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
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
Yahoo
an hour ago
- Yahoo
DeFi Leader Aave Debuts on Sony-Backed Soneium Blockchain
Decentralized finance (DeFi) firm Aave is arriving on Soneium, the Ethereum L2 blockchain linked to Japanese electronics giant Sony, the companies said on Tuesday. Decentralized finance, which shot to prominence during the so-called 'DeFi summer' of 2020, is resurgent, amid a huge growth in stablecoins and crypto-backed lending. The integration of GHO, Aave's decentralized overcollateralized stablecoin, into Sonieum is being explored for real-world use cases in payments, savings, and digital commerce, according to a press release. Soneium's 7-million user ecosystem already includes Uniswap v4, Velodrome, Stargate, Lido, and native protocols like Kyo Finance and SuperVol. 'Aave wants to be where consumers are, in trusted, accessible environments,' said Aave Labs Founder Stani Kulechov in a statement. 'Sony's global reputation and Soneium's focus on consumer-facing applications make this a unique opportunity for Aave to reach new mainstream users.' The partnering will include Aave's participation in upcoming liquidity incentive campaigns, including with the Astar, a blockchain prominent in the Japanese Web3 ecosystem, and a 100 million ASTR allocation (currently valued at $4M) to bootstrap adoption, according to a press release. Soneium, which this week announced a new gaming incubator to help drive more traffic to the blockchain, is developed by Sony Block Solutions Labs, a joint venture between Sony Group and Startale Group.
Yahoo
2 hours ago
- Yahoo
Japan's shift to cashless society prods BOJ call for payment innovation
By Leika Kihara TOKYO (Reuters) -Bank of Japan officials are ramping up calls to keep pace with rapid developments around digital currencies that may accelerate a shift to cashless payments in a country better known for its belief that "cash is king." Once seen as a society favouring physical currency, Japan saw the ratio of cashless payments rise to 42.8% in 2024, up from 13.2% in 2010 and hitting the government's 40% goal a year earlier than targeted, government data showed. While Japan is a global laggard on payment technology, the increase in cashless transactions is forcing policymakers to ensure they are ready to adapt to shifting public preference on payment and settlement means. This includes the option of a central bank digital currency (CBDC). While no decision has been made on whether to issue a digital yen, the BOJ began a pilot programme for developing a CBDC in 2023, and has been consulting with private firms and the government on its framework and design. "Although banknote issuance remains high in Japan, usage of notes could fall significantly in the future amid rapid digitalisation," BOJ Executive Director Kazushige Kamiyama told a meeting last week with private firms on the pilot programme. "As such, Japan must consider what steps it can take now to ensure its retail settlement system is convenient, efficient, accessible universally, while being safe and resilient." BOJ Deputy Governor Shinichi Uchida said a CBDC may become "a critical piece of infrastructure" shaping the future of Japan's payment and settlement systems, but stressed that he did not expect demand for cash to disappear in Japan any time soon. In his speech on Saturday, Uchida spoke about a hypothetical world in which the yen is overtaken by another instrument - such as crypto assets - as the main form of payment in Japan. Such a world would not occur as long as the public believes in the BOJ's ability to keep the value of the yen stable, which could come into question if the central bank fails to fulfil its mission of price stability, Uchida said. "Looking to the future, in a society that has made significant advances in digitalization, there is no guarantee that currency issued by the central bank of a sovereign nation will continue to function as a generally acceptable payment instrument," he said. CBDCs are back in the spotlight after U.S. President Donald Trump issued an executive order in January banning the Federal Reserve from issuing a digital dollar, seemingly as part of a drive to promote cryptocurrencies and stablecoins instead. The growing presence of stablecoins, or cryptocurrencies backed by a hard currency, and the potential risk of an erosion in the U.S. dollar's dominance have also piled pressure on central banks to consider issuing their own digital currencies. The European Central Bank has called for the need to issue a digital euro to respond to Trump's push to promote stablecoins, and develop an electronic means of payment that does not rely on dominant U.S. providers such as Visa. China is racing ahead with global ambitions to internationalise the yuan with transactions of the digital yuan more than tripling between June 2023 and June 2024.
