Modalis Therapeutics: FDA Grants Rare Pediatric Disease Designation to MDL-101 for the Treatment of Congenital Muscular Dystrophy Type 1a (LAMA2-CMD)

TOKYO & WALTHAM, Mass.--(BUSINESS WIRE)--Modalis Therapeutics Corporation (Tokyo Stock Exchange: 4883, CEO: Haru Morita), a leading company pioneering treatments for rare genetic diseases using its proprietary CRISPR-based epigenome editing technology, CRISPR-GNDM^®, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation to MDL-101, a novel precision medicine being developed for the treatment of congenital muscular dystrophy type 1a (LAMA2-CMD).

The FDA grants RPD designations to treatments for serious and life-threatening diseases that primarily affect children under 18 years old and affect fewer than 200,000 people in the U.S. If a product with RPD designation receives marketing approval, the company may be awarded a Priority Review Voucher (PRV), allowing for faster FDA review of another product. Modalis has also submitted an Orphan Drug application for MDL-101, which is currently under separate review by the FDA.

“We are pleased that the FDA has recognized our development efforts for the rare disease and granted us RPD designation. We have received many requests for our efforts from children and families around the world suffering from this disease for which there is currently no treatment, and we feel a mission to respond to the expectations of patients who are eagerly awaiting the start of clinical trials as soon as possible,” said Haru Morita, CEO of Modalis. “This designation from the FDA is proof that our efforts are rational and achieving results, and we hope to further accelerate the development of this world's first product using the cutting-edge technology.”

LAMA2-CMD is a severe, early onset congenital muscular dystrophy caused by the absence of the LAMA2 protein, which is made up of more than 3,000 amino acids. Because it cannot be loaded onto an AAV vector, it is thought that the conventional approach to gene therapy development is difficult. For this reason, there is currently no treatment, including gene therapy, that addresses the underlying cause of LAMA2-CMD.

Modalis’ proprietary CRISPR-GNDM® technology allows for precise modulation of gene expression without introducing double-strand DNA breaks. MDL-101, Modalis’ lead candidate for LAMA2-CMD, represents a first-in-class therapeutic that aims to address the unmet need by inducing expression of the sister gene LAMA1 in muscle tissues, thereby compensating for the deficient function of LAMA2.

Guided by its mission, "Every Life Deserves Attention," Modalis is dedicated to bringing life-changing treatments to patients suffering from diseases for which no cure currently exists.

About MDL-101

MDL-101 is an experimental, epigenetic editing therapy that is being developed for the treatment of LAMA2-Congenital Muscular Dystrophy (LAMA2-CMD). MDL-101 is comprised of a guide nucleotide targeting LAMA1 gene, a highly homologous sister gene of the disease-causing gene LAMA2, 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 LAMA1 gene products in patients’ muscle tissue to compensate for loss-of-function caused by mutation of LAMA2, and therefore has the potential to provide a one-time, durable treatment to benefit people living with LAMA2-CMD.

About Modalis:

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 epigenome editing without the need for DNA cleavage or altering DNA sequence. Headquartered in Tokyo with all research and development operations in Waltham Massachusetts, the company is listed on the Tokyo Stock Exchange’s Growth market. For additional information, visit www.modalistx.com/en/.