Latest news with #Dyne
Yahoo
5 days ago
- Health
- Yahoo
Dyne Therapeutics to Present New Preclinical Data in Facioscapulohumeral Muscular Dystrophy at the FSHD Society International Research Congress
- 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 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. 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@ MediaStacy Nartkersnartker@ in to access your portfolio
Yahoo
24-04-2025
- Business
- Yahoo
Dyne Therapeutics Receives European Medicines Agency (EMA) Orphan Drug Designation for DYNE-251 in Duchenne Muscular Dystrophy
- Recently presented data demonstrated sustained functional improvement with DYNE-251 treatment through 18 months - - Data from the fully enrolled DELIVER registrational expansion cohort is planned for late 2025 - WALTHAM, Mass., April 24, 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 the European Commission (EC) has granted orphan drug designation for DYNE-251 for the treatment of Duchenne muscular dystrophy (DMD). DYNE-251 is being evaluated in the Phase 1/2 DELIVER global clinical trial in individuals with DMD who are amenable to exon 51 skipping. Long-term clinical data from the ongoing DELIVER trial of DYNE-251 that demonstrated unprecedented and sustained functional improvement at the selected registrational dose were presented in March at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference. Functional assessments in the DELIVER trial include Stride Velocity 95th Centile (SV95C), an objective digital outcome that is accepted as a primary endpoint for DMD clinical trials in Europe. 'Our recent long-term DELIVER trial results demonstrated clinically relevant and sustained functional improvement through 18 months, including as assessed by SV95C, which may support a strong rationale for regulatory approval in Europe,' said Doug Kerr, MD, PhD, chief medical officer of Dyne. 'We are pleased that the EC has granted orphan drug designation to DYNE-251, reinforcing our belief that our next-generation exon 51 skipping investigational therapy for DMD may be able to bring clinically meaningful functional improvement to those living with this devastating disease. With full enrollment of the registrational expansion cohort in the DELIVER trial complete, we look forward to sharing data from this cohort in late 2025 and the potential to move forward with our first regulatory submissions in early 2026.' The EC grants orphan drug designation to drugs and biologics intended for the treatment, diagnosis or prevention of rare, life-threatening or chronically debilitating diseases or conditions that affect fewer than five in 10,000 people in the European Union (EU). Orphan designation provides companies with certain benefits, including reduced regulatory fees, clinical protocol assistance, research grants and the potential for up to 10 years of market exclusivity in the EU if approved. DYNE-251 was also granted U.S. Food and Drug Administration (FDA) orphan drug and rare pediatric disease designations in March 2023. Key Milestones for the DELIVER Trial Dyne continues to pursue expedited approval pathways globally for DYNE-251 in patients with DMD who are amenable to exon 51 skipping. Dyne has fully enrolled the registrational expansion cohort of 32 patients as part of the DELIVER trial. Data from this cohort are planned for late 2025. Dyne anticipates filing a Biologics License Application (BLA) submission for US accelerated approval in early 2026. About the DELIVER Trial DELIVER is a randomized, placebo-controlled, double-blind, Phase 1/2 clinical trial evaluating the safety, tolerability and efficacy of DYNE-251 in patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. The multiple ascending dose (MAD) portion of the study resulted in the selection of a registrational dose and regimen of 20 mg/kg every four weeks. A registrational expansion cohort to support potential regulatory submissions for expediated approvals, including accelerated approval in the U.S., is fully enrolled. The primary endpoint for this cohort is the change from baseline in dystrophin protein levels as measured by Western blot. For more information on the DELIVER trial, visit (NCT05524883) and (2023-510351-31-00). About DYNE-251 DYNE-251 is an investigational therapeutic being evaluated in the Phase 1/2 global DELIVER clinical trial for people living with DMD who are amenable to exon 51 skipping. DYNE-251 consists of a phosphorodiamidate morpholino oligomer (PMO) conjugated to a fragment antibody (Fab) that binds to the transferrin receptor 1 (TfR1), which is highly expressed on muscle. It is designed to enable targeted muscle tissue delivery and promote exon skipping in the nucleus, allowing muscle cells to create internally shortened, near full-length dystrophin protein, with the goal of stopping or reversing disease progression. DYNE-251 has been granted fast track, orphan drug and rare pediatric disease designations by the U.S. Food and Drug Administration for the treatment of DMD mutations amenable to exon 51 skipping. In addition to DYNE-251, Dyne is building a global DMD franchise and has preclinical programs targeting other exons, including 53, 45 and 44. About Duchenne Muscular Dystrophy (DMD) DMD is a rare disease caused by mutations in the gene that encodes for dystrophin, a protein critical for the normal function of muscle cells. These mutations, the majority of which are deletions, result in the lack of dystrophin protein and progressive loss of muscle function. DMD occurs primarily in males and affects an estimated 12,000 to 15,000 individuals in the U.S. and 25,000 in Europe. Loss of strength and function typically first appears in pre-school age boys and worsens as they age. As the disease progresses, the severity of damage to skeletal and cardiac muscle often results in patients experiencing total loss of ambulation by their early teenage years and includes worsening cardiac and respiratory symptoms and loss of upper body function by the later teens. There is no cure for DMD, and currently approved therapies provide limited benefit. 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 therapeutic potential of DYNE-251, the anticipated timeline for reporting additional data from the DELIVER clinical trial, the availability of expedited approval pathways for DYNE-251, expectations regarding the timing and outcome of interactions with regulatory authorities, and expectations regarding the timing of submitting applications for U.S. Accelerated Approval and other regulatory approvals, 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-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: InvestorsMia Tobiasir@ MediaStacy Nartkersnartker@ in to access your portfolio
Yahoo
15-04-2025
- Business
- Yahoo
Dyne Therapeutics Announces Chief Business Officer and Chief Scientific Officer Appointments
Vikram Ranade, PhD Ranjan Batra, PhD Oxana Beskrovnaya, PhD - Vikram Ranade, PhD, appointed as chief business officer - - Ranjan Batra, PhD, appointed as chief scientific officer - - Oxana Beskrovnaya, PhD, transitioning to chief innovation officer - WALTHAM, Mass., April 15, 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 the appointments of Vikram (Vik) Ranade, PhD, as chief business officer (CBO) and Ranjan (Ron) Batra, PhD, as chief scientific officer (CSO) of Dyne. Dr. Ranade will lead business development, corporate strategy and strategic partnerships to support the company's late-stage clinical and commercialization plans. Dr. Batra will lead the company's research strategy, pipeline development and activities supporting clinical development. Effective April 28, 2025, Oxana Beskrovnaya, PhD, will transition from CSO to chief innovation officer (CIO) responsible for driving Dyne's comprehensive innovation strategy to maximize the value of its FORCE™ platform and further enable the company's long-term success. 'I am excited to welcome Vik and Ron to our leadership team. Vik's experience in business development and corporate strategy will be invaluable as we prepare for the potential launches of two medicines in 2027 in myotonic dystrophy type 1 and Duchenne muscular dystrophy. Our incoming CSO, Ron, will play a key role in advancing our pipeline and elucidating the power of the FORCE platform to deliver functional improvement in the clinic,' said John Cox, president and chief executive officer of Dyne. 'With the addition of Vik, Ron and our CFO, Erick Lucera, as well as Oxana in her new role as CIO, we are well positioned to drive the business, deliver therapies that bring functional improvement to patients and create significant shareholder value through our clinical programs, pipeline and unique platform.' Dr. Ranade joins Dyne, effective immediately, from Tessera Therapeutics, where he was senior vice president of corporate development, leading strategy and business development, and was member of the executive team. During more than five years at Tessera he also played a key role in equity financings totaling more than $600 million. Previously, he worked in McKinsey & Company's healthcare practice, advising biopharmaceutical and biotechnology companies on strategy, M&A and R&D. He led diligence on completed deals with an aggregate transaction value of more than $15 billion and advised on clinical strategy for more than 20 programs. Dr. Ranade also co-led McKinsey's Center for Asset Optimization, which focuses on clinical-stage asset development strategy. Dr. Ranade holds a PhD in Genetics and Development from Columbia University and a BS in biochemistry from Brandeis University, where he earned highest honors for his research on DNA damage repair pathways. 'I am thrilled to welcome Ron as our new scientific leader. He brings outstanding scientific expertise and the required peer network and passion to continue to expand Dyne's pipeline of potentially transformative therapies,' said Dr. Beskrovnaya. 'In my new role, I look forward to working with our strong leadership team and leveraging our world-class expertise in TfR1 delivery to pioneer and accelerate new therapeutic applications.' Dr. Batra joins Dyne, effective April 28, 2025, from Lexeo Therapeutics, where he was vice president of research and translation. A leading expert in RNA biology and therapeutics, he has developed cutting-edge treatments for genetic diseases. Previously, Dr. Batra served as a senior vice president of R&D at LocanaBio, advancing RNA-targeted therapies for rare disorders including Duchenne muscular dystrophy and myotonic dystrophy. Prior to LocanaBio, he worked at Verily Life Sciences and conducted pioneering research at UC San Diego and the University of Florida with more than 35 publications and patents. Dr. Batra earned a PhD in genetics from the University of Florida, where he studied RNA biology and gene therapy for neuromuscular diseases. About Dyne TherapeuticsDyne 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 StatementsThis 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-101 and DYNE-251, and expectations regarding the timing of commercial launches 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-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: InvestorsMia Tobiasir@ Nartkersnartker@ Photos accompanying this announcement are available at: in to access your portfolio
Boston Globe
26-03-2025
- Health
- Boston Globe
Wave Life Sciences to submit Duchenne drug for approval after Phase 2 success
In Wave's Phase 2 study, 11 patients taking the company's treatment began producing an average of 7.8 percent of normal dystrophin, But perhaps more notably, Wave touted that the drug also appears to improve muscle health. The group of children taking the drug had a 28.6 percent reduction in muscle fibrosis compared to historical data. Advertisement Wave plans to submit the drug to the FDA for approval next year. The company has tested multiple Duchenne drugs since its founding in 2012, largely with Wave's drug is designed for a subset of Duchenne patients who have problems with exon 53, which is an Wave's drug is part of a new generation of so-called 'exon skippers' being developed for the fatal disease. The first such treatment, also made by Sarepta, was Biotechs such as Dyne, Wave, and Avidity are now developing more precise skippers that are either targeted to muscle or tweaked in other ways to improve efficacy.
Yahoo
20-03-2025
- Business
- Yahoo
Dyne Therapeutics Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4) to Newly Appointed Chief Financial Officer
WALTHAM, Mass., March 20, 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 it has granted inducement equity awards to its newly appointed Chief Financial Officer, Erick J. Lucera. The awards were approved by the Compensation Committee of Dyne and made as an inducement material to Mr. Lucera's acceptance of employment with Dyne under Dyne's 2024 Inducement Stock Incentive Plan in accordance with Nasdaq Listing Rule 5635(c)(4). The inducement equity awards were granted to Mr. Lucera effective as of March 31, 2025 and consists of a non-statutory stock option to purchase up to 214,500 shares of Dyne's common stock at a per share exercise price equal to the closing price of Dyne's common stock on The Nasdaq Global Select Market on March 31, 2025, and a restricted stock unit award with respect to 66,100 shares of Dyne's common stock. The stock option award has a ten-year term and is scheduled to vest over four years, with 25% of the original number of shares vesting on March 31, 2026 and the remainder vesting in equal quarterly installments over the three years thereafter, subject to Mr. Lucera's continued service to Dyne through the applicable vesting dates. The restricted stock unit award is scheduled to vest in equal annual installments over four years with the first installment vesting on March 31, 2026, subject to Mr. Lucera's continued service to Dyne through the applicable vesting dates. The inducement equity awards are subject to the terms and conditions of the award agreements covering the grants and Dyne's 2024 Inducement Stock Incentive Plan. 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. Contacts: InvestorsMia Tobiasir@ MediaStacy Nartkersnartker@ in to access your portfolio
