Modalis Therapeutics' (TYO:4883) patent application for CRISPR-GNDM(r) based gene therapy MDL-101 as a treatment for LAMA2-CMD was granted in Japan, according to a Friday filing.
LAMA2-CMD is a severe congenital muscular dystrophy caused by the absence of the LAMA2 protein, for which there are currently no effective treatments due to the gene's large size.
Modalis' CRISPR-GNDM technology offers a novel solution by increasing the expression of the LAMA1 gene to compensate for LAMA2 deficiency without introducing double-strand DNA breaks.
The patent, alongside the US orphan drug designation, strengthens Modalis' intellectual property and paves the way for securing patents in other major countries.
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