SOLVE FSHD Enters Strategic Collaboration with Modalis

Rare Daily Staff

The venture philanthropy organization SOLVE FSHD has entered into a strategic collaboration with Modalis Therapeutics to develop a CRISPR-based epigenome editing therapy for facioscapulohumeral muscular dystrophy, a debilitating muscular disorder.

The novel therapy leverages Modalis’s proprietary CRISPR-GNDM (Guide Nucleotide-Directed Modulation) technology, which can modulate gene expression without introducing double-strand DNA breaks.

Facioscapulohumeral muscular dystrophy (FSHD) is a rare, progressive, and variable hereditary muscle-weakening condition marked by significant pain, fatigue, and disability. It is characterized by progressive and often asymmetric skeletal muscle loss that initially causes muscle weakness in the face, shoulders, arms, and trunk and can progress to weakness in muscles in the lower body.

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 under the control of a strong, muscle-specific promoter. It is delivered to the muscles of patients using a muscle-tropic AAV delivery system.

“SOLVE FSHD identified Modalis as a company committed to finding a cure for this debilitating condition,” said Eva Chin, executive director of SOLVE FSHD. “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.”

Photo: Eva Chin, executive director of SOLVE FSHD