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

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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 Wins The Scientist’s Top 10 Innovations of 2019 Award for its Janus Base Technology

PITTSBURGH, Dec. 02, 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 one of its core technologies, the Janus Base, has been recognized by The Scientist as a Top 10 Innovation of 2019. The bi-specific Janus nucleobases uniquely allow NeuBase to develop short oligonucleotide drugs targeting rare genetic diseases that can reach and bind double helixes in the human genome and RNA secondary structures. The technology’s inventor and NeuBase’s chief scientific officer, Danith Ly, Ph.D., developed the Janus Base in his lab at Carnegie Mellon University.

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NeuBase Therapeutics to Present at Upcoming Investor Conferences in December

Evercore ISI HealthCONx Conference panel discussion at 12:30 p.m. ET on December 3rd

Piper Jaffray 31st Annual Healthcare Conference presentation at 8:30 a.m. ET on December 4th

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NeuBase Therapeutics to Present at the Stifel 2019 Healthcare Conference

PITTSBURGH, Nov. 12, 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 of NeuBase, will present a corporate overview and business update at the Stifel 2019 Healthcare Conference, which is being held from November 19th to 20th in New York City.

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NeuBase Therapeutics Announces Issuance of Foundational Patent Covering the Key Building Blocks of PATrOL™ Enabled Therapies

PITTSBURGH, Oct. 17, 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 the U.S. Patent and Trademark Office has issued U.S. Patent No. 10,370,415 covering the Company’s proprietary DNA and RNA binding technology. This foundational patent enables PATrOL™-based therapies to target the secondary structures of DNA and RNA.

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