Latest news with #DUX4
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
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
06-05-2025
- Business
- Business Wire
SOLVE FSHD Announces $3 Million Investment in Armatus Bio to Advance Gene Therapy Program for FSHD
VANCOUVER, British Columbia & COLUMBUS, Ohio--(BUSINESS WIRE)--SOLVE FSHD, a venture philanthropy organization committed to accelerating therapies for facioscapulohumeral muscular dystrophy (FSHD), today announced a $3 million investment in Armatus Bio, a biotechnology company developing next-generation vectorized RNAi therapeutics for neuromuscular diseases. The investment will support ARM-201, Armatus Bio's AAV-delivered microRNA therapy designed to silence the expression of DUX4, the toxic protein responsible for muscle degeneration in individuals with FSHD. The investment is part of SOLVE FSHD's growing portfolio of therapeutic programs aimed at addressing FSHD through a range of innovative approaches. The investment is part of SOLVE FSHD's growing portfolio of therapeutic programs aimed at addressing FSHD through a range of innovative approaches. ARM-201 is a potential best-in-class, single-dose therapeutic for the treatment of FSHD. Its proprietary miRNA payload is designed to reduce DUX4 expression and thereby arrest muscle weakening and atrophy, prevent further degeneration, and reduce inflammation and oxidative stress associated with the disease. ARM-201 incorporates AAV-SLB101, a next-generation AAV capsid licensed from Solid Biosciences, which was rationally designed to target integrin receptors and has shown enhanced skeletal and cardiac muscle transduction, with decreased liver exposure in preclinical studies. It has also been shown to be well tolerated with robust transduction and expression levels seen in a clinical trial for Duchenne muscular dystrophy. With a pre-IND meeting successfully completed with the FDA, Armatus Bio will deploy SOLVE FSHD's investment towards critical IND-enabling activities, positioning ARM-201 for regulatory submission and entry into FSHD clinical trials. 'We're thrilled to support Armatus Bio in advancing a differentiated therapeutic approach that directly targets the genetic cause of FSHD,' said Eva Chin, PhD, Executive Director of SOLVE FSHD. 'This program represents an important step toward delivering meaningful treatments to the FSHD community. We believe Armatus has a strong scientific foundation, experienced leadership, and a focused development strategy that position them well for clinical and commercial success.' 'SOLVE FSHD's focused commitment to advancing therapeutic development for FSHD, combined with their collaborative approach and scientific expertise, has made them an invaluable partner for Armatus,' said Rachel Salzman, DVM, CEO of Armatus Bio. 'This investment provides critical resources to advance ARM-201 toward the clinic and positions us for continued growth as a company. We're excited to take this next step together and accelerate the path toward a much-needed therapy for the FSHD community.' FSHD is one of the most common forms of muscular dystrophy, affecting approximately 1 in 8,000 individuals globally. With no approved treatments available, advancing promising therapies is critical to meeting the urgent needs of the FSHD community. 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, widely known as the founder and part owner of various technical apparel companies including lululemon and Amer Sports. The Wilson family has committed $100 million to kick-start funding into projects that support the organization's mission to solve 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. About Armatus Bio Armatus Bio is a privately held late preclinical stage biotechnology company leveraging vectorized RNAi to address urgent unmet medical needs in genetically-driven neurological diseases. Based in Columbus, Ohio, the company is led by a seasoned team with expertise in drug development and delivery, and partnered with world renowned experts in vector biology, genomics, and neurology. In addition to ARM-201, Armatus is building a pipeline of precision medicines that can transform care for people with other genetic neuromuscular disorders.
Yahoo
26-03-2025
- Health
- Yahoo
Epicrispr banks $68M to test epigenetic editing on rare muscle disease
This story was originally published on BioPharma Dive. To receive daily news and insights, subscribe to our free daily BioPharma Dive newsletter. Epicrispr Biotechnologies has raised $68 million in pursuit of a first-of-its-kind genetic medicine for a rare neuromuscular disorder called facioscapulohumeral muscular dystrophy. EPI-321, the startup's lead program, uses CRISPR tools to stop errant expression of a gene implicated in the muscle-wasting condition. Epicrispr will start a Phase 1 trial this year in New Zealand, according to the company's Wednesday statement. The San Francisco Bay Area biotech's Series B round was led by Ally Bridge Group and involved Solve FSHD, an advocacy group formed by Lululemon Athletica founder Chip Wilson. FSHD is a rare neuromuscular disorder estimated to affect about 870,000 people worldwide. The disease is characterized by progressive muscle weakness that begins in the face, back and upper arms and can leave people in wheelchairs or with debilitating pain and fatigue. Though there are no available medications for FSHD, drugmakers in recent years have zeroed in on a gene called DUX4. In FSHD, a genetic error causes DUX4 to be overexpressed, eventually resulting in muscle degeneration and atrophy. Biotech companies have been working on various ways, from small molecule drugs to gene therapies, to stop that from happening. One high-profile effort, an oral drug developed by Fulcrum Therapeutics and Sanofi, failed in Phase 3 testing last year. But other companies, including Avidity Biosciences, Novartis, Arrowhead Pharmaceuticals and Dyne Therapeutics have drugs in development as well. There are currently more than a dozen active DUX4-targeting drug programs, according to the nonprofit FSHD Society. Epicrispr says its approach is unique among that group. The company is using CRISPR tools to turn genes on or off instead of altering DNA directly. In FSHD, it's harnessing CRISPR to bind a precise region of the DUX4 gene and make a chemical modification. The hope is doing so might stop expression of the encoded protein, without the health risks associated with cutting into DNA. According to Amber Salzman, the company's CEO, that strategy has shown potential in preclinical tests to impact muscle function and block the DUX4 protein from 'seeping out.' 'We're going after the absolute root cause' of the disease, she said. 'It's a really, really different approach.' Salzman has for years worked on genetic disorders as a biotech executive and patient advocate. While at GSK many years ago, her son and two nephews were diagnosed with a rare disease called adrenoleukodystrophy. She connected with prominent gene therapy researcher Jim Wilson, met several other experts in the field and started the nonprofit Stop ALD Foundation. One of Salzman's nephews died from ALD in 2004. But her son and second nephew received a treatment that was later approved as Skysona. In the meantime, Salzman worked at multiple biotech startups, including eye gene therapy developer Adverum Biotechnologies. In 2021, a recruiter gauged her interest in leading Epicrispr, which was then known as Epic Bio. By then, the startup had already started working on FSHD — a disease that affected her husband's family, Salzman said. That, and the potential to use epigenetic editing against a wide range of diseases, convinced her to take the job. 'All of a sudden, I found a company that had addressed all the limitations I'd come across in genetic medicine,' she said. Epicrispr raised a $55 million Series A round in 2022. Along with FSHD, it's working on drugs for heterozygous familial hypercholesterolemia, alpha-1 antitrypsin deficiency, a pair of eye diseases and certain undisclosed blood cancers. All of its work is preclinical. The company was co-founded by Stanford researcher Stanley Qi, who worked closely with gene editing pioneer Jennifer Doudna at UC Berkeley.
Yahoo
26-03-2025
- Business
- Yahoo
Epicrispr Biotechnologies Secures $68 Million Series B to Initiate Clinical Trial for First-in-Class Disease-Modifying Epigenetic Neuromuscular Therapy for FSHD
- Financing led by Ally Bridge Group, with participation from SOLVE FSHD, a venture philanthropy organization - Lead program, EPI-321, is the first clinical application of epigenetic modulation in neuromuscular diseases, with a first-in-human trial in New Zealand to commence in 2025 as part of a broader global clinical strategy SOUTH SAN FRANCISCO, Calif., March 26, 2025--(BUSINESS WIRE)--Epicrispr Biotechnologies, a biotechnology company focused on developing curative therapies, today announced it has secured $68 million in the first close of its Series B financing. The proceeds will support the clinical development of EPI-321, a first-in-class, disease-modifying therapy for facioscapulohumeral muscular dystrophy (FSHD), a genetic neuromuscular disease. The Series B financing was led by Ally Bridge Group, with participation from SOLVE FSHD, the venture philanthropy organization founded by Chip Wilson, founder of Lululemon Athletica and FSHD patient, along with other new and existing investors. The financing will support Epicrispr's upcoming clinical trial of EPI-321, as well as continued advancement of the company's broader pipeline. Epicrispr also announced clinical trial application (CTA) approval from New Zealand's Medsafe to initiate a first-in-human trial of EPI-321, the first epigenetic therapy to enter the clinic for a neuromuscular disease. The study is expected to begin in 2025, and will evaluate the safety, tolerability, pharmacodynamics, and biological activity of a single intravenous dose of EPI-321 in adults with FSHD. "FSHD is one of the most common adult muscular dystrophies, with estimates of up to 1 million patients affected worldwide. But patients have no disease-modifying therapy for this progressive disease," said Dr. Richard Roxburgh, Associate Professor of Medicine at the University of Auckland and principal investigator for the EPI-321 clinical trial, which is planned to be conducted in partnership with Pacific Clinical Research Network, a leading clinical research center in New Zealand. "We look forward to advancing this clinical trial which could, with a single treatment, permanently address the disease's underlying cause, and are hopeful that it will pave the way for new standards for therapies in genetic diseases." EPI-321 is an investigational one-time gene-modulating therapy designed to silence aberrant expression of DUX4, a gene that is incorrectly activated in FSHD and leads to progressive muscle degeneration. Delivered systemically via a clinically validated AAV vector, EPI-321 has demonstrated robust suppression of DUX4 expression and protection of muscle tissue in preclinical models. EPI-321 has received FDA Fast Track, Rare Pediatric Disease, and Orphan Drug designations. "We are developing a first-in-class, one-time epigenetic therapy that targets the genetic root cause of FSHD," said Amber Salzman, Ph.D., CEO, Epicrispr Biotechnologies. "The Series B financing and regulatory clearance to begin our first-in-human trial marks a pivotal milestone as we become a clinical-stage company. With a strong investor syndicate and recent FDA designations recognizing EPI-321's potential, our team is laser-focused on advancing EPI-321 into the clinic to provide a much-needed therapy to patients and families in desperate need." "With a robust body of data validating the potential of EPI-321 and the GEMS platform broadly, Epicrispr has shown itself to be a leading epigenetic editing company," said Andrew Lam, Pharm.D., Managing Director, Head of Biotech Private Equity, Ally Bridge Group. "We are proud to lead this investment in Epicrispr's future, and we look forward to partnering with their leadership to support their continued success." "As someone living with FSHD, I know the devastating impact of this disease and the urgent need for treatments that target its root cause," said Chip Wilson, founder and Chairman of SOLVE FSHD. "We commend Epicrispr's commitment in advancing EPI-321 for FSHD and are glad to be part of this financing to support its transition to the clinic." Concurrent with the funding, Epicrispr has expanded its Board of Directors with the addition of Andrew Lam, Pharm.D., of Ally Bridge Group, Eric Crombez, M.D., Chief Medical Officer of Ultragenyx, and Jennifer King, Ph.D., former SVP of Business Development at Intellia Therapeutics and an expert in rare diseases and strategic partnerships. About EPI-321 EPI-321 is an investigational epigenetic therapy that aims to address the underlying molecular mechanisms of FSHD with a one-time dose. It has been granted FDA Fast Track, Rare Pediatric Disease, and Orphan Drug designations. Following intravenous administration, EPI-321 is directed to muscle tissue within a single AAV vector, which has been clinically validated for muscle delivery. Preclinical studies on EPI-321 have demonstrated its ability to robustly suppress pathological expression of the DUX4 gene and reduce muscle cell death. A first-in-human clinical trial of EPI-321 is planned for 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 Ally Bridge Group Ally Bridge Group is a global healthcare investment firm focused on private and public high-impact life science innovation. Founded in 2013 by Frank Yu, the firm has led or co-led over $6 billion in healthcare transactions. The firm's mission is to generate superior risk-adjusted returns for investors guided by the core principle of selective investment in healthcare innovation that addresses unmet medical needs. Ally Bridge Group has offices in New York and Hong Kong. For more information, visit or follow us on LinkedIn. View source version on Contacts Investor ContactBenson Media ContactKimberly HaKKH Sign in to access your portfolio
