nL-GARS1-001

nL-GARS1-001 is a personalized antisense oligonucleotide (ASO) developed by the n-Lorem Foundation for the treatment of Charcot-Marie-Tooth disease type 2D (CMT2D). CMT2D is an ultra-rare, dominantly inherited peripheral neuropathy caused by toxic gain-of-function mutations in the *GARS1* gene, which encodes glycyl-tRNA synthetase 1. This enzyme is essential for charging tRNA with glycine during protein synthesis; however, mutant forms of the protein lead to the selective degeneration of motor and sensory axons. nL-GARS1-001 is designed to selectively bind to and trigger the degradation of mutant *GARS1* mRNA transcripts, thereby reducing the levels of the toxic protein while ideally sparing the wild-type version. This targeted knockdown approach aims to halt axonal degeneration and stabilize or improve motor and sensory function. The drug is administered via intrathecal injection and is currently being evaluated in a patient-specific clinical trial.