Latest news with #DUX4
Yahoo
09-06-2025
- Business
- Yahoo
Avidity Biosciences Touts Encouraging Functional Gains And Biomarker Reductions In Rare Muscular Weakness Disease Trial
Avidity Biosciences, Inc. (NASDAQ:RNA) revealed topline data from the dose escalation cohorts of the delpacibart braxlosiran (del-brax) Phase 1/2 FORTITUDE program in Facioscapulohumeral Muscular Dystrophy (FSHD). FSHD is a genetically acquired disease that leads to progressive muscle weakness and severely decreased functional capacity. The data will be presented at the 32nd Annual FSHD Society International Research is the first investigational therapy designed to treat the underlying cause of FSHD by directly targeting the disease-causing gene, double homeobox 4 (DUX4). FSHD affects approximately 45,000 to 87,000 people in the United States and Europe. Topline data from these cohorts for del-brax treated participants, compared to placebo, demonstrated: Consistent improvement of functional mobility and muscle strength as measured by 10-meter Walk-Run test (10MWRT), Timed Up and Go (TUG), and quantitative muscle testing (QMT) as compared to placebo; Consistent improvement in multiple measures of quality of life as measured by patient-reported outcomes and compared to placebo; Rapid and significant reductions in levels of KHDC1L and creatine kinase, a biomarker of muscle damage. Favorable long-term safety and tolerability with most adverse events (AEs) mild or moderate, with no related serious or severe adverse events and no discontinuations. Topline data from the ongoing, fully enrolled del-brax Phase 1/2 FORTITUDE biomarker cohort are anticipated in Q2 2026. The primary endpoint of the FORTITUDE biomarker cohort is reduction of KHDC1L, a novel DUX4-regulated circulating biomarker. Avidity also announced that the accelerated approval regulatory pathway in the U.S. is open for del-brax and that the company has initiated the global, confirmatory Phase 3 FORWARD study in FSHD. In a recent research note, Chardan Capital analyst Keay Nakae set a 12-month price target of $75 per share for Avidity Biosciences. The valuation stems from a Net Present Value (NPV) model of the company's projected revenues from its RNA therapeutics pipeline, forecasted out to the year 2034. Chardan maintained its Buy rating and increased its price forecast from $65 to $75. According to Nakae's analysis, the valuation incorporates specific 'probability of success' rates for each of Avidity's drug candidates to account for clinical and regulatory risks. His model assigns a 60% chance of success for the company's FSHD candidate, 50% for its DM1 and DMD exon 44 candidates, and lower probabilities for other assets. The analyst applied a 14% Weighted Average Cost of Capital (WACC) as the discount rate to reflect the high-risk profile of the development-stage company. Nakae also highlighted several significant risks that could impede Avidity from reaching the $75 price target. He noted that the company, which currently has no revenue-generating products, will likely require additional financing, potentially leading to shareholder dilution. The analyst also pointed to the risks of intellectual property challenges, the critical nature of upcoming clinical trial outcomes, and the intense competition from larger, better-resourced companies in the biotechnology sector. Price Action: RNA stock is trading lower by 11.6% to $32.03 at last check Monday. Read Next:Photo by Gorodenkoff via Shutterstock UNLOCKED: 5 NEW TRADES EVERY WEEK. Click now to get top trade ideas daily, plus unlimited access to cutting-edge tools and strategies to gain an edge in the markets. Get the latest stock analysis from Benzinga? AVIDITY BIOSCIENCES (RNA): Free Stock Analysis Report This article Avidity Biosciences Touts Encouraging Functional Gains And Biomarker Reductions In Rare Muscular Weakness Disease Trial originally appeared on © 2025 Benzinga does not provide investment advice. All rights reserved.
Business Wire
08-06-2025
- Business
- Business Wire
SOLVE FSHD and Modalis Announce Strategic Collaboration to Develop an Innovative CRISPR-Based Epigenome Editing Treatment for Facioscapulohumeral Muscular Dystrophy
VANCOUVER, British Columbia & TOKYO & WALTHAM, Mass.--(BUSINESS WIRE)-- SOLVE FSHD, a venture philanthropy organization dedicated to accelerating treatments for facioscapulohumeral muscular dystrophy (FSHD), and Modalis Therapeutics Corporation (TSE 4883; 'Modalis'), a CRISPR-based epigenome editing therapeutics company focused on rare genetic diseases, today announced a strategic collaboration to develop an innovative therapy for FSHD, a debilitating muscular disorder affecting approximately 1 million individuals worldwide. The novel therapy leverages Modalis's proprietary CRISPR-GNDM ® (Guide Nucleotide-Directed Modulation) technology, which can dynamically modulate gene expression without introducing double-strand DNA breaks. SOLVE FSHD will provide strategic funding to support the development of Modalis's MDL-103 program. MDL-103 is an innovative therapeutic solution that continuously suppresses the expression of the DUX4 gene, the toxic disease-causing gene for FSHD, which becomes abnormally activated due to epigenetic changes in the D4Z4 repeat region on chromosome 4. MDL-103 is designed to have durable activity over long periods of time under the control of a strong, muscle-specific promoter, and is delivered to the muscles of patients using a muscle-tropic AAV delivery system. Modalis's CRISPR-GNDM ® technology has the potential to transform the treatment of FSHD by epigenetically silencing the expression of DUX4. 'SOLVE FSHD is pleased to partner with Modalis and to add them to our diverse portfolio of collaborators that are advancing potential therapies for FSHD,' stated Eva Chin, Executive Director of SOLVE FSHD. 'SOLVE FSHD identified Modalis as a company committed to finding a cure for this debilitating condition. We were impressed by their unique approach to targeting the epigenetic cause of FSHD, using a platform technology that has shown promise in other neuromuscular diseases. We believe that the support from SOLVE FSHD will allow Modalis to accelerate the advancement of MDL-103 into clinical trials.' 'We are delighted to be working in partnership with SOLVE FSHD and greatly appreciate the invaluable support for the development of MDL-103,' said Haru Morita, CEO of Modalis. 'This strategic collaboration is a strong validation of Modalis's CRISPR-GNDM ® technology and our MDL-103 program. As a pioneer in this technology, we have demonstrated promising long-term drug efficacy in mouse models, shown durable target engagement and safety in non-human primates, and exhibited excellent biodistribution in neuromuscular disorders. We believe that MDL-103, which incorporates CRISPR-GNDM ® technology with a muscle tropic AAV delivery system, has significant potential as a breakthrough treatment for FSHD.' 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 organizations' 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, visit Modalis was founded in 2016 and conducts research and development activities in Massachusetts, USA. Modalis is a pioneering leader in the field of epigenetic medicine. Modalis develops therapeutics for patients suffering from serious genetic disorders such as neuromuscular diseases, CNS diseases, and cardiomyopathies. Modalis's proprietary CRISPR-GNDM ® technology is capable of specifically up or down modulating the expression of disease-relevant genes without introducing double-strand DNA breaks. For more information, visit
Yahoo
05-06-2025
- 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.
