Modalis Therapeutics Corporation announced that the company has been accepted for an Oral Presentation at the Global Cell & Gene Therapy Summit (July 8-10, 2024, Boston, USA), and the following research results will be presented by CSO, Dr. Tetsuya Yamagata. Product concept and rationale for MDL-101, a differentiated precision medicine for LAMA2-congenital muscular dystrophy (LAMA2-CMD). Preclinical data supports efficacy in LAMA-2 knockout mice (dyW disease model mice) and target engagement in non-human primates (NHPs).

The possibility that CRISPR-based Epigenome editing technology may show clinical efficacy as a therapeutics approach to treat serious genetic diseases. MDL-101 is an experimental, epigenome modulation therapy under investigation for the treatment of LAMA2-Congenital Muscular Dystrophy (LAMA2-CMD). MDL-101 is comprised of guide nucleotide targeting LAMA-1 gene, a highly homologous sister gene of the disease-causing gene LAMA-2, enzyme-null Cas9 (dCas9) fused with trans-activating domain driven by a muscle-specific promoter and coded in a muscle-specific AAV vector.

MDL-101 upregulates LAMA-1 gene products in patients' muscle tissue to compensate for loss-of-function caused by mutation of LAMA-2, and therefore has the potential to provide a one-time, durable treatment benefit for people living with LAMA2-CMD. Modalis Therapeutics develops precision genetic medicines using epigenome editing technology. Modalis is pursuing therapies for orphan genetic diseases using its proprietary CRISPR-GNDM® technology which enables the gene/locus-specific modulation of gene expression or epigenetic editing without the need for DNA cleavage or altering DNA sequence.

Headquartered in Tokyo with laboratories and facilities in Waltham Massachusetts, the company is listed on Tokyo Stock Exchange's Growth market.