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

Sa

Systemic Administration

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

Ni

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 Announces Appointment of Cancer Biologist and RNA Therapeutics Specialist Steven Dowdy, Ph.D., to Scientific Advisory Board

PITTSBURGH, Oct. 11, 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 the addition of Steven Dowdy, Ph.D., to its scientific advisory board (SAB). Dr. Dowdy joins preeminent scientists George Church, Ph.D., and Samuel Broder, M.D., on the NeuBase SAB.

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NeuBase Therapeutics to Present at the Chardan 3rd Annual Genetic Medicines Conference

PITTSBURGH, Sept. 26, 2019 (GLOBE NEWSWIRE) -- NeuBase Therapeutics, Inc. (NASDAQ: NBSE) (“NeuBase” or the “Company”), a biotechnology company developing next-generation antisense therapies to address genetic diseases, announced today that Dietrich Stephan, Chief Executive Officer, will present a corporate overview and business update at the Chardan 3rd Annual Genetic Medicines Conference, being held in New York City.

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NeuBase Therapeutics Hosting Key Opinion Leader Meeting on Huntington's Disease and PATrOL™-Enabled Therapy on September 16th

PITTSBURGH, Sept. 09, 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 it will host a Key Opinion Leader (KOL) meeting on Huntington's disease and NeuBase’s peptide-nucleic acid (PNA) antisense oligonucleotide (PATrOL™) platform on Monday, September 16, 2019 in New York City.

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NeuBase Therapeutics Reports Financial Results for the Fiscal Third Quarter of 2019

Completed two financings in July 2019 totaling approximately $14 million in gross proceeds with participation from institutional investors including Greenlight Capital             

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