ABOUT EVENT
Welcome to the RNA-Targeted Drug Discovery & Development Summit!
Curated in collaboration with large pharma, innovative biotech and leading academics, the 7th RNA-Targeted Drug Discovery & Development Summit returned with 3-jam packed days and unrivalled networking opportunities with the key stakeholders to accelerate discovery of disease-relevant RNA, optimize small molecule design and uncover novel targets on proteins, enzymes and RNA/protein complexes to increase the landscape of treatable diseases.
25+ industry experts navigated through presentations and interactive discussions to provide you with the specialized roadmap towards developing RNA-targeted small molecules with insights to:
DE-RISK translation of RNA-targeting drugs through improving preclinical modelling strategies and toxicity testing for more accurate and reliable safety profiling with PTC Therapeutics, Expansion Therapeutics & Sanofi
DEEP DIVE into RNA target structures to identify disease relevant RNAs and optimize small molecule selectivity for more efficacious therapeutics with Base4 Biotechnology, Ribometrix & Arrakis Therapeutics
REVOLUTIONIZE treatment of neurological and oncological indications through optimizing selectivity of orally bioavailable RNA-targeting small molecules with PTC Therapeutics, Rgenta Therapeutics, VReviR Therapeutics & University
of Toronto
ILLUMINATE novel targets beyond splicing, such as lncRNAs and repeat RNAs, to expand the potential of the RNA targeting field, enabling treatment of a wider landscape of disease with AstraZeneca & Massachusetts Institute of Technology
LEVERAGE proteins and RNA/protein complexes to target disease causing RNA which is currently undruggable and drive more potent therapeutics towards the clinic with Remix Therapeutics & Accent Therapeutics
New & Noteworthy Sessions in 2024:
“Utilizing RNA-Seq Data At-Scale to Improve the Translatability of mRNA Splicing Modulators”
Steve Lianoglou, Director, Translational Genomics, ReviR Therapeutics
"Large-Scale Discovery of RNA Tertiary Structures & Disease Relevant RNA Targets”
Kevin Weeks, Kenan Distinguished Professor, University of North Carolina at Chapel Hill
“Showcasing Insights to A-Bulge Splice Modulator Selectivity Derived from Studies of Minigenes & Other Genetic Instruments”
Joon Lee, Associate Scientific Director, Biogen
“Leveraging Strategies to Drug RNA-Protein Complexes with Small Molecules to Drive More Novel Therapeutics Towards the Clinic”
Dominic Reynolds, Chief Scientific Officer, Remix Therapeutics
“Fireside Chat - Discussing Investment in RNA-Targeting Therapeutics to Accelerate Advancement of Small Molecule Therapies for Patients in Need”
Joy Ghosh, Managing Director, Abingworth
Yang Zheng, Head, Business Development, Base4 Biotechnology
Perla Breccia
Senior Director, Chemistry
AstraZeneca
The future of drug discovery will be shaped by our understanding on how we will be able to modulate RNA, structure, and function.
Small molecules are ideal probes to interact with RNA and become drugs for the undruggable genome. Attending a meeting where KOLs come together and discuss and share openly their challenges and issues is very important to accelerate success in this emerging field