" class="no-js "lang="en-US"> Modalis to Present Data for Muscular Dystrophy Treatment
Friday, June 09, 2023

Modalis Therapeutics to Present Data for Muscular Dystrophy Treatment

Modalis Therapeutics, a pioneering company developing innovative products for the treatment of rare genetic diseases utilizing its proprietary CRISPR-GNDM® epigenetic editing technology, today announced that its preclinical data for its precision medicine approach for LAMA2 Congenital Muscular Dystrophy has been accepted for a presentation in the late-breaking session at the 26th Annual Meeting of The American Society of Gene & Cell Therapy (ASGCT), being held in Los Angeles CA on May 16-20, 2023. The abstracts present preclinical data from the Company’s LAMA2 Congenital Muscular Dystrophy (LAMA2-CMD).

Modalis presentations at ASGCT will include preclinical data demonstrating that in LAMA-2 knockout mice (dyW disease model mice) and non-human primates, a CRISPR-GNDM® based molecule (MDL-101) targeting the LAMA-1 gene introduced into a muscle-specific AAV vector raised LAMA-1 expression to levels that complement LAMA-2 function in the diseased animals. This suggests that MDL-101 may have therapeutic potential in the clinic.

“Our proprietary and world-first CRISPR-based epigenetic editing technology, CRISPR-GNDM®, controls the expression levels of disease-causing genes and provides a disease-modifying treatment for genetic disorders,” said Haru Morita, CEO of Modalis. “MDL-101 has raised its firepower as a potentially life-changing gene therapy for the treatment of LAMA2 Congenital Muscular Dystrophy by employing a muscle-specific capsid that further increases the selectivity for muscle tissue. Development is currently on track for IND filing by the end of 2024. At this meeting, we will share data from our mice and NHPs studies that have shown efficacy and safety and discuss the potential implications for therapeutic efficacy in the clinic.”

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