Major Drugs - Pittsburgh, PA, US
NeuBase is developing ultra precision genetic medicines targeting rare, monogenic diseases, for which there are no approved therapies, as well as more common genetic disorders, including cancers that are resistant to current therapeutic approaches. NeuBase's pipeline includes therapeutic candidates for the treatment of myotonic dystrophy type 1 (DM1), Huntington's disease (HD), and cancer-driving point mutations in KRAS G12V and G12D, which are involved in many tumor types and have historically been "undruggable."
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