ENTDMD001

ENTDMD001 is a non-viral gene therapy candidate being developed by Entos Pharmaceuticals, in collaboration with CureDuchenne, for the treatment of Duchenne muscular dystrophy (DMD). The program utilizes Entos' proprietary Fusogenix Proteo-Lipid Vehicle (PLV) platform, which incorporates fusion-associated small transmembrane (FAST) proteins into lipid nanoparticles to facilitate direct delivery of genetic payloads into target cells via membrane fusion. This mechanism is designed to bypass endocytosis and lysosomal degradation, potentially offering a more efficient and less immunogenic alternative to traditional viral vector-based gene therapies. ENTDMD001 is currently in the discovery stage, aiming to deliver a therapeutic transgene to restore dystrophin expression in muscle tissues.