Working Group Talk "RNA-based therapies"

Introduction

Nucleotide and in particular RNA-therapeutics are of increasing importance. With Inclisiran (PMID: 27959715), we have the first small interfering RNA (siRNA) therapeutic for the blocking of PCSK9 translation in clinical practice for the lowing of LDL-cholesterol. More recently, CRISPR Cas9 encoding RNA with concomitant single guide RNA targeting misfolded transthyretin (TTR) has demonstrated clinical efficacy in patients with TTR amyloidosis (PMID: 34215024). Numerous RNA therapeutics will follow (PMID: 36329321; PMID: 33816449), riding also on the clinical experience gained from modRNA development for COVID vaccination, because they are easy to develop and can be precisely targeted as highly specific therapies.

Educational goals

• Understand the basics of RNA-based advanced therapeutic medicinal products and their design and manufacturing
• Be updated on current and future clinical-grade RNA-based therapies
• Learn about potential applications and challenges for cardiovascular disease

Programme

Chairs 

Wolfram-Hubertus Zimmermann, DE

Monika Gladka, NL

Presentations

The limitless future of RNA therapeutics 

John P. Cooke, USA

Unlocking the promise of mRNA therapeutic

Kenneth Chien, SE

Questions and Answers

During the Q&A, participants will have the opportunity to interact with the chairs and speaker by asking their questions verbally using the “Raise hand” feature or in written form using the "Q&A" module. Chairs and speakers can answer directly in the module (publicly or privately) or discuss live during the Q&A.

Registration

The registration is now close.

The event recording will be accessible on demand a couple of weeks after the live event.