Dyne Therapeutics to Present New Preclinical Data in Facioscapulohumeral Muscular Dystrophy at the FSHD Society International Research Congress

Rhea-AI Summary

Dyne Therapeutics announced new preclinical data for DYNE-302 in treating facioscapulohumeral muscular dystrophy (FSHD), to be presented at the FSHD Society's International Research Congress in Amsterdam. The study showed promising results in a severe FSHD mouse model, where a single intravenous dose of DYNE-302 restored treadmill running ability and corrected muscle damage and inflammation markers. DYNE-302 uses TfR1-targeting technology to deliver siRNA specifically targeting DUX4 mRNA, a key factor in FSHD disease progression. The data suggests potential reversibility of severe skeletal muscle disease through DUX4 mRNA targeting. The presentation by Dr. Stefano Zanotti will take place on June 13, 2025, focusing on functional improvements and muscle transcriptomic changes in mouse models.

Insights

Biotechnology Research Analyst

Positive preclinical data for Dyne's FSHD therapy shows disease reversal potential, advancing their neuromuscular disease pipeline toward clinical validation.

Dyne Therapeutics has generated compelling preclinical data for DYNE-302, their candidate therapy for facioscapulohumeral muscular dystrophy (FSHD). The results demonstrate remarkable functional recovery in a severe FSHD mouse model, where a single intravenous dose restored treadmill running ability - suggesting potential for reversing existing muscle damage rather than merely halting progression.

The molecular mechanism appears sound: DYNE-302 uses a TfR1-targeting Fab to deliver an siRNA payload specifically targeting DUX4 mRNA. This approach directly addresses FSHD's root cause, as DUX4 de-repression drives muscle damage and functional loss. Gene activity analysis showed correction of muscle damage and inflammation markers, providing mechanistic validation.

What sets this data apart is the demonstration of functional improvement rather than just biomarker changes. For neuromuscular disorders, functional outcomes are the gold standard, and showing that severely weakened muscles can regain function significantly strengthens DYNE-302's therapeutic potential.

This adds substantial value to Dyne's neuromuscular disease pipeline. While still preclinical, these results provide important proof-of-concept for their broader platform technology that combines targeted delivery with RNA modulation. For FSHD patients, who currently have no approved therapies, this represents a potential breakthrough addressing an unmet medical need with high disease burden.

- DYNE-302 Demonstrated Functional Improvement in an FSHD Preclinical Model -

WALTHAM, Mass., June 05, 2025 (GLOBE NEWSWIRE) --  Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company focused on advancing life-transforming therapeutics for people living with genetically driven neuromuscular diseases, today announced that it will be presenting new preclinical data demonstrating the potential of DYNE-302 to achieve functional improvement in facioscapulohumeral muscular dystrophy (FSHD). The data will be presented at the 32^nd Annual FSHD Society’s International Research Congress being held June 12-13, 2025, in Amsterdam.

In a mouse model of severe FSHD, a single intravenous dose of DYNE-302 administered at the peak of muscle weakness restored ability to run on a treadmill. Analysis of gene activity in skeletal muscle indicated correction of muscle damage and inflammation. These findings suggest that preexisting and severe skeletal muscle disease in FSHD has the potential to be reversed by targeting the DUX4 mRNA with DYNE-302. 

FSHD is a rare, progressive, inherited muscle disease. De-repression of DUX4 in skeletal muscle drives disease pathogenesis, leading to muscle damage and loss of function. This results in a range of symptoms that restrict daily activities and have a high physical, emotional, and financial burden. DYNE-302 leverages a TfR1-targeting Fab for muscle delivery of an siRNA payload highly specific for DUX4 mRNA with the aim of suppressing DUX4 expression and the downstream DUX4 transcriptome.

Oral Presentation: DYNE-302 leads to functional improvement and resolves muscle transcriptomic changes in mouse models of FSHD
Session: Mechanisms of Disease & Interventional Strategies
Date/Time: Friday, June 13, 2025, at 12:00 p.m. CEST / 6:00 a.m. ET.
Presenter: Stefano Zanotti, PhD, Head of Neuromuscular Research, Dyne

The presentation will also be available in the Scientific Publications & Presentations section of Dyne’s website following the session.

About Facioscapulohumeral Muscular Dystrophy (FSHD)

FSHD is a rare, progressive, genetic disease caused by a mutation in the DUX4 gene, leading to skeletal muscle loss, muscle weakness and wasting. Individuals with FSHD carry a genetic mutation that allows the DUX4 gene to be sporadically activated in muscle cells, causing their gradual destruction throughout the body. People living with FSHD experience weakness in all major muscle groups throughout the body and limited mobility. An estimated 16,000 to 38,000 individuals in the United States and approximately 35,000 in Europe are affected by FSHD, but there are currently no approved therapies.

About Dyne Therapeutics

Dyne Therapeutics is discovering and advancing innovative life-transforming therapeutics for people living with genetically driven neuromuscular diseases. Leveraging the modularity of its FORCE™ platform, Dyne is developing targeted therapeutics that deliver to muscle and the central nervous system (CNS). Dyne has a broad pipeline for neuromuscular diseases, including clinical programs for myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy (DMD) and preclinical programs for facioscapulohumeral muscular dystrophy (FSHD) and Pompe disease. For more information, please visit and follow us on X, LinkedIn and Facebook.

Forward-Looking Statements

This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this press release, including statements regarding Dyne’s strategy, future operations, prospects and plans, objectives of management, the potential of the FORCE platform, the potential of DYNE-302, and the sufficiency of Dyne’s cash resources for the period anticipated, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “might,” “objective,” “ongoing,” “plan,” “predict,” “project,” “potential,” “should,” or “would,” or the negative of these terms, or other comparable terminology are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Dyne may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various important factors, including: uncertainties inherent in the identification and development of product candidates, including the initiation and completion of preclinical studies and clinical trials; uncertainties as to the availability and timing of results from preclinical studies and clinical trials; the timing of and Dyne’s ability to enroll patients in clinical trials; whether results from preclinical studies and data from clinical trials will be predictive of the final results of the clinical trials or other trials; whether data from clinical trials will support submission for regulatory approvals; uncertainties as to the FDA’s and other regulatory authorities’ interpretation of the data from Dyne's clinical trials and acceptance of Dyne's clinical programs and as to the regulatory approval process for Dyne’s product candidates; whether Dyne’s cash resources will be sufficient to fund its foreseeable and unforeseeable operating expenses and capital expenditure requirements; as well as the risks and uncertainties identified in Dyne’s filings with the Securities and Exchange Commission (SEC), including the company’s most recent Form 10-Q and in subsequent filings Dyne may make with the SEC. In addition, the forward-looking statements included in this press release represent Dyne’s views as of the date of this press release. Dyne anticipates that subsequent events and developments will cause its views to change. However, while Dyne may elect to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Dyne’s views as of any date subsequent to the date of this press release.

Contacts:

Investors
Mia Tobias
ir@dyne-tx.com
781-317-0353  

Media
Stacy Nartker
snartker@dyne-tx.com
781-317-1938

FAQ

What are the key findings of Dyne Therapeutics' DYNE-302 FSHD preclinical study?

The study showed that a single intravenous dose of DYNE-302 restored treadmill running ability in severe FSHD mouse models and corrected muscle damage and inflammation markers.

How does Dyne Therapeutics' DYNE-302 work in treating FSHD?

DYNE-302 uses TfR1-targeting Fab technology to deliver siRNA specifically targeting DUX4 mRNA in skeletal muscle, aiming to suppress DUX4 expression and its downstream effects.

When will DYN present their FSHD research findings?

Dyne Therapeutics will present their findings at the FSHD Society's International Research Congress on June 13, 2025, at 12:00 p.m. CEST in Amsterdam.

What is the significance of targeting DUX4 in FSHD treatment?

DUX4 de-repression in skeletal muscle drives FSHD disease progression, causing muscle damage and loss of function. Targeting it could potentially reverse severe skeletal muscle disease.

What is FSHD and how does it affect patients?

FSHD is a rare, progressive, inherited muscle disease that causes muscle damage and functional loss, resulting in restricted daily activities and significant physical, emotional, and financial burden.