NBSE Profile
NeuBase Therapeutics, Inc. is a preclinical-stage biopharmaceutical company based in Pittsburgh, Pennsylvania, specializing in the development of advanced therapies for rare genetic disorders and cancer. The company is at the forefront of creating novel treatments utilizing its proprietary modular peptide-nucleic acid antisense oligo (PATrOL) platform. This innovative technology is designed to precisely target and modulate mutant genes responsible for a variety of genetic diseases and oncological conditions.
The PATrOL platform represents a significant advancement in therapeutic development by offering a customizable approach to gene modulation. NeuBase Therapeutics' pipeline includes several promising candidates. NT0100 is being developed as a potential treatment for Huntington's disease (HD), a neurodegenerative disorder characterized by the progressive breakdown of nerve cells. NT0200 is targeted at myotonic dystrophy type 1, a genetic condition that causes muscle weakness and wasting. Additionally, NT0300 aims to address cancers associated with mutations in the KRAS gene, which is frequently implicated in various malignancies.
NeuBase's focus on rare genetic diseases and cancer reflects its commitment to addressing unmet medical needs with precision medicine. The company's approach involves leveraging its PATrOL platform to deliver therapies that can potentially correct or mitigate the effects of genetic mutations at a molecular level. This strategy not only aims to provide more effective treatments but also seeks to offer targeted solutions with fewer side effects compared to traditional therapies.
Founded with a vision to revolutionize the treatment of genetic and oncological diseases, NeuBase Therapeutics, Inc. continues to advance its research and development efforts. As it progresses through the preclinical stages and prepares for clinical trials, the company remains dedicated to exploring the full potential of its PATrOL technology. NeuBase’s mission is to transform the landscape of genetic and cancer therapeutics, offering new hope for patients with conditions that are currently underserved by existing treatments.
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