- Data underscore the differentiated capability of the clinically validated FORCE platform to cross the blood-brain barrier -
WALTHAM, Mass., April 27, 2026 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company focused on delivering functional improvement for people living with genetically driven neuromuscular diseases, today announced that it will present new preclinical data demonstrating the ability of the FORCE platform to cross the blood-brain barrier (BBB) and enable robust knockdown of MAPT RNA in the central nervous system (CNS) at the American Society of Gene & Cell Therapy (ASGCT) 2026 Annual Meeting being held May 11-15, 2026, in Boston, MA, and virtually.
“While we maintain our focus on advancing our clinical programs in DMD and DM1, as well as our broader neuromuscular pipeline, we are also exploring additional applications where the differentiated capabilities of our FORCE platform, along with further iterations, could enable potentially transformative therapies for patients, including for neurological diseases,” said Oxana Beskrovnaya, Ph.D., chief innovation officer of Dyne. “Building on previous studies, these data highlight the ability of our clinically validated Fab to enable robust activity in the CNS. Additionally, for the first time, we are presenting data showing the potential to further optimize our Fab for enhanced CNS delivery, which resulted in even more robust knockdown of a well-characterized CNS target.”
Preclinical Study Details
- Preclinical studies were performed using two compounds, Conjugate 1 and Conjugate 2, both of which utilize a TfR1-binding antibody fragment (Fab) conjugated to microtubule associated protein tau (MAPT) siRNA designed to downregulate expression of all MAPT isoforms. The pathological accumulation of tau protein is a hallmark of Alzheimer’s disease and other tauopathies.
- Conjugate 1 utilizes the clinically validated FORCE platform with the same TfR1-targeting Fab as Dyne’s clinical programs for Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1) and preclinical programs for facioscapulohumeral muscular dystrophy (FSHD), Pompe disease and multiple DMD mutations. The FORCE platform was designed to deliver robustly to both muscle and the CNS for the treatment of neuromuscular diseases.
- Conjugate 2 utilizes a modified form of the FORCE TfR1-binding Fab that has been further optimized for enhanced CNS delivery and potential use in neurological indications.
- Both conjugates achieved robust MAPT RNA knockdown (approximately 75% for Conjugate 2) in both mice and nonhuman primates (NHPs), with widespread and consistent delivery across brain regions, including the deep brain.
- Subcutaneous administration in mice achieved an equivalent reduction in MAPT RNA as compared to intravenous administration in mice.
- Data for both conjugates will be presented.
- While maintaining its core focus on advancing its clinical programs in DMD and DM1, as well as its preclinical pipeline in neuromuscular diseases, Dyne is currently evaluating potential next steps for the preclinical development of these conjugates with the goal of maintaining capital efficiency and maximizing shareholder value.
ASGCT Oral Presentation Details
Abstract Title: Targeting tauopathies: robust and widespread MAPT silencing in CNS of mice and NHP with TfR1-mediated oligonucleotide delivery
Presentation Date and Time: Wednesday, May 13, 2026, 10:30 a.m. ET
Presenter: Susana Correia, Ph.D., Director, Research, Dyne Therapeutics
The slides from the presentation will be available in the Scientific Publications & Presentations section of Dyne’s website at the commencement of the presentation.
Previous Studies Supporting CNS Delivery and Activity with FORCE Platform
These results build on previous data supporting the ability of the FORCE Platform to cross the BBB using other payloads, as summarized in a recent publication in Nucleic Acid Insights:
- Robust CNS delivery and pharmacodynamic activity with an ASO payload in a DM1 mouse model
- Robust CNS delivery and pharmacodynamic activity, as well as CNS-related functional improvement, with a PMO payload in a DMD mouse model
- Robust CNS delivery and pharmacodynamic activity with an enzyme payload in a Pompe disease mouse model
- Robust CNS delivery with an ASO payload in NHPs
- Meaningful and sustained improvement from baseline at 12 months across 6 subscales of the Myotonic Dystrophy Health Index (MDHI) patient reported outcome measure assessing CNS disease manifestations (cognitive impairment, sleep disturbances, fatigue, communication, emotional issues and pain) in the ACHIEVE clinical trial of z-basivarsen in DM1
About Dyne Therapeutics
Dyne Therapeutics is focused on delivering functional improvement for people living with genetically driven neuromuscular diseases. We are developing therapeutics that target muscle and the central nervous system (CNS) to address the root cause of disease. The company is advancing clinical programs for Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1) as well as preclinical programs for facioscapulohumeral muscular dystrophy (FSHD), Pompe disease and multiple DMD mutations. At Dyne, we are on a mission to deliver functional improvement for individuals, families and communities. Learn more at https://www.dyne-tx.com/, 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 therapeutic potential of the FORCE platform and its derivatives, and the ability of Dyne’s MAPT Conjugate 1 and Conjugate 2 to cross the blood-brain barrier, apply to neurological indications or to achieve MAPT knockdown, 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,” “will,” 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 initial data from early clinical trials will be predictive of the final results of the clinical trials or future trials or longer-term performance than is measured in the clinical trial; 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 the regulatory approval process, including the availability of accelerated approval pathways; 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-K 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:
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781-317-0353
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