REGENXBIO Announces Presentations at the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference

REGENXBIO (Nasdaq: RGNX) announced presentations on its investigational Duchenne gene therapy RGX-202 at the 2026 Muscular Dystrophy Association Clinical & Scientific Conference in Orlando, March 8-11, 2026.

Rhea-AI Summary

REGENXBIO (Nasdaq: RGNX) announced presentations on its investigational Duchenne gene therapy RGX-202 at the 2026 Muscular Dystrophy Association Clinical & Scientific Conference in Orlando, March 8-11, 2026.

Events include an interim Phase I/II podium presentation on March 11, a preclinical poster, and a company-hosted symposium on March 9 at 12 p.m. ET. Presentation materials will be posted in the company's Publications section.

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Key Figures

Conference dates: March 8-11, 2026 Podium session time: March 11, 2026; 11:45 a.m. ET Phase designation: Phase I/II +1 more

Conference dates

March 8-11, 2026

MDA Clinical & Scientific Conference schedule in Orlando, FL

Podium session time

March 11, 2026; 11:45 a.m. ET

Interim Phase I/II RGX-202 clinical data presentation slot

Phase designation

Phase I/II

Clinical safety, biomarker, and functional data for RGX-202

Symposium time

March 9, 2026; 12 p.m. ET

Company-hosted symposium on Duchenne gene therapy trials

Historical Context

5 past events · Latest: Mar 02

5 events

1. Mar 02

   Investor conferences

   24h Move

   +2.2%

   Participation in three March 2026 healthcare investor conferences with webcasts.

2. Feb 25

   Earnings call notice

   24h Move

   +5.6%

   Scheduled March 5, 2026 call for Q4 and full-year 2025 results.

3. Feb 09

   CRL for RGX-121

   24h Move

   -14.2%

   FDA Complete Response Letter citing multiple concerns for RGX-121 BLA.

4. Jan 29

   Disclosure investigation

   24h Move

   -3.8%

   Announcement of investigation into adequacy of company disclosures before FDA action.

5. Jan 28

   Clinical holds MPS trials

   24h Move

   -17.9%

   FDA clinical holds on RGX-111 and RGX-121 after CNS tumor case.

24h Move is the share-price change in the day after each event; other market factors may also have contributed.

Key Terms

gene therapy, duchenne muscular dystrophy, biomarker, phase i/ii, +2 more

6 terms

gene therapy medical

"RGX-202 investigational gene therapy for Duchenne muscular dystrophy at the 2026 Muscular"

Gene therapy is a medical technique that involves altering or replacing faulty genes in a person's cells to treat or prevent disease. It is considered a promising area of innovation because it has the potential to provide long-term or even permanent solutions to genetic conditions. For investors, advancements in gene therapy can signal opportunities in biotech companies and emerging treatments with significant growth potential.

duchenne muscular dystrophy medical

"RGX-202 investigational gene therapy for Duchenne muscular dystrophy at the 2026 Muscular"

A rare, inherited condition that progressively weakens muscles, Duchenne muscular dystrophy causes the body’s muscle fibers to break down over time, often leading to severe disability. For investors, it matters because the small, well-defined patient population, high unmet medical need and complex regulatory and pricing dynamics mean successes or failures in clinical trials, approvals, or therapies can have outsized effects on a company’s valuation and future revenue prospects.

biomarker medical

"Presentations include preclinical and Phase I/II clinical safety, biomarker, and functional data."

A biomarker is a measurable indicator found in the body, such as in blood or tissues, that provides information about health, disease, or how the body responds to treatment. For investors, biomarkers can signal the potential success or risk of medical products or therapies, influencing the value of related companies and industry trends. They act like signals or clues that help assess the progress of medical advancements and their market impact.

phase i/ii medical

"Presentations include preclinical and Phase I/II clinical safety, biomarker, and functional data."

"Phase I/II" describes early stages of testing a new medicine or treatment, where researchers first evaluate its safety and then begin to see if it works. For investors, these phases are important because they indicate whether a product is progressing toward potential approval and commercialization, which can impact future value and success prospects. These stages help gauge how close a new treatment is to reaching the market.

microdystrophin medical

"Title: Microdystrophin with an extended C-Terminal domain protects against pharmacologica"

A microdystrophin is a deliberately shortened, functional version of the dystrophin protein or its gene that is used in gene therapies to replace or mimic the missing protein in disorders like Duchenne muscular dystrophy. Think of it as a compact, working blueprint small enough to fit into a delivery vehicle that restores some muscle function; investors watch its safety, durability, and manufacturing because those determine clinical success, approval and commercial potential.

mdx mice medical

"domain protects against pharmacologically induced cardiac damage and remodeling in mdx mice"

Mdx mice are lab-bred mice that carry a genetic defect causing loss of dystrophin, a protein that helps muscle cells stay intact; they serve as a standard animal model for Duchenne muscular dystrophy. For investors, positive results in mdx mice act like a crash-test for experimental therapies—providing early, live-animal evidence that a treatment can improve muscle structure or function and helping to de-risk and prioritize funding before costly human trials.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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ROCKVILLE, Md., March 4, 2026 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced presentations on its RGX-202 investigational gene therapy for Duchenne muscular dystrophy at the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, taking place in Orlando, FL March 8-11, 2026.

Presentations include preclinical and Phase I/II clinical safety, biomarker, and functional data.

Podium Presentation:
Title: RGX-202, An Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy: Interim Phase I/II Clinical Data (P432 O)
Session: Clinical Trial Updates
Date/Time: March 11, 2026; 11:45 a.m. ET
Presenter: Carolina Tesi Rocha, M.D., Clinical Professor, Neurology, Stanford School of Medicine, Stanford Children's Health

Poster Presentation:
Title: Microdystrophin with an extended C-Terminal domain protects against pharmacologically induced cardiac damage and remodeling in mdx mice (P173 M)
Session: Pre-Clinical Research
Presenter: Steven Foltz, Ph.D., Principal Scientist, Gene Therapy Research at REGENXBIO

The presentations will be available in the Publications section of REGENXBIO's website.

REGENXBIO will also host a symposium titled, "Advancing Duchenne Gene Therapy Trials in a New Era: Optimizing Design and Interpretation." The event will feature leading experts and take place March 9, 2026 at 12 p.m. ET.

ABOUT REGENXBIO Inc.
REGENXBIO is a biotechnology company on a mission to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the field of AAV gene therapy. REGENXBIO is advancing a late-stage pipeline of one-time treatments for rare and retinal diseases, including RGX-202 for the treatment of Duchenne; clemidsogene lanparvovec (RGX-121) for the treatment of MPS II and RGX-111 for the treatment of MPS I, both in partnership with Nippon Shinyaku; and surabgene lomparvovec (ABBV-RGX-314) for the treatment of wet AMD and diabetic retinopathy, in collaboration with AbbVie. Thousands of patients have been treated with REGENXBIO's AAV platform, including those receiving Novartis' ZOLGENSMA®. REGENXBIO's investigational gene therapies have the potential to change the way healthcare is delivered for millions of people. For more information, please visit WWW.REGENXBIO.COM.

Contacts:
Dana Cormack
Corporate Communications
dcormack@regenxbio.com 

Investors:
George E. MacDougall
Investor Relations
IR@regenxbio.com

 

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SOURCE REGENXBIO Inc.

FAQ

AI-generated questions and answers. How Rhea-AI works. Not financial advice.

When will REGENXBIO (RGNX) present RGX-202 clinical data at the MDA 2026 conference?

REGENXBIO will present interim Phase I/II clinical data for RGX-202 on March 11, 2026 at 11:45 a.m. ET. According to REGENXBIO, the podium covers safety, biomarker, and functional interim results from the Phase I/II study.

What RGX-202 preclinical research will REGENXBIO present at the MDA 2026 poster session?

REGENXBIO will present a preclinical poster on microdystrophin with an extended C‑terminal domain protecting against cardiac damage. According to REGENXBIO, the poster reports results from mdx mouse pharmacologic cardiac-damage models.

Who will present REGENXBIO's RGX-202 podium session at the MDA 2026 meeting?

Carolina Tesi Rocha, M.D., will present the RGX-202 interim Phase I/II clinical data on March 11, 2026. According to REGENXBIO, Dr. Rocha is listed as presenter and is affiliated with Stanford School of Medicine and Stanford Children's Health.

When and what is REGENXBIO's symposium at the MDA 2026 conference?

REGENXBIO will host a symposium titled "Advancing Duchenne Gene Therapy Trials in a New Era" on March 9, 2026 at 12 p.m. ET. According to REGENXBIO, it will feature leading experts on trial design and interpretation.

Where can investors access REGENXBIO (RGNX) presentations from the MDA 2026 conference?

Presentations will be posted in the Publications section of REGENXBIO's website for later access. According to REGENXBIO, both the podium and poster materials will be made available online after the conference.