Modalis Therapeutics Corporation posted the preprint paper titled "Efficient and durable gene activation by Cas9-mediated epigenome editing in vivo" on bioRxiv. The company reported data demonstrating exceptional durability, robust efficacy and safety in dyW mouse disease model of LAMA2-CMD and in adult and juvenile NHPs. LAMA2-CMD is a severe, early onset congenital muscular dystrophy caused by the absence of the LAMA2 protein.

Despite significant advances in gene therapy and the approval of about a dozen therapies, the size of the disease-causing gene of LAMA2-CMD, which exceeds 3,000 amino acids, hinders the conventional gene therapy approach using AAV vectors to deliver the healthy version of the mutated gene. No approved therapies to address the root cause of this condition exist or are in clinical trials. Modalis' proprietary CRISPR-GNDM, is capable of specific modulation of the expression of disease-relevant genes, without introducing double-strand DNA breaks, and the company's MDL-101 is potentially the first-in-class therapeutics to solve the challenge and provide life-changing therapeutics for the patients of LAMA2-CMD.