Discovery of small molecules that target RNA. NMR is the method of choice.

Webinar Overview

Fragment-based screening (FBS) by NMR has become a central tool in the pharmaceutical industry to identify new ligands that bind to selected biological targets. NMR is also used to validate drug candidates obtained through classical high throughput screening (HTS) methods. In HTS, thousands to millions of samples are tested for biological activity at the model organism, cellular, pathway or molecular level. NMR is particularly suited to these tasks since the screening process takes places on a sample in solution under conditions where both the target and the ligands are very close to their physiological state. Typically, both 1H and 19F nuclei are used for FBS by NMR. Nowadays, most pharmaceutical groups have incorporated NMR screening strategies into their drug discovery and drug design programs. While the usual targets of these programs are classically enzymes or protein receptors, noncoding RNAs, which have proven roles in diseases and in the regulation of biochemical pathways, are increasingly becoming a therapeutic target of choice.

By applying machine learning in combination with fragment-based screening using NMR, it is possible to revolutionize the lead-finding process. In addition, NMR allows for the rapid validation of the secondary structure of new RNA targets. Fragment screening validates the presence of small molecule binding sites. This webinar given by Marcel Blommers, Chief Scientific Officer at Saverna Therapeutics, and moderated by Martial Piotto, Pharma product manager of Biologics solutions at Bruker BioSpin, demonstrates how to use NMR spectroscopy to identify novel RNA drug targets and inhibitors of those targets. You will learn how FBS by NMR and machine learning can be used jointly to discover new drugs that interact with RNA targets in the pharma and biopharma industry. NMR not only plays a unique role in hit finding, but also in characterizing RNA and RNA-ligand interactions. This information can guide hit-to-lead optimization. The complexity of dynamic RNA molecules in identifying new targets is discussed using various examples from Saverna’s portfolio. The implementation of various methods in Saverna’s RNA targeting NMR platform aims to perform early drug discovery with unprecedented speed without sacrificing quality.

Wednesday, February 08, 2023
5:00 PM CET

Key Learning Points

  • NMR is one of the best methods for fragment-based screening (FBS)
  • FBS by NMR can be applied to both protein and RNA targets
  • The dynamic nature of RNA makes NMR the method of choice for structural characterization and hit finding
  • Discover how NMR and machine learning join forces to identify new ligands of RNA targets

Who should attend?

This webinar will be of interest to anyone interested in improving their lead discovery capabilities. This includes pharmaceutical companies, contract research organizations and academia, with and without previous experience in FBS and structural biology. Additionally, any company which is part of this ecosystem, like fragment library vendors and computational chemistry software companies.


Dr. Marcel Blommers

Chief Scientific Officer at Saverna Therapeutics

Marcel Blommers is an expert in drug discovery of RNA targets. After completing his PhD in structure determination of nucleic acids (Prof. Hilbers) and a postdoc (Prof. Kaptein), he joined Novartis in 1992. He co-founded Saverna Therapeutics in 2017 to establish a pipeline of small molecules that target RNA.

Dr. Martial Piotto

Pharma product manager of Biologics solutions

Martial Piotto obtained his Ph.D. in NMR spectroscopy at the University of Purdue, USA (Pr. D. Gorenstein). He then joined Bruker France as head of NMR application where he worked on various aspects of NMR spectroscopy, notably the development of new pulse sequences and HRMAS technology. Over the years, he grew an interest in applications of NMR in the pharmaceutical and medical fields. His work currently focusses on the characterization of Biologics (mAb, vaccines and therapeutic oligonucleotides) by combining NMR and multivariate statistical tools.


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