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Yahoo
a day ago
- Business
- Yahoo
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., June 08, 2025--(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 About Modalis Therapeutics Corporation 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 View source version on Contacts SOLVE FSHDAlexandra Grant, House of Wilsonalexandrag@ 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
a day ago
- 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
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
25-03-2025
- Business
- Yahoo
SOLVE FSHD Announces Strategic Collaboration with Transcripta Bio to Advance Drug Discovery for FSHD
VANCOUVER, British Columbia, March 25, 2025--(BUSINESS WIRE)--SOLVE FSHD, a venture philanthropy organization dedicated to accelerating treatments for facioscapulohumeral muscular dystrophy (FSHD), is pleased to announce a strategic collaboration with Transcripta Bio, a cutting-edge AI-driven drug discovery company. This collaboration will focus on leveraging Transcripta Bio's advanced drug screening platform to identify and develop promising therapeutic candidates for FSHD, a debilitating muscular disorder affecting approximately 38,000 individuals in the U.S. alone. Under this agreement, SOLVE FSHD will provide strategic funding to support a drug screening initiative led by Transcripta Bio. The project will analyze a broad range of approved and late-stage clinical compound libraries in FSHD cell lines for their potential efficacy in targeting fibro-adipogenic progenitor (FAP) cells, known to contribute to the disease pathology. This initiative represents a significant step forward in drug repurposing for the disease with a novel target space, offering a faster path to clinical translation and one of the first dedicated efforts to systematically explore repurposed drugs for FSHD. "This collaboration aligns perfectly with SOLVE FSHD's mission to de-risk and accelerate therapeutic development for FSHD," said Eva Chin, Executive Director of SOLVE FSHD. "By partnering with Transcripta Bio, we are not only leveraging state-of-the-art AI-driven drug discovery but also working with a team who has the experience and expertise to ensure that the best potential candidates are rapidly validated and prepared for clinical trials, while going after a novel approach to attenuate muscle fibrosis." Through this partnership, both organizations will work together to evaluate promising drug candidates using advanced transcriptomics and data-driven insights. The collaboration aims to accelerate the identification of potential treatments and advance them toward clinical testing as efficiently as possible. Transcripta Bio will lead the advancement and commercialization of promising candidates into the clinic. "We are excited to collaborate with SOLVE FSHD on this important initiative," said Transcripta Bio CEO Chris Moxham. "Our platform has been successful in identifying novel drug candidates across multiple therapeutic areas, and we are eager to apply our technology to accelerate FSHD drug development." This partnership underscores SOLVE FSHD's commitment to funding high-impact, innovative approaches to address the urgent unmet need for FSHD therapies by 2027. By harnessing Transcripta Bio's AI-driven approach, the partnership has the potential to identify fast-tracked, repurposed drug candidates that could reach patients sooner than traditional drug development pathways. About SOLVE FSHDSOLVE 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, which holds brands such as Arc'teryx, Salomon and Wilson 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 Transcripta BioTranscripta Bio is an AI drug discovery company specializing in transcriptomics-driven insights to identify and develop novel therapeutics across multiple disease areas. Using its proprietary AI platform, Transcripta Bio accelerates the discovery and validation of promising drug candidates, leveraging both repurposed and novel molecules for targeted therapeutic interventions. View source version on Contacts Media Contact: Andrea MestrovicVery Polite Agencyandrea@ Sign in to access your portfolio
