Modalis Therapeutics Earns FDA Rare Pediatric Disease Designation

Modalis Therapeutics Corporation (JP:4883) has released an update.

Modalis Therapeutics Corporation, leveraging its CRISPR-GNDM® epigenome editing technology, has received the FDA’s Rare Pediatric Disease designation for MDL-101, a potential first-in-class therapy targeting congenital muscular dystrophy type 1a (LAMA2-CMD). The designation could lead to a priority review voucher for Modalis upon FDA marketing approval. MDL-101 aims to compensate for the LAMA2 protein deficiency by inducing the expression of the LAMA1 gene across muscle tissues.

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