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NeuBase Therapeutics Announces Gene Editing Research Agreement with Global Healthcare Company

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NeuBase Therapeutics (Nasdaq: NBSE) has entered a research agreement with a Top 10 Global Healthcare company to evaluate its PATrOL™ technology for three monogenic genetic diseases. The PATrOL™ platform aims to address genetic disorders by using PNA-based gene editing, which is designed to increase fidelity and reduce immunogenicity compared to traditional methods like CRISPR-Cas9. The collaboration will explore novel drug candidates, providing the Healthcare Company with potential exclusive licensing opportunities based on the evaluation outcomes.

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  • Research collaboration with a major healthcare company expands NeuBase's market potential.
  • PATrOL™ technology offers a differentiated approach to gene editing, potentially increasing safety and efficacy.
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PITTSBURGH, Oct. 21, 2022 (GLOBE NEWSWIRE) -- NeuBase Therapeutics, Inc. (Nasdaq: NBSE) (“NeuBase” or the “Company”), a biotechnology platform company Drugging the Genome™ to address disease at the base level using a new class of precision genetic medicines, today announced a research agreement with a Top 10 Global Healthcare company (“Healthcare Company”) (based on 2021 revenues). For this research, the Healthcare Company will evaluate NeuBase’s PATrOL™ technology for three monogenic genetic diseases.

NeuBase’s PATrOL™ platform is a PNA-based technology designed to address disease at the root of causality to help patients with rare and common diseases by editing, upregulating, or downregulating gene function. PATrOL™ gene editing is differentiated in that it does not require bacterial enzymes (e.g., CRISPR-Cas9), which potentially increases fidelity and reduces immunogenicity to provide a safer solution to patients for in vivo gene editing.

“Our PATrOL technology enables us to create a series of peptide nucleic acids (PNAs) designed to target a genetic mutation and recruit the cell’s own high fidelity nucleic acid repair machinery to resolve the mutation,” stated Dietrich A. Stephan, Ph.D., Chief Executive Officer of NeuBase. “We look forward to working with our Healthcare Company partner to explore our novel gene editing technology for several genetic diseases.”

Under the terms of the agreement, NeuBase and the Healthcare Company will collaborate on the evaluation of drug candidates for three undisclosed indications. The Healthcare Company will have the exclusive opportunity, subject to certain terms and conditions, to license and develop the drug candidates created under this research evaluation agreement.

About NeuBase Therapeutics
NeuBase is accelerating the genetic revolution by developing a new class of precision genetic medicines that Drug the Genome™. The Company’s therapies are built on a proprietary platform called PATrOL™ that encompasses a novel peptide-nucleic acid antisense oligonucleobase technology combined with a novel delivery shuttle that overcomes many of the hurdles to selective mutation engagement, repeat dosing, and systemic delivery of genetic medicines. To learn more, visit www.neubasetherapeutics.com.

Use of Forward-Looking Statements
This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act. These forward-looking statements are distinguished by the use of words such as “will,” “would,” “anticipate,” “expect,” “believe,” “designed,” “plan,” or “intend,” the negative of these terms, and similar references to future periods. These forward-looking statements include, among others, those related to the potential and prospects of the Company’s proprietary PATrOL™ platform. These views involve risks and uncertainties that are difficult to predict and, accordingly, our actual results may differ materially from the results discussed in our forward- looking statements. Our forward-looking statements contained herein speak only as of the date of this press release. Factors or events that we cannot predict, including those risk factors contained in our filings with the U.S. Securities and Exchange Commission (the “SEC”), may cause our actual results to differ from those expressed in forward-looking statements. The Company may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements, and you should not place undue reliance on these forward-looking statements. Because such statements deal with future events and are based on the Company's current expectations, they are subject to various risks and uncertainties, and actual results, performance or achievements of the Company could differ materially from those described in or implied by the statements in this press release, including: the Company's plans to research, develop and commercialize any product candidates; the timing of initiation of any clinical trials; the risk that prior data will not be replicated in future studies; the timing of any investigational new drug application or new drug application; the clinical utility, potential benefits and market acceptance of any product candidates; the Company's commercialization, marketing and manufacturing capabilities and strategy; global health conditions, including the impact of COVID-19; the Company's ability to protect its intellectual property position; and the requirement for additional capital to continue to advance these product candidates, which may not be available on favorable terms or at all, as well as those risk factors contained in our filings with the SEC. Except as otherwise required by law, the Company disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date hereof, whether as a result of new information, future events or circumstances or otherwise.

NeuBase Investor Contact:NeuBase Media Contact:
Dan FerryJessica Yingling, Ph.D.
Managing DirectorPresident
LifeSci Advisors, LLCLittle Dog Communications Inc.
daniel@lifesciadvisors.comjessica@litldog.com
OP: (617) 430-7576OP: (858) 344-8091

FAQ

What is the recent partnership announced by NeuBase Therapeutics (NBSE)?

NeuBase Therapeutics announced a research agreement with a Top 10 Global Healthcare company to evaluate its PATrOL™ technology for three monogenic genetic diseases.

What does the PATrOL™ technology from NeuBase Therapeutics do?

PATrOL™ technology is a PNA-based gene editing platform designed to address genetic disorders by editing, upregulating, or downregulating gene function without the use of bacterial enzymes.

What are the implications of the research agreement for NeuBase (NBSE)?

The agreement could lead to exclusive licensing opportunities for NeuBase if the drug candidates developed prove successful.

What type of diseases will NeuBase's PATrOL™ technology address?

The technology will be evaluated for three undisclosed monogenic genetic diseases.

NeuBase Therapeutics, Inc.

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