The Problem:
The field of nucleic acid therapeutics depends critically on effective delivery systems to protect and transport the therapeutic into the target cells. Ionizable lipids are the key components in the state-of-the-art delivery systems for RNA. However, current major limitations include poor biodegradability, low thermostability, limited functionalities, and labor intensive and high-cost synthesis requirements for manufacturing. As the applications of RNA therapies expand, there is a growing need for developing scalable and customizable delivery systems that offer improved stability and cellular uptake while minimizing adverse effects, thus broadening the potential applications and efficacy of RNA therapeutics.
The Solution:
Researchers at The University of Alabama and Imperial College London developed a library of novel ionizable and cationic lipids derived from glycerol for use as vectors for nucleic acid delivery. These scalable, versatile lipids incorporate both symmetric and asymmetric tail configurations into the design which, in combination with diverse chemical features, enhance vector stability and offer multiple customizable functionalities. The synthesis process for these lipids has been streamlined presenting an opportunity for cost-effective, scalable manufacturing beyond that which is possible with current industry standard while remaining in accordance with Good Manufacturing Practices.
Benefits: