Targeting Orphan Diseases Using Macrocycles
AlloMek strives to develop innovative therapeutics to patients with rare and debilitating diseases
Welcome to Allomek
AlloMek, founded in 2011, is a privately held orphan disease-focused company, developing therapeutics for a catastrophic cardiomyopathy (based on discovery by Dr. Worman’s group at Columbia University) and congenital muscular disease for which there are no disease-modifying treatments, caused by LMNA genetic mutations (laminopathies). In parallel, the Company plans to pursue other MEK-associated orphan diseases, specifically, Neurofibromatosis Type 1 and Noonan Syndrome. AlloMek’s the lead compound, CIP-137401, is a unique, proprietary, macrocyclic small molecule MEK kinase inhibitor, it has demonstrated powerful proof of concept (POC) in a well-established mouse model of Emery-Dreifuss muscular dystrophy caused by LMNA gene mutations. CIP-137401 can potentially be used as a single agent and in combination with other treatment options to enhance clinical response.