Drug Discovery and RNA Target Validation Workshops

A number of aspects of drug discovery need to be reconsidered when shifting the paradigm from targeting protein to targeting RNA. These areas range from target selection to lead discovery and assays to support lead optimization as well as in vivo pharmacodynamics markers.

Showcasing the most up to date scientific advances and more, the 2^nd RNA-Targeted Drug Discovery Summit boasts an entire workshop day specifically tailored to enable attendees to navigate their way from early discovery towards robust translation into the clinic with RNA targeted small molecules.

Find the full workshop details in the official event guide.

Johan Potin, Founder and Former CEO of The RNA Medicines Company will be heading up the morning with a session on Empowering RNA Drug Target Validation: “An Essential Initial Step for Every Drug Discovery Effort”.

In this session, Potin will be covering a wide range of important topics and posing key questions, including:

• How do we guarantee that the RNA we are targeting is a high value drug target?
• As drug developers, how do we validate an RNA as a drug target? Translational medicine in the area of RNA Medicine is still in its infancy; there is a paucity of examples in literature that inspire confidence in the part RNA has in disease.
• What are the best tools to illuminate the part RNA plays in disease?
• Once we validate a specific RNA as a drug target, how do we produce the right drug-like tool molecules to make sure we direct our drug discovery effort with specificity? Drug discovery on Target is vital and remains challenging, especially with small molecules that have promiscuous natures.
• Mechanism of Action; are CRISPR and oligonucleotides appropriate control tools when you develop small molecules targeting RNA?

Margaret Porter Scott, VP at Biochemical & Cellular Pharmacology and John Moffat, Senior Scientist at Biochemical & Cellular Pharmacology at Genentech will lead a discussion-based workshop on Exploring Drug Discovery Approaches to RNA, covering:

• What mechanisms have the potential or are likely to work? The range of known approaches to affecting splicing, 5’ UTR interference to decrease transcription, binding to mRNA, and more, will be discussed.
• What are the drawbacks when transitioning from targeting protein to targeting RNA, and what mindsets need to change?
• What are the important technologies useful for drugging RNA? What is the role of structure-based design, computational methods, biophysics, cell-based assays and more?
• What are appropriate pharmacodynamic (PD) markers for RNA targeted therapy and what level of PD can we expect (partial or full PD responses)?

Join over 100 professionals to improve understanding, discuss research and learn how other researchers are tackling common challenges.

About PREDiCT

The hard reality of drug development is a rising rate of attrition and development costs.

Dedicated to exploring the latest advances in the application of preclinical models, the PREDiCT Event Series is helping to fight back and make inroads in the ‘war on attrition’ by showcasing candidate selection strategies advancing disease modifying therapies from bench to patient with more confidence.

Focused predominantly on immunogenic, fibrotic and oncogenic diseases, we connect expert model developers alongside end users and technology champions from biopharma, to define what’s possible for current and emerging in vitro/in vivo models.

Our disease and model specific events, reveal effective model characterization and selection strategies that are better enabling drug candidates to target patient responders, identify safety concerns and robustly translate candidates to clinic faster.

The core questions to be answered at every PREDiCT: Tumor Models meeting:

1. What new model and additive technology initiatives are helping biopharma leaders drive translational and early-stage decision making?
2. How do biopharma leaders balance advances in model complexity and relevance with the cost-per-data-point?
3. What are the key criteria the leading biotech and pharma use to assess, select and characterize the array of tumor models?

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