhelmingly US-based revenue and operations; EU regulatory submissions ongoing but no material ex-US commercial revenue yet

Low intensity; commodities: Active Pharmaceutical Ingredients, Rare Earth Elements, Natural Gas (energy), Plastics/Resins, Ethanol, Packaging Materials; geopolitical: China and India supply >60% of global API inputs, creating concentration risk.

High dependency on single-source AAV capsid manufacturing CMOs; no single vendor >30% cost but switching risk for GMP biologics is severe

Clinical-stage gene therapy biotech; cloud disruption would impair research informatics but not core wet-lab manufacturing or clinical operations

Hybrid licensing (NAV Technology Platform royalties) plus proprietary pipeline development; licensing revenue partially de-risks cloud dependency

Low cloud API coupling risk; research informatics standard; primary operational risk is GMP CMO switching, not cloud provider switching

Revenue model is product/license-based, not investment contracts; Howey Test not applicable; low securities law reclassification risk

Very High

Limited consumer data processed; patient clinical trial data subject to HIPAA, GCP regulations; GDPR exposure modest given minimal EU commercial operations

Low antitrust risk; niche ultra-rare disease focus; NAV platform licensing is non-exclusive; market share insufficient to trigger scrutiny

Very High burden; regimes: FDA, CMS, HHS, HIPAA, EMA, OIG; Drug pricing legislation and approval delays directly compress revenue visibility.

~80% transactional/milestone-based licensing revenue; ~20% recurring royalties from AbbVie/Ultragenyx deals; predominantly non-subscription

Dual vector: upfront licensing fees plus milestone payments from partners, and future product sales royalties on approved therapies

Gene therapy one-time treatment pricing ($1M–$3M range expected); pricing supported by durable cure value proposition but payer access risk high

Moderate-high for approved rare disease therapies; pricing constrained by payer/ICER scrutiny and small patient populations limiting volume leverage

Target gross margins 70–85% on licensing/royalty revenue; product gross margins post-launch estimated 60–75% given high COGS for gene therapies

No free-rider problem; proprietary NAV capsid IP creates hard barriers; no meaningful open-source alternative to licensed AAV platform

Revenue growth is sublinear to headcount; licensing royalties scale without proportional headcount addition; manufacturing scaled via CMO partners

Near-zero marginal cost on royalty revenue stream; pipeline advancement requires incremental R&D headcount but commercial scaling is largely outsourced

Not applicable; REGENXBIO does not operate a franchise network

At 10x scale, CAC remains low for licensing (B2B pharma deals); patient identification costs for ultra-rare diseases are high per patient

Weak direct network effects; indirect platform effect as more licensees validate NAV technology, attracting further partners and clinical data

-42.8% Return on Assets (Negative equity)

Moderate resilience; rare genetic disease treatments medically necessary but reimbursement risk and capital market dependency create recession vulnerability

Large pharmaceutical/biotech licensees (AbbVie, Ultragenyx) seeking AAV gene therapy platform access; high concentration in few enterprise partners

Ultra-rare disease patients (wet AMD, MPS, PKU indications); very small addressable populations with no substitutable curative alternatives

["11-0000 Management Occupations", "13-0000 Business and Financial Operations Occupations", "15-0000 Computer and Mathematical Occupations", "19-0000 Life, Physical, and Social Science Occupations", "21-0000 Community and Social Service Occupations", "23-0000 Legal Occupations", "29-0000 Healthcare Practitioners and Technical Occupations", "31-0000 Healthcare Support Occupations", "41-0000 Sales and Related Occupations", "43-0000 Office and Administrative Support Occupations", "51-0000 Production Occupations"]

0.32 (HIL — ~32% of characteristic roles agent-automatable)

1.4% CapEx / Revenue (Low-CapEx Asset-Light)

0001590877

RGNX