Rare genetic diseases collectively affect hundreds of millions of people worldwide

Neurological Disorders
Muscular Dystrophies
Ocular Diseases

The next generation of gene silencing technology

NeuBase has developed a modular antisense peptide nucleic acid (PNA) platform with the capability to address rare genetic disease caused by mutant proteins with a single, cohesive approach. NeuBase has improved gene silencing therapies to include the advantages of synthetic approaches with the precision of antisense technologies.


The advantages of the NeuBase PATrOL™ platform enable the company to cohesively develop therapies for a wide range of rare genetic diseases, including repeat expansion disorders, dominant and recessive genetic disorders and cancers driven by specific oncogenic mutations.

NeuBase is first addressing Huntington’s disease and myotonic dystrophy.

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The NeuBase technology has several advantages over traditional ASOs


Scalable & Modular Development

Pre-organized peptide backbone and toolkit of engineered nuclear bases allow drugs to be quickly snapped together for many targets


Targeting Secondary RNA Structures

Higher specificity for mutant over wild type transcript, allowing for fewer off-target effects and more effective deactivation of disease-related mRNA


Systemic Administration

Ability to cross the blood brain barrier and disseminate broadly, allowing for systemic administration in neurological indications


No Immune Response

Do not cause an immune response when systemically administered, enabling us to develop solutions for pre-symptomatic individuals who are gene positive

NeuBase Therapeutics to Present at the LD Micro Conference

NeuBase Therapeutics, Inc. (“NeuBase”), a biotechnology company developing next generation antisense therapies to address genetic diseases, announced today that Dietrich Stephan, Chief Executive Officerwill present a corporate overview and business update at the LD Micro 9th Annual Invitational, being held at the Luxe Sunset Boulevard Hotel in Los Angeles, California.

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NeuBase Therapeutics Designates Industry Leaders to Board of Directors

NeuBase Therapeutics, Inc. (“NeuBase”), a biotechnology company developing next generation antisense therapies to address genetic diseases, today announced that it has designated four experienced executives from the biotechnology industry for its board of directors (the “Board”), including Dov A. Goldstein, M.D., M.B.A., Diego Miralles, M.D., Franklyn Prendergast, M.D., Ph.D., and Eric Richman, M.B.A, to become effective upon the closing of the Company’s merger with Ohr Pharmaceutical, Inc. (NASDAQ: OHRP) (“Ohr”). The four designees, together with Dietrich A. Stephan, Ph.D., chairman and chief executive officer of the company, will comprise the entire board of directors for the combined company.

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NeuBase Therapeutics Announces Agreements for $9 Million Financing

NeuBase Therapeutics, Inc. (“NeuBase”), a biotechnology company developing next generation antisense therapies to address rare genetic diseases, today announced that it has entered into definitive agreements for a $9.0 million financing that will close immediately prior to the closing of the company’s proposed merger with Ohr Pharmaceutical, Inc. (Nasdaq: OHRP) (“Ohr”). The oversubscribed financing includes institutional and accredited investors, including participation by Cyto Ventures, a life sciences-focused venture capital fund affiliated with Dietrich Stephan, Ph.D., the CEO of NeuBase Therapeutics, and a member of Ohr’s board of directors.

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Shape selective bifacial recognition of double helical DNA

An impressive array of antigene approaches has been developed for recognition of double helical DNA over the past three decades; however, few have exploited the ‘Watson–Crick’ base-pairing rules for establishing sequence-specific recognition. One approach employs peptide nucleic acid as a molecular reagent and strand invasion as a binding mode. However, even with integration of the latest conformationally-preorganized backbone design, such an approach is generally confined to sub-physiological conditions due to the lack of binding energy. Here we report the use of a class of shape-selective, bifacial nucleic acid recognition elements, namely Janus bases, for targeting double helical DNA or RNA. Binding occurs in a highly sequence-specific manner under physiologically relevant conditions. The work may provide a foundation for the design of oligonucleotides for targeting the secondary and tertiary structures of nucleic acid biopolymers.

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