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 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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NeuBase Therapeutics to Ring the NASDAQ Closing Bell Today

PITTSBURGH, Aug. 07, 2019 (GLOBE NEWSWIRE) -- NeuBase Therapeutics, Inc. (Nasdaq: NBSE) (“NeuBase” and the “Company”), a biotechnology company developing next generation antisense therapies to address genetic diseases, announced today that its chairman and chief executive officer, Dietrich A. Stephan, Ph.D., will be joined by other members of the management team to ring the NASDAQ closing bell today, August 7, 2019, to celebrate the Company’s recent listing on the NASDAQ.

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NeuBase Therapeutics Announces Addition of Industry Pioneer Dr. Samuel Broder and World-Renowned Geneticist Dr. George Church to Scientific Advisory Board

PITTSBURGH, July 30, 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 Samuel Broder, M.D, Ph.D. and George Church, Ph.D. to its scientific advisory board (SAB), bringing additional expertise in genetics, genomics, and oncology to a team already recognized for leadership in neurology, genomics and therapeutic development. The SAB will work closely with the NeuBase management team as the Company advances its next-generation gene regulating therapies through development, first in genetic diseases such as the repeat expansion disorders Huntington’s disease and myotonic dystrophy, as well as oncology, and then more broadly.

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NeuBase Therapeutics Appoints Danith Ly, Ph.D. as Chief Scientific Officer

PITTSBURGH, July 24, 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 appointment of Danith Ly, Ph.D., as chief scientific officer. Dr. Ly is the primary inventor of NeuBase’s peptide nucleic acid (PNA) antisense oligonucleotide (PATrOL™) platform technology, which the Company is leveraging to develop a pipeline of gene modifying therapies. Dr. Ly joins NeuBase as the Company is advancing its first PATrOL-enabled therapies through preclinical development and expanding its pipeline across rare genetic diseases as well as additional genetically-driven disorders. In addition to his role at NeuBase, Dr. Ly will maintain his faculty appointment at Carnegie Mellon University’s Mellon College of Science.

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