NT-0231.F

**NT-0231.F** is an investigational genetic therapy developed by NeuBase Therapeutics for **myotonic dystrophy type 1 (DM1)**, a genetic disorder caused by a trinucleotide expansion in the *DMPK* gene that produces toxic RNA hairpins sequestering RNA splicing proteins. It utilizes a novel **peptide-nucleic acid (PNA) pharmacophore** to selectively target mutant *DMPK* pre-mRNA, disrupting the RNA hairpin to release splicing proteins like MBNL, thereby restoring normal RNA splicing, reducing nuclear aggregates, and reversing myotonia. Preclinical studies in the HSA^LR mouse model demonstrated splice rescue (including *Clcn1* transcript), nuclear aggregate resolution, and myotonia reversal following single IV or IM doses, or multiple SC doses, with effects lasting at least 6 weeks and broad tissue distribution including muscle, heart, and brain.[1][3][9]