NeuBase Presents Non-Human Primate Data Illustrating Stealth Editors are Non-Immunogenic, Opening the Door to Redosing

• New preclinical data validates that Stealth Editors are non-immunogenic, as featured in a poster presentation at the 28th American Peptide Symposium

• First non-human primate (NHP) study to successfully redose a gene editor without an immune response

• Ex vivo data show Stealth Editors are titratable, and editing efficiency is in range for clinical benefit in various conditions

• Company expects to present additional data showcasing gene editing capabilities of Stealth Editors throughout remainder of 2023
  

PITTSBURGH, June 29, 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 data, including non-human primate (“NHP”) repeat dosing 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 a poster presentation by Dr. Dani Stoltzfus, Vice President of Research at NeuBase, titled, “Stealth Editing With Peptide Nucleic Acids: A New Class of In Vivo Gene Editors,” at the 28^th American Peptide Symposium (APS), which is being held in Scottsdale, AZ, from June 24-29, 2023.

“We are pleased to report that no innate nor acquired immune responses in NHPs were observed after repeat dosing of our Stealth Editor, as measured across a panel of key cytokines after each dose. We believe this is the first NHP study that has successfully redosed a gene editor without observing an immune response for either dose,” stated Dietrich Stephan, Ph.D., Founder and Chief Executive Officer of NeuBase.

“Patient immune reactions against gene therapies are well documented and can result in serious adverse events, including death, along with a lack of pharmacology due to neutralizing antibodies. We are beginning to see reports of immune responses after delivery of gene editing solutions, possibly due to viral vector-based delivery or the expression of bacterial nucleases in vivo. For these reasons, we are on a mission to develop the next generation of editors that are non-immunogenic,” continued Dr. Stephan. “Our nuclease-free synthetic editors resemble oligonucleotides, but can uniquely engage the double-stranded human genome and harness the cell’s own DNA repair machinery to elicit editing. We believe this lack of immunogenicity also enables repeat dosing, should tissue editing efficiency thresholds be high or if durability wanes with time. These latest data support a differentiated pipeline of in vivo editing therapeutic programs, which we expect to announce later this year, as well as the pursuit of additional partnerships with pharmaceutical and agricultural partners in areas of mutual interest,” concluded Dr. Stephan.