Nucleic Acid Therapeutics Toolbox for Modifications and Cell Delivery (GalNAc case study with MiNA Therapeutics)
Abstract
Oligonucleotide-based therapies are now a validated technology and in recent years the number of FDA-approved oligonucleotide-based drugs has rapidly expanded, with many more drugs currently under development. However, oligonucleotides’ stability, target affinity and cell delivery remain challenging and prevent their widespread clinical use. In this talk, we will provide an overview of the different chemical modifications used in designing nucleic acid-based drugs and how they can be utilized to improve the potency, duration, and safety of those therapies. The focus will be on cell delivery and the use of GalNAc conjugates, and we will discuss some of the most recent technologies. Moreover, we will have a look at how selecting the most appropriate solid support can ensure the manufacturing of high-quality oligonucleotide-based drugs on a large scale and at optimized costs.
Learning Points:
Evolution of delivery technologies and chemistry patterndesign for RNA therapeutics including antisense and siRNA
How to overcome challenges when synthesizing complex oligonucleotides for therapeutics?
What lessons you could learn from the leading RNA therapeutics companies to accelerate your own drug discovery program?
How LGC Biosearch Technologies have partnered with a leading saRNA therapeutics company to customize their cell delivery strategy using GalNAc?
Valerie started her career in the pharmaceutical industry specializing in quality assurance forGMP drug manufacturing. She later moved on to strategic roles in the healthcare industry. Currently she is managing the product portfolio of nucleic acid chemistry in LGC Biosearch Technologies. Valérie holds both a MSc and BSc in Biomedical Sciences from Bern and Fribourg Universities respectively, and also a Ph.D. in Medical Neuroscience from the Charité University Hospital.
Shireen started with LGC Biosearch Technologies as a chemist in 2016. Throughout her years being a chemist, her role has been primarily lab based as part of the production team, working on the synthesis of LGC’s catalogue products at both Bellshill and Petaluma sites in UK and US respectively. She is currently the product manager for nucleic acid chemistry portfolio. Shireen graduated from University of Strathclyde, Glasgow with BSc in Chemistry specializing on Drug Discovery.