NeuBase Therapeutics, Inc. is a biotechnology company, which engages in the development of drugs for patients with genetic neurological disorders. The company is headquartered in Pittsburgh, Pennsylvania. The company went IPO on 2014-05-28. The company is developing a modular peptide-nucleic acid (PNA) antisense oligo (PATrOL) platform to address genetic diseases, with a single, cohesive approach. The Company’s programs are NT-0100 in HD, NT-0200 in myotonic dystrophy type 1 (DM1) and NT-0300 in KRAS-driven cancers. The NT-0100 program is a PATrOL-enabled therapeutic program being developed to target the mutant expansion in the HD DNA or RNA. The NT-0200 program is a PATrOL-enabled therapeutic program being developed to target the mutant expansion in the DM1 disease RNA. The NT-0300 program is a PATrOL-enabled therapeutic program being developed to target the mutated KRAS gene. The company uses its platform to address diseases which have a genetic source, with an initial focus on gene silencing in DM1, Huntington’s disease (HD), and oncology and in gene editing applications. The company also focused on identifying and evaluating multiple indications for potential development.

Industry: Pharmaceutical Preparations Peers: Bone Biologics Corp American CryoStem Corp BIOCEPT INC Ernexa Therapeutics Inc Calithera Biosciences, Inc. Skye Bioscience Inc MetaVia Inc Transcode Therapeutics Inc