NeuBase Therapeutics, Inc. Email Format

PharmaceuticalsPennsylvania, United States11-50 Employees

NeuBase Therapeutics is a Pittsburgh-based biotechnology company focused on developing ultra-precision genetic medicines that address disease at its genetic root. It targets rare monogenic diseases with no approved therapies and also aims at more common genetic disorders, including cancers that are resistant to existing treatments. Its pipeline includes therapeutic candidates for myotonic dystrophy type 1 and Huntington's disease, as well as cancer-driving KRAS mutations G12V and G12D. The company operates with a relatively small workforce within the pharmaceutical manufacturing sector.

In August 2023, NeuBase announced progress on Stealth Editors, a technology intended to enable in vivo gene editing while avoiding immune system activation. This development reflects the company's focus on advancing gene-based therapies that reach the genetic basis of disease.

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