Develops antisense therapies for genetic diseases, focusing on next-generation oligonucleotide therapies.
NeuBase Therapeutics, Inc., headquartered in Pittsburgh, Pennsylvania, is an innovative preclinical-stage biopharmaceutical company dedicated to advancing therapies for rare genetic diseases and cancers driven by genetic mutations. Established with a focus on pioneering treatments, NeuBase leverages its proprietary modular peptide-nucleic acid antisense oligo (PATrOL) platform.
The company's PATrOL platform is specifically designed to target and address the underlying causes of diseases such as Huntington's disease (HD), myotonic dystrophy, and various genetic disorders. NeuBase's pipeline includes promising candidates like NT0100 for Huntington's disease, NT0200 targeting myotonic dystrophy type 1, and NT0300 focusing on mutations in the KRAS gene, pivotal in numerous cancer and oncology applications.
Through its cutting-edge approach and strategic research initiatives, NeuBase Therapeutics aims to revolutionize the treatment landscape by providing effective therapies for patients with significant unmet medical needs. By focusing on precision medicine and genetic targeting, the company continues to drive innovation in biopharmaceuticals, poised to make a profound impact on improving patient outcomes worldwide.