SONJA MERKAS
Livinovea, Founder, Basel, Switzerland

Abstract

This article explores the dynamic field of nucleic acid therapeutics, highlighting its evolution from targeting specific genetic disorders to addressing multifactorial diseases, incorporating insights from industry experts collected through interviews. It refers to the developmental trajectory of these therapies, including key scientific breakthroughs, advancements in drug delivery systems like GalNAc-siRNA conjugates, and the ongoing challenges in manufacturing scalability and regulatory compliance. Furthermore, the article discusses the integration of digital technologies that enhance the efficiency and effectiveness of research and development processes.

Introduction
In the ever-evolving landscape of medicine, nucleic acid therapeutics stand at the forefront of innovation. These groundbreaking treatments hold the promise of targeting diseases at their genetic roots, offering gene inhibition, addition, replacement or editing. They offer advantages in terms of target specificity aiming at proteins that were once ‘undruggable’, broad therapeutic potential through multifactorial targets, and potential for personalised medicine based on the genetic blueprint of a disease. Besides addressing protein-coding RNAs, the Encyclopedia of DNA Elements (ENCODE) project has shown that a significant part of the human genome encodes for non-protein coding RNAs (1). Among those, long noncoding RNAs are of particular interest from a clinical viewpoint (2).

This article explores the multifaceted journey of short-coding, synthetic RNAs, from their discovery to regulatory challenges, manufacturing advancements and challenges, and opportunities for digitalisation and automatisation. These molecules come with ongoing challenges to survive on their way to the inside of cells due to their size and physic ...