Under the Spotlight: Genentech & The RNA Medicines Company Workshops on Drug Discovery Approaches to RNA & Empowering RNA Drug Target Validation
Shifting the paradigm from targeting protein to targeting RNA requires rethinking several aspects of drug discovery including target selection, lead discovery, assays to support lead optimization as well as in vivo pharmacodynamics markers.
Capturing the latest scientific advances and much more, the 2nd RNA- Targeted Drug Discovery Summit has a whole workshop day dedicated to enabling you to navigate your way from early discovery towards robust translation into the clinic with RNA targeted small molecules.
Check out the full workshop details in the official event guide.
Johan Potin, Founder and Former CEO, The RNA Medicines Company will be kicking the morning off with a session on Empowering RNA Drug Target Validation: “An Essential Initial Step for Every Drug Discovery Effort”.
John will be covering important points, such as:
- How do we ensure that the RNA we are targeting is a high value drug target?
- As drug hunters how do we validate a RNA as a drug target? Translational medicine in the area of RNA Medicine is still in its nascency, there are very few examples in literature that gives us strong confidence of the role RNA plays in disease.
- What are the best tools to elucidate the role of RNA in disease?
- Once we validate a specific RNA as a drug target how do we generate the appropriate drug like tool molecules to ensure we guide our drug discovery effort with specificity? Drug discovery on Target is critical and always a challenge, particularly with small molecules that are promiscuous in their nature.
- Mechanism of Action; are CRISPR and oligonucleotides appropriate control tools when you develop small molecules targeting RNA?
Margaret Porter Scott, VP, Biochemical & Cellular Pharmacology and John Moffat, Senior Scientist, Biochemical & Cellular Pharmacology at Genentech will lead a discussion-based workshop on Exploring Drug Discovery Approaches to RNA, encompassing:
- What mechanisms are possible or likely to work? We will discuss the various known approaches of affecting splicing, 5’ UTR interference to decrease transcription, binding to mRNA and more.
- What are the pitfalls when transitioning from targeting protein to targeting RNA (what mindsets need to change)?
- What are the key 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?).
Click here to view the full workshop details
Join 100+ of your colleagues to improve your understanding, discuss your research and learn how your peers are tackling the same challenges you have.