LMT801

LMT801 is a first-in-class, orally available small molecule therapy being developed by Lmito Therapeutics in partnership with the Charcot-Marie-Tooth Research Foundation (CMTRF) for the treatment of Charcot-Marie-Tooth disease type 1A (CMT1A). CMT1A is a rare genetic peripheral neuropathy caused by a duplication of the PMP22 gene, which leads to the overproduction of the PMP22 protein and subsequent demyelination of peripheral nerves. LMT801 is described as a disease-modifying therapy intended to restore nerve function and promote remyelination. As of early 2024, the drug is in the discovery phase of development, and its specific biological target and precise mechanism of action remain undisclosed.