Presentation Overview
Title:Design of NOF’s Ionizable Lipids Achieving Low Toxicity and Reduced Immunogenicity for Non-Viral RNA Therapeutics
Speaker:
Ryosuke Fukuda, Research Scientist at NOF CORPORATION
Ryosuke Fukuda, Research Scientist at NOF CORPORATION
Session:
Innovation Stage – Manufacturing & Supply Chain Track
Innovation Stage – Manufacturing & Supply Chain Track
Abstract:
Since the COVID‑19 pandemic, there has been growing interest in using ionizable lipids for gene delivery in gene editing and enzyme replacement therapy. However, many of the ionizable lipids currently available were originally developed for vaccines and were selected based on immunogenicity-related properties. Consequently, repurposing vaccine-derived LNPs for repeated systemic dosing in mRNA therapeutics is challenging because cumulative exposure can produce dose-limiting toxicities. In this study, we report on the rational design and characterization of biodegradable ionizable lipids that provide improved tolerability, while maintaining robust in vivo mRNA expression. We further provide metabolism and excretion data to support their favorable safety and clearance profiles. This presentation will cover hepatic gene delivery using LNPs formulated with NOF’s proprietary ionizable lipids, spleen‑targeted delivery by formulation optimization, and potential applications for infectious disease vaccines and repeated‑dose mRNA therapeutics.
Since the COVID‑19 pandemic, there has been growing interest in using ionizable lipids for gene delivery in gene editing and enzyme replacement therapy. However, many of the ionizable lipids currently available were originally developed for vaccines and were selected based on immunogenicity-related properties. Consequently, repurposing vaccine-derived LNPs for repeated systemic dosing in mRNA therapeutics is challenging because cumulative exposure can produce dose-limiting toxicities. In this study, we report on the rational design and characterization of biodegradable ionizable lipids that provide improved tolerability, while maintaining robust in vivo mRNA expression. We further provide metabolism and excretion data to support their favorable safety and clearance profiles. This presentation will cover hepatic gene delivery using LNPs formulated with NOF’s proprietary ionizable lipids, spleen‑targeted delivery by formulation optimization, and potential applications for infectious disease vaccines and repeated‑dose mRNA therapeutics.
Inquiries
NOF CORPORATION extends its gratitude to all who visited our booth and attended the presentation.
We remain committed to providing innovative DDS solutions that address the complex challenges of modern drug development.
For those who were unable to attend or would like to learn more about our presented technologies, please contact us via our Contact Form.
* Please select “Life Science” for the Business division field.