Modalis Therapeutics to Present Data Supporting of Development of Transformative… – Press Release


MDL-101 preclinical data support efficacy and safety of a differentiated precision medicine approach for LAMA2 Congenital Muscular Dystrophy (LAMA2-CMD)

Preclinical data showing that our CRISPR-based epigenetic editing technology regulates the expression of target genes in mammals, suggesting the possibility of clinical efficacy as a therapeutic approach for serious genetic disorders

Modalis Therapeutics Corporation (Tokyo Stock Exchange: 4883), a pioneering company developing innovative products for the treatment of rare genetic diseases utilizing its proprietary CRISPR-GNDM® epigenetic editing technology, today announced that the abstract 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 (NHPs), 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 disease animals. This suggests that MDL-101 may have therapeutic potential in the clinic.

At ASGCT, we will present preclinical data from MDL-101 validating the efficacy and safety of our differentiated therapeutic strategy utilizing our CRISPR-GNDM® technology for the previously difficult-to-treat LAMA2-CMD. “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-CMD by…