Industry Session
Syed Reza, MD, PhD (he/him/his)
Scientific and Sales Consultant
NOF AMERICA Corporation
White Plains, New York, United States
There is growing interest in use of ionizable lipids for delivery in of gene editing or for enzyme replacement therapy (ERT). Yet, many lipids currently available were originally developed for vaccines and the lipids selected for these applications are the result of a selection bias for immunogenicity. This poses certain challenges when attempting to adapt the vaccine LNPs for mRNA therapy due to significant dose limiting toxicity after repeated and systemic exposure. We here present our work on the development of biodegradable ionizable lipids (COATSOME® SS Series) that demonstrate improved tolerability, reduced organ toxicity and immunotoxicity while at the same time enabling high levels of mRNA expression. The COATSOME® SS Series exhibit exceptional stability in the circulating state and only break down when triggered by specific stimuli present in the cytosol. This presentation will also present our efforts to engineer LNPs specifically designed for gene therapy applications, for infectious disease vaccines and cancer immunotherapy applications.