Latest news with #FSHD
Yahoo
3 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
12-05-2025
- Business
- Yahoo
Modalis has been selected as a finalist in the XPRIZE Healthspan FSHD Bonus Prize Competition and awarded research funds.
TOKYO & WALTHAM, Mass., May 12, 2025--(BUSINESS WIRE)--Modalis Therapeutics Corporation (Tokyo Stock Exchange: 4883), a pioneering company developing innovative products for the treatment of rare genetic diseases utilizing its proprietary CRISPR-GNDM® epigenome editing technology, announced its recognition as a top eight finalist in the prestigious XPRIZE Healthspan competition's FSHD Bonus Prize and award of $250,000 for demonstrating a feasible solution to treat Facioscapulohumeral muscular dystrophy (FSHD) . XPRIZE, the world's leader in designing and operating large-scale incentive competitions to solve humanity's grand challenges, launched the $101 million, 7-year global XPRIZE Healthspan competition in 2023. The competition's FSHD Bonus Prize is designed to ignite solutions for FSHD, a genetic disease that impacts muscular function that has limited treatment options due to its complexity. In the FSHD Bonus Prize Competition, the $8 million grand prize will be awarded to the team that successfully completes a clinical trial demonstrating significant advancements in FSHD treatment, making a major step toward finding a cure. FSHD is the third most common form of muscular dystrophy, affecting around 1 million people worldwide. FSHD is a genetic disorder in which the muscles of the face, shoulder blades, and upper arms are typically among the most affected. The onset is typically in the teenage and early adult years, but it can present in infancy, which tends to be a more aggressive course. The disease is slowly progressive and approximately 20% of patients are wheelchair bound by age 50. There are currently no FSHD-specific or disease-modifying treatments available. Modalis' proprietary CRISPR-GNDM®, is capable of specific modulation of the expression of disease-relevant genes, without introducing double-strand DNA breaks. Our MDL-103 is potentially the first-in-class therapeutics to solve the challenge and provide life-changing therapeutics for the patients of FSHD by suppressing expression of Dux4, the gene responsible for deleterious FSHD symptoms, across muscle tissues. "We are honored to be named as one of the top 8 finalists in the XPRIZE Healthspan FSHD Bonus Prize competition" said Haru Morita, CEO of Modalis. "This is not only an honor, but also a validation of our vision of developing treatments for patients suffering from diseases for which there is no cure and of our innovative epigenome editing technology (CRISPR-GNDM®). We are also excited about the opportunity to leverage the global innovation network provided by the XPRIZE and to explore potential collaborations with leading research institutions and companies from around the world." About XPRIZE XPRIZE is the recognized global leader in designing and executing large-scale competitions to solve humanity's greatest challenges. For over 30 years, our unique model has democratized crowd-sourced innovation and scientifically scalable solutions that accelerate a more equitable and abundant future. Donate, learn more, and co-architect a world of abundance with us at About SOLVE FSHD SOLVE FSHD is a venture philanthropic organization established to catalyze innovation and accelerate key research in finding a cure for FSHD, established by renowned Canadian entrepreneur and philanthropist, Chip Wilson. The Wilson family has committed $100 million to kick-start funding into projects that support the organization's mission to find a cure for FSHD by 2027. The goal of SOLVE FSHD is to find a solution that can slow down or stop muscle degeneration, increase muscle regeneration and strength, and improve the quality of life for those living with FSHD. If you want to find out more about our efforts at SOLVE FSHD, please see our website - About Modalis: Modalis Therapeutics develops precision genetic medicines using epigenome editing technology. Modalis is pursuing therapies for orphan genetic diseases using its proprietary CRISPR-GNDM® technology which enables the gene/locus-specific modulation of gene expression or epigenetic editing without the need for DNA cleavage or altering DNA sequence. Headquartered in Tokyo with laboratories and facilities in Waltham Massachusetts, the company is listed on Tokyo Stock Exchange's Growth market. For additional information, please visit View source version on Contacts Modalis Therapeutics CorporationCorporate Planning Departmentmedia@ Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Business Wire
12-05-2025
- Business
- Business Wire
Modalis has been selected as a finalist in the XPRIZE Healthspan FSHD Bonus Prize Competition and awarded research funds.
TOKYO & WALTHAM, Mass.--(BUSINESS WIRE)-- Modalis Therapeutics Corporation (Tokyo Stock Exchange: 4883), a pioneering company developing innovative products for the treatment of rare genetic diseases utilizing its proprietary CRISPR-GNDM ® epigenome editing technology, announced its recognition as a top eight finalist in the prestigious XPRIZE Healthspan competition's FSHD Bonus Prize and award of $250,000 for demonstrating a feasible solution to treat Facioscapulohumeral muscular dystrophy (FSHD) . XPRIZE, the world's leader in designing and operating large-scale incentive competitions to solve humanity's grand challenges, launched the $101 million, 7-year global XPRIZE Healthspan competition in 2023. The competition's FSHD Bonus Prize is designed to ignite solutions for FSHD, a genetic disease that impacts muscular function that has limited treatment options due to its complexity. In the FSHD Bonus Prize Competition, the $8 million grand prize will be awarded to the team that successfully completes a clinical trial demonstrating significant advancements in FSHD treatment, making a major step toward finding a cure. FSHD is the third most common form of muscular dystrophy, affecting around 1 million people worldwide. FSHD is a genetic disorder in which the muscles of the face, shoulder blades, and upper arms are typically among the most affected. The onset is typically in the teenage and early adult years, but it can present in infancy, which tends to be a more aggressive course. The disease is slowly progressive and approximately 20% of patients are wheelchair bound by age 50. There are currently no FSHD-specific or disease-modifying treatments available. Modalis' proprietary CRISPR-GNDM ®, is capable of specific modulation of the expression of disease-relevant genes, without introducing double-strand DNA breaks. Our MDL-103 is potentially the first-in-class therapeutics to solve the challenge and provide life-changing therapeutics for the patients of FSHD by suppressing expression of Dux4, the gene responsible for deleterious FSHD symptoms, across muscle tissues. 'We are honored to be named as one of the top 8 finalists in the XPRIZE Healthspan FSHD Bonus Prize competition' said Haru Morita, CEO of Modalis. 'This is not only an honor, but also a validation of our vision of developing treatments for patients suffering from diseases for which there is no cure and of our innovative epigenome editing technology (CRISPR-GNDM ®). We are also excited about the opportunity to leverage the global innovation network provided by the XPRIZE and to explore potential collaborations with leading research institutions and companies from around the world.' About XPRIZE XPRIZE is the recognized global leader in designing and executing large-scale competitions to solve humanity's greatest challenges. For over 30 years, our unique model has democratized crowd-sourced innovation and scientifically scalable solutions that accelerate a more equitable and abundant future. Donate, learn more, and co-architect a world of abundance with us at About SOLVE FSHD SOLVE FSHD is a venture philanthropic organization established to catalyze innovation and accelerate key research in finding a cure for FSHD, established by renowned Canadian entrepreneur and philanthropist, Chip Wilson. The Wilson family has committed $100 million to kick-start funding into projects that support the organization's mission to find a cure for FSHD by 2027. The goal of SOLVE FSHD is to find a solution that can slow down or stop muscle degeneration, increase muscle regeneration and strength, and improve the quality of life for those living with FSHD. If you want to find out more about our efforts at SOLVE FSHD, please see our website - About Modalis: Modalis Therapeutics develops precision genetic medicines using epigenome editing technology. Modalis is pursuing therapies for orphan genetic diseases using its proprietary CRISPR-GNDM ® technology which enables the gene/locus-specific modulation of gene expression or epigenetic editing without the need for DNA cleavage or altering DNA sequence. Headquartered in Tokyo with laboratories and facilities in Waltham Massachusetts, the company is listed on Tokyo Stock Exchange's Growth market. For additional information, please visit
Business Wire
12-05-2025
- Business
- Business Wire
Armatus Bio Selected as Finalist for XPRIZE Healthspan FSHD Bonus Prize
COLUMBUS, Ohio--(BUSINESS WIRE)--Armatus Bio, a late-preclinical stage biotech innovator developing vectorized RNAi medicines in neuromuscular disorders, today announced its recognition as one of eight finalists in the XPRIZE Healthspan FSHD Bonus Prize, led by XPRIZE, the world's leader in designing and operating large-scale incentive competitions to solve humanity's grand challenges. 'We applaud XPRIZE and SOLVE FSHD for their visionary leadership to champion innovative new ideas that carry the potential to dramatically impact care." - Rachel Salzman, DVM, Armatus CEO Share SOLVE FSHD, a venture philanthropy organization dedicated to catalyzing innovation and overcoming barriers to accelerate new therapies for facioscapulohumeral muscular dystrophy (FSHD), is a co-sponsor of XPRIZE Healthspan and funder of the FSHD Bonus Prize, which is being run in parallel with the XPRIZE Healthspan. 'The XPRIZE Healthspan FSHD Bonus Prize is designed to solicit bold solutions intended to deliver measurable improvements in muscle function and biomarkers of FSHD disease progression in individuals affected by FSHD,' said Eva Chin, PhD, Executive Director of SOLVE FSHD. 'Armatus' drug candidate, ARM-201, represents a highly promising strategy that addresses the underlying genetic defect that causes FSHD. Furthermore, Armatus has devised a clear roadmap for bringing this experimental therapeutic into human clinical trials to demonstrate its ability to transform outcomes for this population.' ARM-201 is a vectorized microRNA engineered with a second-generation myotropic capsid that has been designed to effectively, safely, and durably silence toxic DUX4 expression. Preclinical evaluations have generated multiple datasets that strongly support continued pursuit of the vectorized RNAi strategy, including improvements in FSHD-linked biomarkers and motor function. 'Recognition as a finalist in this highly competitive forum is a reflection of the promise, hard work, and rigor embedded within our scientific strategy for ARM-201, which originated in the Harper Lab at Nationwide Children's Hospital,' said Rachel Salzman, DVM, Chief Executive Officer of Armatus Bio. 'We applaud XPRIZE and SOLVE FSHD for their visionary leadership to champion innovative new ideas that carry the potential to dramatically impact care.' The $101 million XPRIZE Healthspan is a 7-year global competition to catalyze the development of proactive, accessible therapeutic solutions that restore muscle, cognition, and immune function by a minimum of 10 years, with an ambitious goal of 20 years, in persons aged 50-80 years, in one year or less. Read more at About Armatus Armatus Bio is a late-preclinical stage, privately held biotech innovator developing advanced medicines that leverage vectorized RNAi. RNAi is a well-validated therapeutic approach that modifies protein expression via innate cellular biogenesis pathways without altering the cell's genetic make-up. The company is advancing two assets addressing urgent unmet needs in neuromuscular disorders: TVR110 for Charcot-Marie-Tooth disease type 1A (CMT1A), and ARM-201 for Facioscapulohumeral Muscular Dystrophy (FSHD). In preclinical studies, these investigational drugs demonstrated robust early signals of precision target engagement and biomarker improvement, and both are now advancing toward preparations for clinical trials. For more information, visit
Business Wire
12-05-2025
- Business
- Business Wire
Epicrispr Biotechnologies Named Finalist in XPRIZE Healthspan Competition FSHD Bonus Prize
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Epicrispr Biotechnologies, a biotechnology company focused on developing curative therapies, today announced it has been named a Finalist Team in XPRIZE Healthspan FSHD Bonus Prize, earning a $250,000 Milestone Award for its work addressing facioscapulohumeral muscular dystrophy (FSHD), one of the most prevalent forms of muscular dystrophy globally. Epicrispr was one of eight global teams selected from hundreds of applicants to receive the $10M Milestone 1 FSHD Bonus Prize, part of the broader $101M XPRIZE Healthspan initiative supported by Hevolution Foundation and SOLVE FSHD. The award recognizes groundbreaking innovation aimed at extending healthy human lifespan and improving outcomes for patients affected by degenerative diseases of aging. 'Being recognized by XPRIZE as a finalist and Milestone 1 recipient of the FSHD Bonus Award is a major validation of our platform and mission,' said Amber Salzman, Ph.D., CEO, Epicrispr Biotechnologies. 'Our team is deeply motivated by the urgent need for novel therapies that can meaningfully help patients with FSHD and other epigenetically driven diseases.' 'We designed the XPRIZE Healthspan FSHD Bonus Prize to spark innovative solutions for FSHD that could have ripple effects across aging, muscle regeneration, and healthspan,' said Jamie Justice, Ph.D., Executive Director of XPRIZE Healthspan. 'Each of the eight finalist teams receiving this milestone award are demonstrating compelling approaches to address FSHD and push the boundaries of what aging with FSHD can look like.' Epicrispr's approach leverages proprietary technology to modulate gene expression without cutting DNA, offering a new class of therapeutics with the potential to precisely control disease-causing genes. Its lead program targets DUX4, the gene at the root of FSHD, with a goal of restoring muscle function and halting disease progression through safe, tunable interventions. Finalists were selected by a panel of independent judges based on the scientific rigor, feasibility, and transformative potential of their work. Epicrispr will be honored at the XPRIZE Healthspan Awards Ceremony and Investor Summit in New York City, taking place May 12–14, 2025. About Epicrispr Biotechnologies Epicrispr Biotechnologies is a biotechnology company pioneering gene-modulating therapies, leading with treatments for neuromuscular diseases. The company's proprietary Gene Expression Modulation System (GEMS) enables precise, durable control of gene expression, unlocking first-in-class treatments for previously untreatable conditions. Epicrispr's lead program, EPI-321 is in clinical trials for FSHD, and the company is advancing additional gene-modulating therapies. Epicrispr also has a research collaboration with Kite Pharma to develop next-generation CAR T-cell therapies. Learn more at or follow us on LinkedIn. About XPRIZE XPRIZE is the recognized global leader in designing and executing large-scale competitions to solve humanity's greatest challenges. For over 30 years, our unique model has democratized crowd-sourced innovation and scientifically scalable solutions that accelerate a more equitable and abundant future. Donate, learn more, and co-architect a world of abundance with us at
