Dyne Therapeutics to Present New Preclinical Data in Facioscapulohumeral Muscular Dystrophy at the FSHD Society International Research Congress
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 32nd 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 FSHDSession: Mechanisms of Disease & Interventional StrategiesDate/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:
InvestorsMia Tobiasir@dyne-tx.com781-317-0353
MediaStacy Nartkersnartker@dyne-tx.com781-317-1938Sign in to access your portfolio
Hashtags
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Business Upturn
3 hours ago
- Business Upturn
Clarification on Leadership Announcement: Annette Slyman not to join Life Backup Plan as Chief Operating Officer
Aliso Viejo, CA, July 11, 2025 (GLOBE NEWSWIRE) — Galacxia, Inc., the developer of the Life Backup Plan app, is issuing a clarification to our July 10, 2025 press release. Annette Slyman will not be joining the team in any capacity, due to extenuating circumstances. Plans evolve. Our mission stays the course. We continue to hold Ms. Slyman in the highest regard. Galacxia remains committed to delivering innovative digital health and safety solutions that improve lives and support communities nationwide. About Life Backup Plan by Galacxia, Inc. Life Backup Plan is an end-to-end safety, emergency detection, digital health and multi-faceted care app that reduces tragedies, improves outcomes, lowers medical costs, and eases the burden on families and the healthcare system. Unlike other health and personal safety systems that require pressing a button to summon help, Life Backup Plan proactively checks on people, shares critical health data, coordinates support, and handles everything from emergency response to long-term caregiving, end-of-life planning, and daily logistics. We're delivering a comprehensive solution that bridges fragmented systems, supports value-based care, and brings modern tools into a system still largely reliant on fax machines.. Galacxia, Inc. is a SBA certified Woman-Owned Small Business and Delaware C Corporation committed to transforming personal safety, emergency response, and health access and equity. Eyes on the stars. Hands on the wheel. Heart in the mission. Press inquiries Life Backup Plan by Galacxia, Inc Sandy Eulitt [email protected] 8588480860 49 El Prado Lane, Oceanside, CA 92058
Business Upturn
3 hours ago
- Business Upturn
The Texas Chiropractic Association (TCA) Recognizes Parker University and Several Alumni at Chiro Texpo 2025
By GlobeNewswire Published on July 12, 2025, 02:43 IST Dallas, Texas, July 11, 2025 (GLOBE NEWSWIRE) — The Texas Chiropractic Association (TCA) recently recognized chiropractic leaders during Chiro Texpo 2025, the annual state chiropractic convention, which was held at the Hyatt Regency Frisco-Dallas in Frisco. TCA is the voice for chiropractic in Texas, providing protection, state-wide advocacy, information, and education for more than 7,000 DCs who are licensed to practice in Texas. TCA advocates the position that citizens of Texas should have adequate access to the healthcare of their choice and that DCs are adequately represented in the government's decision-making process. Awards were presented to Texas chiropractors and other chiropractic supporters at the President's Gala, with Parker University alumni walking away with awards from key categories. Parker University Chiro Texpo 2025 Honorees: Young Chiropractor of the Year – Korey Rose, DC President's Award – Jon Blackwell, DC, and Korey Rose, DC Executive Director's Award – Steve Van Osdale, DC Regional Doctor of the Year – Region 1: Kaci Roach, DC, Region 2: Joseph Adams, DC Texas Women's Leadership and Service Award – Mary Collings, DC, and Clare Morgan, DC Student Leadership Award – Parker University Tri 9 student Hayden Hadley During Chiro Texpo 2025, TCA also announced and swore in its new executive officers for the 2025-2026 term, all of whom are Parker University graduates! TCA's executive officers preside over an executive committee, including four department coordinators in charge of TCA's state committees, as well as a board of directors comprised of representatives from TCA's 12 districts, which cover all of Texas. TCA's New Executive Officers for 2025-2026: Don White, DC, FICC – president Andrew Oteo, DC – vice president Korey Rose, DC – secretary/treasurer Todd Whitehead, DC (served as TCA's president in 2024-2025) – immediate past president Congratulations to all the leaders who were recognized and honored for their dedication to furthering the positive impact of chiropractic care in the state of Texas. About Parker University Parker University, the fastest-growing college in Dallas and the fourth-fastest-growing in Texas, was founded in 1982 by Dr. James William Parker as Parker College of Chiropractic. Today, the university serves over 2,300 students and offers more than 25 accredited academic programs and certificates, including its flagship Doctor of Chiropractic program, which is home to the second-largest chiropractic cohort in the world. Parker University also leads in innovation with master's degrees in Clinical Neuroscience, Strength and Human Performance, and Functional Nutrition. Additionally, five new board-approved programs are set to launch soon. Attachment The Texas Chiropractic Association (TCA) Recognizes Parker University and Several Alumni at Chiro Texpo 2025. Disclaimer: The above press release comes to you under an arrangement with GlobeNewswire. Business Upturn takes no editorial responsibility for the same. Ahmedabad Plane Crash GlobeNewswire provides press release distribution services globally, with substantial operations in North America and Europe.
Yahoo
5 hours ago
- Yahoo
Aegis Capital Reaffirms Buy on Atai Life Sciences N.V. (ATAI) with $8 Target
Atai Life Sciences N.V. (NASDAQ:ATAI) is among the . Aegis Capital analysts have reaffirmed their Buy rating on Atai Life Sciences N.V. (NASDAQ:ATAI), with an unchanged price target of $8.00, following the favorable Phase 2b results of the BPL-003 treatment for the company's Treatment Resistant Depression (TRD). Around 193 depression patients, who didn't respond to previous treatment, were part of the Phase 2b trial testing three doses: 0.3 mg, 8 mg, and 12 mg. Not only the 8mg but also the 12mg dose showcased statistically significant improvement in an FDA-accepted measurement of depression, with a healthy side-effect profile. A close-up of a medical professional providing advice to a patient struggling with opioid use disorder. The research firm believes these robust results strengthen the company's efforts to proceed to a Phase 3 clinical program, which would result in marketing approval. Going forward, Atai Life Sciences N.V. (NASDAQ:ATAI) plans to schedule a post-Phase 2 meeting with the FDA, with the Phase 3 trial expected to begin only at the end of the upcoming year. Another noteworthy development is the company's recent $50 million private capital raise, which involved the sale of 18,264,840 common shares with 25% warrant coverage at the market price. Moves like these highlight Atai Life Sciences N.V. (NASDAQ:ATAI)'s capacity to raise cash when needed. Atai Life Sciences N.V. (NASDAQ:ATAI) is a German clinical-stage biopharmaceutical company that researches, develops, and markets mental health treatments in the United States, Germany, and Canada. With a market capitalization of $587.639 million, the company is committed to healing mental health disorders. While we acknowledge the potential of ATAI as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the . READ NEXT: The Best and Worst Dow Stocks for the Next 12 Months and 10 Unstoppable Stocks That Could Double Your Money. Disclosure: None.
