Genetic medicines developer Modalis Therapeutics Corporation (Tokyo:4883) announced on Friday that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to MDL-101, a potential treatment for congenital muscular dystrophy type 1a (LAMA2-CMD).

The ODD will provide Modalis with certain development incentives, including tax credits, user fee waivers and market exclusivity.

LAMA2-CMD is a rare, life-threatening disease with no approved treatments currently available. MDL-101, which uses Modalis' proprietary CRISPR-GNDM technology, aims to address the underlying cause of the disease by activating LAMA1, the sister gene of LAMA2.