NeuBase Therapeutics Inc
announced a broad set of preclinical safety and efficiency data for its Stealth Editors development program, demonstrating the ability to achieve gene editing with a non-immunogenic system.
- The data showed a dose-dependent increase in the correction of the gene mutation based on the expression of the new functional fluorescent protein compared with various controls, highlighting a titratable increase in efficiency with which the Stealth Editors can engage the genome to harness the cell's machinery to correct the mutation.
- The results show that human peripheral blood mononuclear cells treated with a Stealth Editor did not impact cell viability. In addition, there was no cellular immune response for the Stealth Editor-treated cells compared with the control (PBS) across the five cytokines measured, in direct juxtaposition to the positive controls, which elicited a strong cytokine response.
- 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.
- The company plans to present additional data showcasing the gene-editing capabilities of Stealth Editors throughout the remainder of 2023, including initial non-human primate and mouse data.
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