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)--Jun 8, 2025--
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.
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20250608851515/en/
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 https://www.solvefshd.com.
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 https://www.modalistx.com/en/.
View source version on businesswire.com:https://www.businesswire.com/news/home/20250608851515/en/
CONTACT: SOLVE FSHD
Alexandra Grant, House of Wilson
[email protected]
Modalis Therapeutics Corporation
Corporate Planning Department
[email protected]
KEYWORD: MASSACHUSETTS NORTH AMERICA UNITED STATES ASIA PACIFIC JAPAN CANADA
INDUSTRY KEYWORD: RESEARCH GENETICS HEALTH TECHNOLOGY PHILANTHROPY BIOTECHNOLOGY GENERAL HEALTH PHARMACEUTICAL HEALTH OTHER PHILANTHROPY SCIENCE
SOURCE: SOLVE FSHD
Copyright Business Wire 2025.
PUB: 06/08/2025 07:30 PM/DISC: 06/08/2025 07:31 PM
http://www.businesswire.com/news/home/20250608851515/en
Hashtags
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Yahoo
an hour ago
- Yahoo
GPTBots Drives Enterprise AI Innovation at WaytoAGI Tokyo Hackathon, Showcasing Real-World Agent Solutions
HONG KONG, June 11, 2025 (GLOBE NEWSWIRE) -- a leading enterprise-grade AI agent building platform, recently sponsored the "WaytoAGI Global AI Conference – Tokyo 2025" hackathon, which took place on June 7-8 at J.F. Oberlin University in Tokyo. The event attracted over 300 participants from Japan, China, and around the world. During the hackathon, developers utilized GPTBots' enterprise AI agent framework to create impactful solutions addressing global business challenges, showcasing how AI agents can transform enterprise operations. Tokyo Hackathon Spotlight: AI Agents Redefining Enterprise SolutionsThe GPTBots hackathon featured four competition tracks—Enterprise Process Automation, AI-Driven Customer Interaction, Data Analysis & Decision Insights, and Open Innovation—challenging teams to build functional AI agents on the GPTBots platform. Three standout projects demonstrated the versatility of GPTBots' platform across industries: Campai: A Web3 marketing campaign management platform leveraging GPTBots for real-time sentiment analysis of social media trends. The solution assigns sentiment scores to Web3 projects by analyzing mention frequency and emotional tone, enabling data-driven marketing strategies. AI-Driven Nail Art Design Agent: Addressing the $12 billion global nail industry's design inefficiency, this agent generates personalized nail art concepts based on user preferences, reducing design time from hours to minutes and enabling salons to offer custom creations at scale. Movie Agent: A modular solution breaking down video production into AI agents—from creative development and script generation to storyboard design—streamlining workflows and cutting production costs by 40% for independent creators. Other notable projects included: DC Audit Agent: A Hong Kong-focused legal compliance tool that reviews employment contracts against local labor laws, generating compliance scores and optimization reports. Demand Lens: An analytics agent scraping Fiverr order data to reveal service demand trends, pricing insights, and market opportunities. Enterprise-Grade Capabilities Showcase at HackathonAt the event, Alen Hu, Senior Innovation Manager at led a practical workshop titled "Mastering GPTBots: Building AI Agents for Enterprise," providing participants with step-by-step guidance on: Core capabilities of GPTBots' agent framework, including LLM integration, workflow orchestration, and RAG-based knowledge retrieval. Hands-on development of AI agents for real-world scenarios, from customer service to data analysis. Best practices for deploying scalable, secure enterprise AI solutions, highlighted by case studies of GPTBots' global deployments. Global Collaboration and Technical Depth"The Tokyo hackathon exemplified how AI agents bridge technology and tangible business value," said Jerry Yin, VP of "Enterprises now seek scalable AI solutions that integrate seamlessly with existing operations. GPTBots' platform empowers this through secure, low-code development and enterprise-grade security." The event showcased cross-cultural innovation, with Japanese teams focusing on retail and hospitality applications, while participants from China led in Web3 and DeFi solutions. Developers demonstrated real-world deployments and shared best practices, highlighting the versatility and impact of enterprise AI agents across different industries. Advancing Scalable AI Adoption for EnterprisesAs part of the WaytoAGI conference, the hackathon highlighted tangible trends in enterprise AI adoption, with projects directly addressing real-world business needs showcased on the GPTBots platform. The diversity of solutions—from automated compliance and personalized design to advanced analytics and marketing—demonstrated how AI agents are being tailored for industry-specific challenges. By enabling rapid prototyping, secure deployment, and seamless integration with existing workflows, GPTBots empowers organizations to accelerate digital transformation and unlock new value streams. The event reinforced the growing demand for enterprise-ready AI solutions that are both scalable and adaptable, as businesses worldwide look to harness AI agents to drive efficiency, innovation, and competitiveness in a rapidly evolving market. About is an enterprise AI agent platform that empowers businesses to streamline operations, enhance customer experiences, and drive growth. Offering end-to-end AI solutions across customer service, knowledge search, data analysis, and lead generation, GPTBots enables enterprises to harness the full potential of AI with ease. With seamless integration into various systems, and support for scalable, secure deployments, GPTBots is dedicated to reducing costs, accelerating growth, and helping businesses thrive in the AI era. For more information, visit Media Contact: Tanya Marketing Directormarketing@ 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
Yahoo
2 hours ago
- Yahoo
Japan remains cautious on tariff impact in June econ report
TOKYO (Reuters) -Japan's government reiterated its warning on the risks from U.S. tariffs on economic growth, saying in a monthly report on Wednesday that the potential impact on corporate profits needed attention. The economy faces pressure from U.S. import levies of 24% from July unless officials can negotiate them down. Japan's government is also seeking an exemption for its carmakers from a separate 25% import tariff on vehicles. Japan's gross domestic product shrank an annualised 0.2% in the January-March quarter, even before U.S. President Donald Trump announced his so-called "reciprocal" tariffs on April 2. "The economy is recovering moderately, while uncertainty is arising from U.S. trade policies," the Cabinet Office said in its monthly report for June released on Wednesday, keeping the assessment unchanged for a third month. In addition to tariffs, the report maintained its reference to "the effect of continued price rises on household sentiment and consumption" as another negative risk to the economy. It also noted that corporate profits were recovering, citing first-quarter business statistics, but added a fresh reference to the potential impact of trade disruptions. "With some firms withholding financial forecasts amid uncertainties surrounding U.S. tariffs, their impact on earnings may come to light only in the data for the April-June quarter or beyond," an official at the Cabinet Office told a press briefing. While no significant impact from U.S. tariffs on Japan's export volumes has been seen so far, trade trends from May onwards required attention, the official added. On wages, this spring's pay negotiations probably led to an average raise of more than 5%, exceeding last year's, although smaller firms lagged bigger companies, the official said, based on the national labour group Rengo's data. Prime Minister Shigeru Ishiba said earlier this week that his ruling party would pledge to achieve growth of more than 50% in Japan's average income by 2040 in campaigning for the upper house elections next month.
Yahoo
3 hours ago
- Yahoo
Japan's Longer Debt Attractive for Foreigners, Morgan Stanley Says
(Bloomberg) -- Foreign investors are looking with interest at longer-term Japanese bonds, while it remains uncertain when the government will adjust how much of the debt it sells, Morgan Stanley says. Trump's Military Parade Has Washington Bracing for Tanks and Weaponry NY Long Island Rail Service Resumes After Grand Central Fire NYC Mayoral Candidates All Agree on Building More Housing. But Where? Senator Calls for Closing Troubled ICE Detention Facility in New Mexico California Pitches Emergency Loans for LA, Local Transit Systems 'You still have a tremendous amount of supply hitting the long-end of the Japanese government bond market, which is presenting value for investors outside of Japan' from Canada to Europe to Asia, the firm's global head of macro strategy Matthew Hornbach said on Bloomberg Television. 'The Ministry of Finance, which has a slower cadence in how it adjusts its issuance program, will eventually get the message from the investor community that it needs to pare back its issuance, but exactly the time frame for that is unclear,' Hornbach said. Yields for Japan's 30-year and 40-year government bonds have recently scaled new heights as the central bank pulls back from its massive bond-buying program and major Japanese financial institutions fail to fill in the gap. But global funds bought a record amount of Japan's super-long government bonds in April for a third straight month. There is speculation that Japan will adjust its issuance or buy back some of the debt to calm investor nerves. Hornbach said he expects long-term US Treasury yields and the dollar to move lower over time as the negative impacts of tariffs start coming through more clearly. He added that even if Japan's bonds can't hold down yields globally, central bank policies around the world should eventually bring down yields. 'Japanese government bond yields, for the large majority of my career in markets, were seen as the anchor point for global duration,' Hornbach says. 'Even though Japanese government bonds may no longer be considered the global anchor for bond markets, other central banks are acting in ways that I think ultimately lead to lower interest rates around the world.' New Grads Join Worst Entry-Level Job Market in Years The Spying Scandal Rocking the World of HR Software American Mid: Hampton Inn's Good-Enough Formula for World Domination Cavs Owner Dan Gilbert Wants to Donate His Billions—and Walk Again The SEC Pinned Its Hack on a Few Hapless Day Traders. The Full Story Is Far More Troubling ©2025 Bloomberg L.P. 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
