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 Added to Russell 3000® and Russell Microcap® Indexes

PITTSBURGH, June 29, 2020 (GLOBE NEWSWIRE) -- NeuBase Therapeutics, Inc. (Nasdaq: NBSE) (“NeuBase” or the “Company”), a biotechnology company developing next-generation antisense oligonucleotide (ASO) therapies using its scalable PATrOL™ platform to address genetic diseases, announced today that it has been added to the Russell 3000® and Russell Microcap® Indexes at the conclusion of the 2020 Russell indexes annual reconstitution, effective after the US market opens today.

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NeuBase Therapeutics Reports Financial Results for the Second Fiscal Quarter of 2020

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Announced positive preclinical data that validates the key advantages of the proprietary NeuBase peptide-nucleic acid antisense oligonucleotide (PATrOL™) platform
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Completed a public offering with net proceeds of approximately $33.3 million in April 2020, strengthening the Company’s balance sheet to support the ongoing development of its product candidates and expand its pipeline

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NeuBase Therapeutics to Present at Upcoming Investor Conferences in May 2020

6th Annual SunTrust Robinson Humphrey Life Sciences Summit Virtual Series Fireside Chat
at 2:30 p.m. ET on May 15TH

RBC Capital Markets 2020 Global Healthcare Conference presentation
at 1:20 p.m. ET on May 20TH

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NeuBase Therapeutics Announces Closing of Public Offering and Full Exercise of Underwriters’ Option to Purchase Additional Shares

PITTSBURGH, April 30, 2020 (GLOBE NEWSWIRE) -- NeuBase Therapeutics, Inc. (Nasdaq: NBSE), a preclinical-stage biotechnology company focused on developing next generation therapies to treat rare genetic diseases caused by mutant genes, announced today the closing of its previously announced underwritten public offering of 6,037,500 shares of its common stock (inclusive of 787,500 shares that were sold pursuant to the underwriters’ full exercise of their option to purchase additional shares of NeuBase’s common stock), at a price to the public of $6.00 per share. The net proceeds to NeuBase from the offering are expected to be approximately $33.3 million, after deducting the underwriting discounts and commissions and other estimated offering expenses payable by NeuBase. NeuBase intends to use the net proceeds from this offering for working capital and general corporate purposes and to advance the development of its product candidates and expand its pipeline.

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