NeuBase Therapeutics presents NHP repeat dosing data for Stealth Editors

NeuBase Therapeutics, 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 28th American Peptide Symposium, which is being held in Scottsdale, AZ, from June 24-29, 2023. Overview of Preclinical Studies and Results Presented at APS 2023: Stealth Editors are Non-Immunogenic in Non-Human Primates – The Company demonstrated that Stealth Editors do not elicit an innate nor acquired immune response in NHPs as measured by a cytokine panel: Cynomolgus NHPs were given two doses of an intravenous administration, on days 1 and 29: vehicle control; MC3 lipid nanoparticle control; 0.10 mg/kg Stealth Editor encapsulated in an LNP; and 0.16 mg/kg Stealth Editor co-encapsulated with a single-stranded oligonucleotide donor in an LNP; On days 0, 1 and 29, blood was drawn and serum was assessed for IFN-gamma, IL-2, IL-6, IL-8, IL10 and IL-1beta cytokine induction using a Meso Scale Discovery multi-spot assay; Normal cytokine ranges were identified in healthy untreated cynomolgus monkeys by MSD; and All absolute values of cytokines were within the historical control ranges. The conclusion from these data is that Stealth Editors do not elicit innate nor acquired immune responses in the encapsulated format in which they would be administered systemically. The Company believes this is likely to be an important differentiator of its technology when transitioned to in vivo gene editing.