Genetic medicines company Modalis Therapeutics Corporation (Tokyo Stock Exchange: 4883) announced on Monday that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation to MDL-101, a novel treatment for congenital muscular dystrophy type 1a (LAMA2-CMD).

This designation applies to serious diseases affecting children under 18, with fewer than 200,000 cases in the US. Should MDL-101 receive marketing approval, Modalis may obtain a Priority Review Voucher (PRV) for expedited FDA review of another product.

LAMA2-CMD is a severe genetic disorder caused by the absence of the LAMA2 protein, which poses challenges for conventional gene therapy development. Modalis' proprietary CRISPR-GNDM technology enables precise modulation of gene expression without DNA breaks. MDL-101 aims to compensate for LAMA2 deficiency by inducing expression of the sister gene, LAMA1, in muscle tissues, potentially providing a one-time, durable treatment.

Modalis is also pursuing an Orphan Drug application for MDL-101, currently under FDA review. The company's mission is to deliver life-changing treatments for rare genetic diseases.