Modalis Therapeutics Corporation (Tokyo Stock Exchange: 4883, CEO: Haru Morita) has announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to MDL-101, a novel precision medicine for congenital muscular dystrophy type 1A (LAMA2-CMD). Modalis specializes in treatments for rare genetic diseases using its proprietary CRISPR-based epigenome editing technology, CRISPR-GNDM®.
The ODD, established under the Orphan Disease Act of 1983, supports the development of drugs for conditions affecting fewer than 200,000 patients in the U.S. This designation aims to expedite the availability of safe, effective treatments for rare diseases that currently lack sufficient research and development.
CEO Haru Morita emphasized the severity of LAMA2-CMD, a life-threatening condition that leads to muscle weakness and reduced survival, noting that there are no approved treatments in the U.S. He expressed hope that MDL-101 could be the first therapy to improve patient outcomes by activating LAMA1, a sister gene to the disease-causing LAMA2 gene.
LAMA2-CMD is a severe congenital muscular dystrophy resulting from the absence of the LAMA2 protein, which is too large to be effectively delivered via conventional gene therapy. Modalis’ CRISPR-GNDM® technology allows for precise modulation of gene expression without altering the patient’s DNA, aiming to induce LAMA1 expression in muscle tissues to compensate for the missing LAMA2 function.
Modalis is committed to its mission, “Every Life Deserves Attention,” focusing on developing life-changing treatments for patients with currently incurable diseases.
About MDL-101
MDL-101 is an experimental epigenetic editing therapy targeting LAMA2-CMD. It consists of a guide nucleotide that targets the LAMA1 gene, an enzyme-null Cas9 (dCas9) fused with a trans-activating domain, all delivered via a muscle-specific AAV vector. By upregulating LAMA1 expression in muscle tissue, MDL-101 aims to provide a one-time, durable treatment for those affected by LAMA2-CMD.
About Modalis
Modalis Therapeutics develops precision genetic medicines utilizing its epigenome editing technology. The company is dedicated to addressing orphan genetic diseases through its CRISPR-GNDM® platform, which allows for gene-specific modulation of expression without the need for DNA cleavage. Headquartered in Tokyo, with research and development operations in Waltham, Massachusetts, Modalis is listed on the Tokyo Stock Exchange’s Growth market.
