NeuBase Announces Positive Preclinical Data Supporting the Capabilities of Stealth Editors

• Data show that Stealth Editors achieve ex vivo gene editing using simple synthetic reagents to harness the cell's own high-fidelity editing machinery
• Data show that Stealth Editors do not elicit a cell-based immunity and promise a non-immunogenic in vivo solution
  
• Data show that Stealth Editors can be encapsulated via established lipid nanoparticle technologies promising a non-immunogenic delivery solution and a de-risked pathway to address therapeutic targets in the liver
  
• Data featured in an oral presentation at the American Society of Gene & Cell Therapy (“ASGCT”) 2023 Annual Meeting
  
• Company to present additional data showcasing gene editing capabilities of Stealth Editors throughout remainder of 2023, including initial non-human primate and mouse data
  
• Conference call and webcast scheduled for Monday, May 22, 2023, at 8:30 am ET
  

PITTSBURGH, May 22, 2023 (GLOBE NEWSWIRE) -- NeuBase Therapeutics, Inc. (Nasdaq: NBSE) (“NeuBase” or the “Company”), a biotechnology company developing Stealth Editors to perform in vivo gene editing without triggering the immune system, today announced a broad set of preclinical safety and efficiency data for its Stealth Editors development program that demonstrate the ability to achieve gene editing with a non-immunogenic system.

These preclinical data were featured in an oral presentation by Dr. Dani Stoltzfus, Vice President of Research at NeuBase, titled, “Nuclease-Free Gene Editing with Peptide Nucleic Acids: A New Class of In Vivo Gene Editors,” at the ASGCT 2023 Annual Meeting.

“We are excited to announce preclinical data from our Stealth Editors development program that were presented during the ASGCT 2023 Annual Meeting. These data support our novel in vivo gene editing approach that allows us to tag a locus in the genome that harbors a mutation with a simple synthetic compound and recruit the cell’s own machinery to repair the mutation. This high-fidelity non-immunogenic approach is expected to offer advantages compared to modified bacterial CRISPR/Cas enzymes for in vivo gene editing applications from a safety and durability of effect perspective,” stated Dietrich A. Stephan, Ph.D., Founder and Chief Executive Officer of NeuBase. “We continue to rapidly advance the development of our Stealth Editors platform, which we believe has the potential to generate best-in-class, next-generation therapies capable of addressing diverse types of high-value genetic mutations. Throughout the remainder of 2023, we expect to announce additional preclinical data from several ongoing and planned studies in non-human primates and mice.”