WALTHAM, Mass. — Modalis Therapeutics Corporation said a new study demonstrating efficient activation of the LAMA1 gene as a potential treatment for LAMA2-related congenital muscular dystrophy has been published in the peer-reviewed journal Human Gene Therapy.
The research article, titled “Efficient LAMA1 gene activation by epigenome editing as a therapeutic approach for LAMA2-CMD,” presents preclinical data showing that the company’s CRISPR-GNDM epigenome editing platform can safely and effectively activate the LAMA1 gene, offering a novel therapeutic strategy for LAMA2-CMD, a severe pediatric neuromuscular disorder for which no approved treatments currently exist.
According to the company, the study demonstrated robust, muscle-specific activation of LAMA1 following a single administration of muscle-tropic adeno-associated virus vectors encoding the GNDM activator. In DyW mouse models of the disease, researchers observed marked improvements in survival and muscle function, including dose-dependent gains in grip strength and normalization of muscle tissue structure. The study also reported favorable safety and pharmacodynamic profiles in non-human primates, with strong LAMA1 induction observed even at reduced doses in infant subjects.
“This work represents one of the first demonstrations of systemic, single-vector epigenome activation in large animals,” said Tetsuya Yamagata, M.D., Ph.D., chief scientific officer of Modalis. “By enabling targeted activation of large genes that are not compatible with conventional AAV gene replacement approaches, this study opens the door to a new class of treatments for a wide range of genetic diseases.”
Chief executive officer Haru Morita said the publication validates the company’s lead development program and broader technology platform. “The publication of this study demonstrates that the nonclinical results supporting our lead program MDL-101 have been recognized by the global scientific community as clear evidence of both efficacy and safety,” Morita said. “These data form the scientific foundation for our planned clinical trials next year and significantly reinforce confidence in the broader CRISPR-GNDM platform.”
Morita added that development of MDL-101 is progressing, with GLP toxicology studies and GMP manufacturing underway. The company plans to submit an investigational new drug application after those efforts are completed, with the goal of advancing a one-time treatment option for patients living with LAMA2-related congenital muscular dystrophy.
