REGENXBIO

REGENXBIO

Develops AAV gene delivery therapies

Overview

REGENXBIO focuses on gene therapy using its NAV Technology Platform, an adeno-associated virus (AAV) gene delivery system with exclusive rights to more than 100 AAV vectors. The company generates revenue by licensing its platform, collecting royalties, and engaging in collaborations, while also developing its own one-time gene therapies for retinal, rare, and neurodegenerative diseases. Key products in later-stage trials include ABBV-RGX-314 for wet AMD and diabetic retinopathy, RGX-202 for Duchenne muscular dystrophy, and RGX-121 for Hunter syndrome, with a goal to advance five new therapies to pivotal-stage or commercial status by 2025 and FDA review for RGX-121 targeted in early 2026. REGENXBIO differentiates itself by owning a broad AAV vector library and offering end-to-end capabilities—from research to manufacturing—alongside a diversified revenue model that includes royalties, collaborations, and milestones.

About REGENXBIO

Simplify's Rating
Why REGENXBIO is rated
C+
Rated B on Competitive Edge
Rated B on Growth Potential
Rated D+ on Differentiation

Industries

Biotechnology

Healthcare

Company Size

201-500

Company Stage

IPO

Headquarters

Rockville, Maryland

Founded

2009

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Simplify's Take

What believers are saying

  • RGX-202 met its pivotal endpoint, with 93% exceeding the microdystrophin threshold.
  • AFFINITY DUCHENNE showed statistically significant functional gains and favorable one-year safety.
  • RGX-121 and ABBV-RGX-314 provide additional 2026 catalysts beyond Duchenne.

What critics are saying

  • FDA clinical holds on RGX-111 and RGX-121 undermine confidence in REGENXBIO’s AAV safety.
  • Zolgensma royalties remain concentrated, and any Novartis pressure would hit cash generation.
  • Cash of $150.5 million funds operations only into early 2027, forcing financing soon.

What makes REGENXBIO unique

  • REGENXBIO’s NAV platform owns rights to more than 100 engineered AAV vectors.
  • RGX-202 uses AAV8 and a novel microdystrophin retaining the C-terminal domain.
  • ABBV-RGX-314 and RGX-121 extend the platform across retina and lysosomal diseases.

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Funding

Total Funding

$1.2B

Above

Industry Average

Funded Over

11 Rounds

Post IPO Debt funding comparison data is currently unavailable. We're working to provide this information soon!
Post IPO Debt Funding Comparison
Coming Soon

Benefits

401(k) Retirement Plan

401(k) Company Match

Stock Options

Performance Bonus

Professional Development Budget

Stock Price

Growth & Insights and Company News

Headcount

6 month growth

1%

1 year growth

1%

2 year growth

-1%
Wallowa.com
Mar 19th, 2026
RGNX investors have opportunity to lead REGENXBIO Inc. securities fraud lawsuit with the Schall Law Firm.

RGNX investors have opportunity to lead REGENXBIO Inc. securities fraud lawsuit with the Schall Law Firm. GlobeNewswire | Schall Law Today at 8:30am PDT LOS ANGELES, March 19, 2026 (GLOBE NEWSWIRE) - The Schall Law Firm, a national shareholder rights litigation firm, reminds investors of a class action lawsuit against REGENXBIO Inc. ("Regenxbio" or "the Company") (NASDAQ: RGNX) for violations of §§10(b) and 20(a) of the Securities Exchange Act of 1934 and Rule 10b-5 promulgated thereunder by the U.S. Securities and Exchange Commission. Investors who purchased the Company's securities between February 9, 2022 and January 27, 2026, inclusive (the "Class Period"), are encouraged to contact the firm before April 14, 2026. Wallowa also encourage you to contact Brian Schall of the Schall Law Firm, 2049 Century Park East, Suite 2460, Los Angeles, CA 90067, at 310-301-3335, to discuss your rights free of charge. You can also reach Wallowa through the firm's website at www.schallfirm.com, or by email at [email protected]. The class, in this case, has not yet been certified, and until certification occurs, you are not represented by an attorney. If you choose to take no action, you can remain an absent class member. According to the Complaint, the Company made false and misleading statements to the market. Regenxbio's statements to investors about product candidate RGX-111 were overwhelmingly positive while it concealed negative data on its efficacy and safety. The Company then revealed an intraventricular CNS tumor was discovered in a participant treated as part of a RGX-111 study. Based on these facts, the Company's public statements were false and materially misleading throughout the class period. When the market learned the truth about Regenxbio, investors suffered damages. The Schall Law Firm represents investors around the world and specializes in securities class action lawsuits and shareholder rights litigation. The Schall Law Firm This is a paid placement. For further inquiries, please contact GlobeNewswire directly.

pharmaphorum
Mar 13th, 2026
Regenxbio, Solid report data with Duchenne gene therapies

Regenxbio, Solid report data with Duchenne gene therapies. This week saw both Regenxbio and Solid Bio report new data with their gene therapy candidates for Duchenne muscular dystrophy (DMD), vying to join Sarepta's Elevidys on the market. Regenxbio gave an update on the phase 2/3 AFFINITY DUCHENNE trial of its RGX-202, reporting interim results that showed it was well tolerated, achieved robust expression of microdystrophin to replace the mutated dystrophin protein in DMD, and led to early functional improvements in ambulatory DMD patients aged one and over. The data, from seven subjects in the study treated with the target dose of RGX-202, showed that their motor function 12 months later - measured using the North Star Ambulatory Assessment (NSAA) scale and timed function tests - was improved compared to matched external natural history comparisons and predictive modelling. The results also showed that patients demonstrated cardiac stability at one year post-treatment as measured by MRI endpoints, an important finding as DMD causes progressive cardiac muscle degeneration (cardiomyopathy). The company is now expecting to report pivotal data from the study in the second quarter of this year, and said it will ask for a meeting with the FDA to look at possible pathways toward filing RGX-202 for accelerated approval while it runs an ongoing confirmatory study. Solid Bio, meanwhile, reported this week that its SGT-003 candidate had also achieved strong expression of microdystrophin, along with improvements in muscle integrity, including stabilisation and improvement in cardiac function in its phase 1/2 INSPIRE DUCHENNE trial. For now, the investigators remain blinded to the motor function data in the study, which has now enrolled 40 subjects. Solid Bio said it is hoping to schedule meetings with the FDA in the first half of this year to seek guidance on accelerated approval, and indicated that it already has an agreement with the agency on the design of a phase 3 trial that will be called IMPACT DUCHENNE and is due to start dosing patients before the end of this month. Both companies are hoping to provide an alternative one-shot treatment to Elevidys, which was launched in 2023, but ran into trouble last year after three fatalities were reported in non-ambulatory DMD recipients, leading to a pause in distribution. The fatalities, linked to liver toxicity, eventually resulted in Elevidys' label being restricted to ambulatory patients only, but did not stop the product knocking on the door of blockbuster status last year, with sales of $899 million. Regenxbio and Solid Bio have both pointed to the safety profile of their gene therapies, saying there was no evidence of liver injury in their trial updates. The two studies were presented at the 2026 Muscular Dystrophy Association (MDA) annual meeting earlier this week. 13 March, 2026

GlobeNewswire
Feb 16th, 2026
Gainey McKenna & Egleston Announces A Class Action Lawsuit Has Been Filed Against REGENXBIO Inc. (RGNX)

Gainey McKenna & Egleston announces A class action lawsuit has been filed against REGENXBIO Inc. (RGNX). NEW YORK, Feb. 16, 2026 (GLOBE NEWSWIRE) - Gainey McKenna & Egleston announces that a securities class action lawsuit has been filed in the United States District Court for the District of Maryland on behalf of all persons or entities who purchased or otherwise acquired REGENXBIO Inc. ("REGENXBIO" or the "Company") (NASDAQ: RGNX) securities between February 9, 2022 and January 27, 2026, inclusive (the "Class Period"). The Complaint alleges that Defendants provided overwhelmingly positive statements to investors while, at the same time, disseminating false and misleading statements and/or concealing material adverse facts concerning the efficacy and safety of its RGX-111 trial study. The Complaint further alleges that the truth began to emerge on January 28, 2026, when REGENXBIO issued a press release announcing that the FDA placed a clinical hold on its investigational gene therapy RGX-111. The Complaint alleges that Defendants announced that an intraventricular CNS tumor was found in a participant treated in its RGX-111 Phase I/II study. The Complaint continues to allege that investors and analysts reacted immediately to REGENXBIO's revelation. The price of the Company's common stock declined from a closing market price of $13.41 per share on January 27, 2026; the Company's stock price fell to $11.01 per share on January 28, 2026, a decline of 17.8% in the span of just a single day. Investors who purchased or otherwise acquired shares of REGENXBIO should contact the Firm prior to the April 14, 2026 lead plaintiff motion deadline. A lead plaintiff is a representative party acting on behalf of other class members in directing the litigation. If you wish to discuss your rights or interests regarding this class action, please contact Thomas J. McKenna, Esq. or Gregory M. Egleston, Esq. of Gainey McKenna & Egleston at (212) 983-1300, or via e-mail at [email protected] or [email protected]. Please visit our website at http://www.gme-law.com for more information about the firm. Company Profile Gainey McKenna & Egleston Industry: Consumer Services Recommended reading. Explore. February 16, 2026 11:00 ET February 16, 2026 11:07 ET February 16, 2026 11:01 ET February 16, 2026 11:00 ET February 16, 2026 11:07 ET February 16, 2026 11:01 ET February 16, 2026 11:00 ET

Pipeline Review
Aug 7th, 2025
REGENXBIO Announces Pivotal Program for Surabgene Lomparvovec in Diabetic Retinopathy

REGENXBIO announces Pivotal Program for Surabgene Lomparvovec in Diabetic Retinopathy.

PR Newswire
May 19th, 2025
REGENXBIO Announces Strategic Royalty Monetization Agreement for Up to $250 Million

/PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced the closure of a non-dilutive, limited recourse royalty bond agreement of up to $250 million with...

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