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1. Company Snapshot

1.a. Company Description

REGENXBIO Inc., a clinical-stage biotechnology company, provides gene therapy product candidates to deliver genes to cells to address genetic defects or to enable cells in the body to produce therapeutic proteins or antibodies that are intended to impact disease.Its gene therapy product candidates are based on NAV Technology Platform, a proprietary adeno-associated virus gene delivery platform.The company's lead product candidate is RGX-314, which is in Phase III clinical trial for the treatment of wet age-related macular degeneration.


It is also developing RGX-121 that is in Phase I/II clinical trial to treat mucopolysaccharidosis type II;RGX-111, which is in Phase I/II clinical trial for treating mucopolysaccharidosis type I;RGX-181 which is in pre clinic stage for the treatment of late-infantile neuronal ceroid lipofuscinosis type II disease;RGX-202, to treat Duchenne muscular dystrophy which is in phase I/II clinical trial; and RGX-381, to treat the ocular manifestations of CLN2 disease which is in preclinical stage.REGENXBIO Inc.also licenses its NAV Technology Platform to other biotechnology and pharmaceutical companies; and has a collaboration and license agreement with Neurimmune AG to develop novel gene therapies.


REGENXBIO Inc.was founded in 2008 and is headquartered in Rockville, Maryland.

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1.b. Last Insights on RGNX

Negative drivers behind REGENXBIO Inc.'s recent 3-month performance include: * Weaker-than-expected Q4 2024 earnings, with a loss of $1.01 per share versus the Zacks Consensus Estimate of a loss of $1.27. * Goldman Sachs' downgrade of the company, citing uncertain appeal of gene therapy for wet AMD and diabetic retinopathy due to newer anti-VEGF treatments offering longer-lasting effects at lower costs. * Negative sentiment from investor conferences, including a downgrade by Goldman Sachs and a warning about rival treatments gaining ground. * Uncertainty surrounding the company's gene therapy pipeline, with some investors expressing concerns about the competitive landscape. * A lack of clear catalysts for the stock, with some analysts warning that the company's valuation may be stretched.

1.c. Company Highlights

2. RegenixBio: From Development to Commercial Catalyst

RegenixBio closed 2025 with $170 million in revenue, up 17.7% YoY, while R&D spend reached $228 million, reflecting a 2.6% operating margin. EPS fell to –$1.3 versus the –$1.01 estimate, underscoring the heavy pipeline investment. The company’s P/E sits at –2.35 and its P/B at 4.43, indicating a valuation driven by future milestones rather than current earnings.

Publication Date: Apr -13

📋 Highlights
  • Duchenne Program Progress: RGX-202 completed pivotal study dosing, with topline data expected Q2 2026 and BLA submission via accelerated approval pathway.
  • Retinal Disease Milestones: Subretinal Cirovec topline data for wet AMD expected Q4 2026; NAVIGATE trial triggered $100M AbbVie milestone.
  • Financials: $241M cash balance as of 12/31/2025; $228M R&D expenses, $170M total revenue; cash sufficient through early 2027.
  • MPS Program Hurdles: Received CRL for RGX-121 and clinical holds for RGX-111/RGX-121; plans Type A meeting to resubmit BLA post-additional analysis.
  • Commercial Transition: Aiming to become a commercial entity in 2025, driven by RGX-202 data and regulatory submissions, with 12-month functional data anticipated by fall 2025.

Q4 2025 Financials

Revenue growth was fueled by $50 million in product sales and $120 million from licensing and milestone income. Gross margin hovered around 45%, with SG&A and R&D consuming the majority. Despite a negative operating margin, cash burn remained manageable with $241 million in liquid assets, projecting a runway to early 2027.

Pipeline Highlights

The company’s late‑stage pipeline is accelerating: RGX‑202 for Duchenne DMD completed dosing in its pivotal study, with topline data slated for Q2 and a BLA submission via accelerated approval. In retinal disease, subretinal Cirovec data for wet AMD is due Q4, while the NAVIGATE study for diabetic retinopathy is poised to trigger a $100 million AbbVie milestone.

Duchenne Milestones

RGX‑202’s Phase 1/2 data show no liver injury or thrombocytopenia, a safety edge over existing therapies. The company anticipates 12‑month functional data for most patients by fall, strengthening the case for a pre‑BLA meeting. The FDA may request extended follow‑up, but the firm is prepared to provide it.

MPS Program Status

RegenixBio faced a CRL for RGX‑121 and holds on RGX‑111/121, yet believes the issues are addressable. They are targeting a Type A meeting to resubmit the BLA. Meanwhile, the company addressed variability in heparan sulfate measurements and is ready to present additional analyses.

Cash Position & Funding Outlook

With $241 million in cash and equivalents, the company expects to sustain operations into early 2027, assuming no significant fundraising. The free cash flow yield stands at –27.82%, reflecting the heavy investment in R&D and regulatory milestones. A robust cash runway supports the ambitious 2025 milestone strategy.

3. NewsRoom

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REGENXBIO Inc. Sued for Securities Law Violations - Contact the DJS Law Group to Discuss Your Rights - RGNX

Apr -15

Source: prnewswire.com

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RGNX Deadline Today: RGRX Investors Have Opportunity to Lead REGENXBIO, Inc. Securities Fraud Lawsuit

Apr -14

Source: prnewswire.com

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Bronstein, Gewirtz & Grossman LLC Urges Corcept Therapeutics Incorporated Investors to Act: Class Action Filed Alleging Investor Harm

Apr -14

Source: newsfilecorp.com

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CLASS ACTION DEADLINE TONIGHT: Faruqi & Faruqi, LLP Reminds REGENXBIO (RGNX) Investors of the Securities Class Action Lawsuit Deadline on April 14, 2026

Apr -14

Source: businesswire.com

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Portnoy Law Firm Announces Class Action on Behalf of REGENXBIO, Inc. Investors

Apr -14

Source: globenewswire.com

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RGNX Shareholder Alert: Investors With Losses May Seek to Lead the Class Action in REGENXBIO Inc. Securities Lawsuit -- The Gross Law Firm

Apr -14

Source: prnewswire.com

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REGENXBIO DEADLINE: ROSEN, GLOBAL INVESTOR COUNSEL, Encourages REGENXBIO, Inc. Investors with Losses in Excess of $100K to Secure Counsel Before Important April 14 Deadline in Securities Class Action - RGNX

Apr -13

Source: newsfilecorp.com

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Levi & Korsinsky, LLP: RGNX Disclosure Timeline Reveals Pattern of Alleged Investor Harm

Apr -13

Source: globenewswire.com

4. Business Breakdown

4.a. Revenues by Country

4.b. Revenues by Segment

5. Expected revenues mid-term growth (11.30%)

6. Segments

Gene Therapy

Expected Growth: 11.3%

REGENXBIO's 11.3% growth in Gene Therapy is driven by increasing adoption of novel gene therapies, strong pipeline of rare disease treatments, and strategic partnerships. Growing demand for one-time curative treatments, advancements in gene editing technologies, and expanding regulatory approvals also contribute to the segment's growth.

7. Detailed Products

NAV Technology Platform

A proprietary adeno-associated virus (AAV) gene therapy platform that enables the development of gene therapies for a wide range of diseases and disorders.

RGX-111

A gene therapy for the treatment of Mucopolysaccharidosis Type I (MPS I), a rare genetic disorder.

RGX-121

A gene therapy for the treatment of Mucopolysaccharidosis Type II (MPS II), a rare genetic disorder.

RGX-314

A gene therapy for the treatment of wet age-related macular degeneration (wet AMD), a leading cause of vision loss in older adults.

RGX-501

A gene therapy for the treatment of Homozygous Familial Hypercholesterolemia (HoFH), a rare genetic disorder.

8. REGENXBIO Inc.'s Porter Forces

Forces Ranking

Threat Of Substitutes

REGENXBIO Inc. faces moderate threat from substitutes due to the presence of alternative gene therapy companies, but the company's unique NAV Technology Platform and strong pipeline of products mitigate this threat.

Bargaining Power Of Customers

REGENXBIO Inc. has a diverse customer base, and no single customer accounts for a significant portion of its revenue, reducing the bargaining power of customers.

Bargaining Power Of Suppliers

REGENXBIO Inc. relies on a few key suppliers for raw materials and services, but the company's strong relationships and diversified supply chain mitigate the bargaining power of suppliers.

Threat Of New Entrants

The gene therapy industry is highly competitive, and new entrants with innovative technologies and significant resources could potentially disrupt REGENXBIO Inc.'s market position.

Intensity Of Rivalry

The gene therapy industry is highly competitive, with several established players and new entrants vying for market share, leading to a high intensity of rivalry.

9. SWOT Analysis

Strength

Weaknesses

Opportunities

Threats

10. Capital Structure

10.a. Balance Sheet

10.b. Weighted Average Cost of capital

Value
Debt Weight 22.27%
Debt Cost 7.69%
Equity Weight 77.73%
Equity Cost 10.82%
WACC 10.12%
Leverage 28.64%

11. Quality Control: REGENXBIO Inc. passed 3 out of 9 key points

12.a Historical Valuation

12.b Price/Earnings Ratio

12.c Margin Valuation

12.d Peers Valuation

Peers Group Analysis

Stock-Card
Phathom Pharmaceuticals

A-Score: 4.9/10

Value: 8.2

Growth: 4.6

Quality: 5.8

Yield: 0.0

Momentum: 10.0

Volatility: 1.0

1-Year Total Return ->

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Fortress Biotech

A-Score: 4.7/10

Value: 7.7

Growth: 5.3

Quality: 6.1

Yield: 0.0

Momentum: 7.5

Volatility: 1.3

1-Year Total Return ->

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Adaptive Biotechnologies

A-Score: 4.3/10

Value: 6.0

Growth: 3.8

Quality: 4.7

Yield: 0.0

Momentum: 9.5

Volatility: 1.7

1-Year Total Return ->

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Stock-Card
REGENXBIO

A-Score: 3.8/10

Value: 6.8

Growth: 2.2

Quality: 4.5

Yield: 0.0

Momentum: 7.5

Volatility: 1.7

1-Year Total Return ->

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Stock-Card
Tonix Pharmaceuticals

A-Score: 3.7/10

Value: 7.8

Growth: 5.3

Quality: 3.8

Yield: 0.0

Momentum: 5.0

Volatility: 0.3

1-Year Total Return ->

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Stock-Card
Curis

A-Score: 3.6/10

Value: 9.4

Growth: 3.8

Quality: 5.9

Yield: 0.0

Momentum: 1.5

Volatility: 1.3

1-Year Total Return ->

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Peers Metrics

12.e Scoring Insights

12.f DCF BETA

Parameters

Short Term Growth

Short term Time

Long-Term Growth

WACC

Target Price

9.19$

Current Price

9.19$

Potential

-0.00%

Expected Cash-Flows