SOLVE FSHD and Modalis Partner to Develop CRISPR Treatment for FSHD

VANCOUVER, Canada & TOKYO, Japan & WALTHAM, MA, USA | June 09, 2025

SOLVE FSHD, a venture philanthropy organization focused on accelerating therapies for facioscapulohumeral muscular dystrophy (FSHD), has entered into a strategic partnership with Modalis Therapeutics Corporation. Modalis, a leader in CRISPR-based epigenome editing aimed at treating rare genetic disorders, will collaborate with SOLVE FSHD to create an innovative treatment for FSHD, a muscle disorder affecting around one million people globally. This new therapy will utilize Modalis's proprietary CRISPR-GNDM® (Guide Nucleotide-Directed Modulation) technology, which can adjust gene expression without causing double-strand DNA breaks.

The collaboration will see SOLVE FSHD providing strategic funding to support the development of Modalis's MDL-103 program. This program aims to deliver a cutting-edge treatment by persistently reducing the expression of the DUX4 gene, identified as the toxic gene responsible for FSHD, which becomes abnormally active due to epigenetic alterations in the D4Z4 repeat region on chromosome 4. MDL-103 is engineered for sustained effectiveness over lengthy periods under the guidance of a robust, muscle-specific promoter. It is administered to patients' muscles using a muscle-tropic AAV delivery method. Modalis’s CRISPR-GNDM® technology holds the promise of revolutionizing FSHD treatment by epigenetically silencing DUX4 expression.

Eva Chin, Executive Director of SOLVE FSHD, expressed satisfaction in partnering with Modalis and incorporating them into their diverse array of collaborators working towards FSHD therapies. "SOLVE FSHD identified Modalis for their commitment to curing this challenging condition. We were impressed by their unique approach to addressing the epigenetic origins of FSHD, using a platform that has shown potential in other neuromuscular disorders. We believe our support will expedite Modalis's progress of MDL-103 into clinical trials," noted Chin.

Haru Morita, CEO of Modalis, expressed enthusiasm about the collaboration and appreciated the support for developing MDL-103. "This strategic partnership is a major endorsement of Modalis’s CRISPR-GNDM® technology and our MDL-103 program. As pioneers in this area, we have shown promising long-term drug efficacy in mouse models, stable target engagement and safety in non-human primates, and excellent biodistribution in neuromuscular disorders. We believe MDL-103, which combines CRISPR-GNDM® technology with a muscle tropic AAV delivery system, represents a significant opportunity as a novel treatment for FSHD," Morita stated.

About SOLVE FSHD:
Established by Canadian entrepreneur and philanthropist Chip Wilson, SOLVE FSHD is a venture philanthropy group dedicated to sparking innovation and advancing research to find a cure for FSHD. With a commitment of $100 million from the Wilson family, the organization aims to slow or halt muscle degeneration, boost muscle regeneration and strength, and improve the quality of life for individuals with FSHD by 2027.

About Modalis Therapeutics Corporation:
Founded in 2016 and based in Massachusetts, USA, Modalis is a leading entity in the field of epigenetic medicine. The company is engaged in developing therapies for patients affected by serious genetic conditions such as neuromuscular and CNS disorders, along with cardiomyopathies. Modalis’s proprietary CRISPR-GNDM® technology is adept at specifically modulating the expression of genes related to diseases without causing double-strand DNA breaks.