JUL 24, 2024 11:20 AM EDT

Exploring the Development & Optimization of mRNA Delivery Systems to Advance mRNA-based medicines

Speaker

Abstract

The rapid advancements in mRNA-based therapeutics have ushered in a new era of medicine, promising revolutionary treatments for a wide range of diseases. However, despite the tremendous potential, several bottlenecks hinder the full realization of these therapies. This presentation will delve into the current challenges in mRNA therapeutics development, focusing on tissue distribution and the stability issues associated with traditional lipid nanoparticle (LNP) delivery systems. By leveraging novel mRNA delivery technologies and strong knowledge and expertise in mRNA medicines, we aim to enhance the stability, affordability, and efficacy of mRNA-based medicines and address unmet medical needs.

One of the primary hurdles in mRNA therapeutics is achieving targeted tissue distribution. The ability to deliver mRNA precisely to the desired cells and tissues is critical for therapeutic efficacy and minimizing off-target effects. Traditional LNPs, while effective in certain contexts, often face challenges in stability and specificity, limiting their widespread application. To address these issues, Zipcode Bio is pioneering the development of next-generation mRNA delivery systems. Our approach involves the use of novel nanoparticle formulations designed to enhance stability and cellular uptake. By modifying the composition and properties of these nanoparticles, we aim to improve their interaction with biological membranes and evade immune detection, thereby increasing their delivery efficiency to target tissues.

Our efforts also extend to the selection of therapeutic programs and diseases that can best leverage the advantages of targeted delivery and mRNA expression. By focusing on diseases where precise control of protein expression can yield significant therapeutic benefits, we aim to maximize the impact of our mRNA-based interventions. One example is to tackle prevalent diseases where traditional treatment options are limited or inadequate.

In summary, the development and optimization of mRNA delivery systems are critical to advancing mRNA-based medicines. Through innovative nanoparticle technology and strategic therapeutic targeting, Zipcode Bio is at the forefront of overcoming the current bottlenecks in mRNA therapeutics. This keynote will provide an exploration of these approaches, highlighting the progress made and the future directions for this promising field.

Learning Objectives:

  • Understand the primary challenges in the development of mRNA-based therapeutics, including issues with tissue distribution and the stability of traditional lipid nanoparticle (LNP) delivery systems.
  • Explore innovative approaches in mRNA delivery technology, focusing on the development of next-generation nanoparticles designed to enhance stability and organ specificity
  • Identify the strategic selection of therapeutic programs and diseases that can benefit most from targeted mRNA delivery and precise protein expression to maximize therapeutic impact

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