Restoring the Genome to its Organic State

Neurological Disorders
Muscular Dystrophies
Ocular Diseases

A revolutionary technology that tackles disease at its root cause with scalpel-like precision

We are developing an entirely new class of post-precision medicine with remarkable characteristics and near limitless scalability: ultra-precision.  Our proprietary PATrOL™ platform can shape the function of genes with a novel exactness and individualized approach that we believe will increase treatment options and reduce suffering for the hundreds of millions of people impacted by genetic diseases.


The advantages of the NeuBase PATrOL™ platform enable the company to cohesively develop therapies for a wide range of diseases including single-gene disorders, cancers and common diseases

NeuBase Therapeutics’ platform has evolved precision medicine into an entirely new category of restorative genomic medicine: Ultra-Precision. The PATrOL™ platform can output compounds that have the potential to restore the 6 billion letter human genome to its natural function with unprecedented accuracy, efficiency and near limitless scalability - without compromise - shepherding in an entirely new class of therapeutics that is poised to transform the landscape of precision medicine. Our technology has consistently delivered consequential preclinical results against initial rare disease and cancer targets, leveraging a scalable and proprietary approach that temporarily engages with the genetic code to address the root cause of individual disease and restore genes to their natural, proper state. This revolutionary platform offers a spectrum of applications unmatched by any other technologies: it can turn genes on, turn genes off and restore gene function all under one near infinite umbrella. We call this new category of restorative medicine ultra-precision - a near limitless solution that is more specific, more potent, more tolerable and more scalable than other biotechnologies on the horizon. Where precision medicine opened the door for hope, NeuBase’s ultra-precision has the potential to change the world by delivering real solutions.

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The NeuBase technology has several advantages over other genetic medicines


Scalable & Modular Development

Pre-organized rigid synthetic scaffold increases precision of engagement with target and toolkit of engineered nucleobases allow drugs to be quickly snapped together for many targets


Targeting Structures

Higher specificity for mutant over wild type genes, allowing for fewer off-target effects and more effective deactivation of disease-causing genes at the DNA and RNA levels


Broad Organ Distribution

Modular delivery technologies enable targeting a variety of organ systems


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 mutation positive

NeuBase Therapeutics Reports Financial Results for the First Quarter of Fiscal Year 2021

Recently demonstrated single-dose intravenous administration of a PATrOL™-enabled compound resolves the causal genetic defect in myotonic dystrophy type 1 (DM1) in transgenic animals; Company on course to move one program into clinical development in CY2022

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NeuBase Therapeutics Announces Acquisition of Gene Modulating Technology from Vera Therapeutics

Acquisition includes intellectual property for peptide nucleic acid genetic medicine portfolio that has demonstrated in vivo activity in several disease indications

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NeuBase Therapeutics to Present at the H.C. Wainwright Virtual BioConnect Conference

PITTSBURGH, Jan. 11, 2021 (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 during the H.C. Wainwright Virtual BioConnect Conference, which is being held from January 11 - 14, 2021. A webcast of the on-demand presentation will be available beginning today at 6:00 a.m. Eastern Time and archived on the NeuBase website,  

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NeuBase Therapeutics Reports Business Update and Financial Results for Fiscal Year 2020

Data presented throughout 2020 have validated the potential of the PATrOL™ platform to develop highly targeted therapies that increase, decrease or change causal protein function

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Covid-19 Update

Read our statement related to operations during COVID-19