Accelerating the Genetic Revolution

Neurological Disorders
Muscular Dystrophies
Ocular Diseases
Oncology

A new class of synthetic medicines to address the root cause of genetic diseases

We are at a fundamental inflection point in the history of the pharmaceutical industry where, as a result of the Human Genome Project, we now have the ability to leverage nature’s own information encoding system (the genetic code) to “digitally” encode our drugs so as to target a precise gene that is malfunctioning and neutralize the disease process at its root cause.

As every human disease has genetic drivers, our vision is to dramatically reduce the suffering and death attributable to innumerable diseases across the globe with our first-in-class technology platform.

The therapies we are creating are analogous to heat-seeking missiles in that they are highly specific to only the target gene and promise easy to take and well tolerated medicines.

Technology

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 is first addressing Huntington’s disease and myotonic dystrophy

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The NeuBase technology has several advantages over other precision genetic medicines

Sd

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

Ts

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

BrOD

Broad Organ Distribution

Modular delivery technologies enable targeting a variety of organ systems

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 mutation positive

NeuBase Therapeutics Demonstrates Functional Rescue in Myotonic Dystrophy Type 1 Model and Allele-Selective Inhibition of KRAS Mutations to Inhibit Tumor Growth

PITTSBURGH, June 08, 2021 (GLOBE NEWSWIRE) -- NeuBase Therapeutics, Inc. (Nasdaq: NBSE) ("NeuBase" or the "Company"), a biotechnology company accelerating the genetic revolution using a new class of precision genetic medicines, today announced positive new data and program updates for its development pipeline of PATrOL-enabled genetic medicines.

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NeuBase Therapeutics Announces Speakers for Virtual R&D Day Taking Place on June 8th

PITTSBURGH, June 02, 2021 (GLOBE NEWSWIRE) -- NeuBase Therapeutics, Inc. (Nasdaq: NBSE) ("NeuBase" or the "Company"), a biotechnology company accelerating the genetic revolution using a new class of precision genetic medicines, today announced that it will host a virtual R&D day for investors and analysts on Tuesday, June 8, 2021 at 12:30 p.m. EDT.

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NeuBase Therapeutics Appoints Dr. Kia Motesharei Chief Business and Strategy Officer

PITTSBURGH, May 25, 2021 (GLOBE NEWSWIRE) -- NeuBase Therapeutics, Inc. (Nasdaq: NBSE) ("NeuBase" or the "Company"), a biotechnology company accelerating the genetic revolution using a new class of precision genetic medicines, today announced the appointment of Kia Motesharei, Ph.D., as Chief Business and Strategy Officer, effective May 24, 2021. Dr. Motesharei has more than 20 years of experience in business development, licensing and transactions, alliance management and strategy in the biotechnology and pharmaceutical industry.

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NeuBase Therapeutics Appoints Dr. Sandra Rojas-Caro Chief Medical Officer

PITTSBURGH, May 20, 2021 (GLOBE NEWSWIRE) -- NeuBase Therapeutics, Inc. (Nasdaq: NBSE) ("NeuBase" or the "Company"), a biotechnology company accelerating the genetic revolution using a new class of precision genetic medicines, today announced the appointment of Sandra Rojas-Caro, M.D., as Chief Medical Officer, effective May 24, 2021. With more than 20 years of drug development experience across all phases of drug development, Dr. Rojas-Caro will oversee the preclinical and clinical development, medical, and regulatory strategy of NeuBase’s pipeline to address disease at its base cause.

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