Rare genetic diseases collectively affect hundreds of millions of people worldwide

Neurological Disorders
Muscular Dystrophies
Ocular Diseases
Oncology

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.

Technology

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

Sd

Scalable & Modular Development

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

Ts

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

BrOD

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

Ni

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 Announces Addition of Peter Nielsen, Ph.D., Inventor of Peptide Nucleic Acid Technology, to Scientific Advisory Board

PITTSBURGH, Oct. 13, 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, announced the addition of Peter Nielsen, Ph.D. to its scientific advisory board. Dr. Nielsen, the primary inventor of peptide nucleic acid (PNA) technology, brings extensive experience in genetic medicine to NeuBase as the Company optimizes its PATrOL™ therapies and moves them towards the clinic.

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NeuBase Therapeutics to Present at the Chardan Virtual 4th Annual Genetic Medicines Conference on Monday, October 5th

PITTSBURGH, Sept. 28, 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, announced today that Dietrich A. Stephan, Ph.D., Chief Executive Officer of NeuBase, will present a corporate overview at the Chardan Virtual 4TH Annual Genetic Medicines Conference being held on October 5 - 6.

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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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