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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


Broad Organ Distribution

Ability to cross the blood-brain barrier and disseminate broadly, allowing for systemic administration in neurological indications and reaching organ systems previously not accessible by traditional antisense approaches


No Immune Response

Therapies 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 Myotonic Dystrophy Foundation 2020 Virtual Conference

PITTSBURGH, Sept. 17, 2020 (GLOBE NEWSWIRE) -- NeuBase Therapeutics, Inc. (NASDAQ: NBSE) (“NeuBase” or the “Company”), a biotechnology company accelerating the genetic revolution using a new class of synthetic medicines, today announced that Dietrich A. Stephan, Ph.D., chief executive officer, is scheduled to present at the Myotonic Dystrophy Foundation 2020 Virtual Conference on September 25 between 4:00 p.m. and 4:45 p.m. ET.

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NeuBase Therapeutics to Present at Upcoming Investor Conferences in September 2020

22nd Annual H.C. Wainwright Global Investor Conference presentation at 1:30 p.m. ET on September 16TH

Oppenheimer Fall Healthcare LifeSciences & Medtech Summit Fireside Chat at 10:00 a.m. ET on September 23 RD

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NeuBase Therapeutic’s CEO, Dietrich A. Stephan, Ph.D., to Present at Tribe Public’s Presentation and Q&A Webinar Event on August 26, 2020

SAN FRANCISCO, CA / ACCESSWIRE / August 24, 2020 / Tribe Public announced today that Dietrich Stephan, Chief Executive Officer of NeuBase Therapeutics, Inc. (NASDAQ:NBSE), a biotechnology company developing next-generation antisense oligonucleotide (ASO) therapies using its scalable PATrOL™ platform to address genetic diseases, will present at Tribe Public's Presentation and Q&A Webinar Event at 8 am pacific/11 am eastern on Wednesday, August 26th, 2020. During this complimentary, 30-minute event, Dr. Stephan will introduce the NeuBase's next-generation gene silencing technology and discuss the company's progress with treatment candidates in Huntington's Disease (HD) and Myotonic Dystrophy (DM1). A question and answer session will follow the presentation. To register to join the complimentary event, please visit the Tribe Public LLC website:, or send a message to Tribe's management at to request your seat for this limited capacity Zoom-based event.

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

Strong Pharmacokinetic and Pharmacodynamic Data Presented in March Validate Platform and Position Company for Scalable Output of Synthetic Precision Genetic Medicines

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