Latest news with #CRISPR-GNDM®
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
2 days 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
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
