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 Receives Notification of Deficiency from Nasdaq Related to Delayed Quarterly Report on Form 10-Q

PITTSBURGH, Feb. 21, 2020 (GLOBE NEWSWIRE) -- NeuBase Therapeutics, Inc. (Nasdaq: NBSE) (“NeuBase” or the “Company”) announced today that it received a standard notification letter dated February 20, 2020 (the “Notice”) from The Nasdaq Stock Market LLC (“Nasdaq”) stating that, as a result of not having timely filed its Quarterly Report on Form 10-Q for the period ended December 31, 2019, NeuBase is not in compliance with Nasdaq Listing Rule 5250(c)(1), which requires timely filing of periodic financial reports with the Securities and Exchange Commission (the “SEC”). This Notice has no immediate effect on the listing of NeuBase’s common stock.

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NeuBase Therapeutics Reports Financial Results for Fiscal Year 2019

PITTSBURGH, Jan. 09, 2020 (GLOBE NEWSWIRE) -- NeuBase Therapeutics, Inc. (Nasdaq: NBSE) (“NeuBase” or the “Company”), a biotechnology company developing next-generation antisense therapies using its scalable PATrOL™ platform to address genetic diseases, today reported its financial results for the fiscal year ended September 30, 2019.

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NeuBase Therapeutics Wins The Scientist’s Top 10 Innovations of 2019 Award for its Janus Base Technology

PITTSBURGH, Dec. 02, 2019 (GLOBE NEWSWIRE) -- NeuBase Therapeutics, Inc. (Nasdaq: NBSE) (“NeuBase” or the “Company”), a biotechnology company developing next-generation antisense therapies to address genetic diseases, today announced that one of its core technologies, the Janus Base, has been recognized by The Scientist as a Top 10 Innovation of 2019. The bi-specific Janus nucleobases uniquely allow NeuBase to develop short oligonucleotide drugs targeting rare genetic diseases that can reach and bind double helixes in the human genome and RNA secondary structures. The technology’s inventor and NeuBase’s chief scientific officer, Danith Ly, Ph.D., developed the Janus Base in his lab at Carnegie Mellon University.

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NeuBase Therapeutics to Present at Upcoming Investor Conferences in December

Evercore ISI HealthCONx Conference panel discussion at 12:30 p.m. ET on December 3rd

Piper Jaffray 31st Annual Healthcare Conference presentation at 8:30 a.m. ET on December 4th

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