NeuBase Therapeutics Inc

NeuBase is accelerating the genetic revolution by developing a new class of precision genetic medicines which can be designed to increase, decrease, or change gene function, as appropriate, to resolve genetic defects that drive disease. NeuBase’s targeted PATrOL™ therapies are centered around its proprietary drug scaffold to address genetic diseases at the DNA or RNA level by combining the highly targeted approach of traditional genetic therapies with the broad organ distribution capabilities of small molecules. With an initial focus on silencing disease-causing mutations in debilitating neuromuscular, neurological and oncologic disorders, NeuBase is committed to redefining medicine for the millions of patients with both common and rare conditions.

NeuBase has developed its PATrOL platform to overcome the hurdles of delivery, tolerability, selectivity, manufacturability, durability and scalability that limit other genetic medicine technologies.

In preclinical studies, NeuBase has successfully validated the platform's ability to efficiently deliver genetic medicines with broad tissue distribution, including into the deep brain, and precisely engage DNA and RNA mutations in a manner that is well-tolerated with the potential for sustained efficacy.