Optimizing RNA-Targeting Small Molecules Through Structure-Guided Design, Selective Modulation, and Translational Strategies to Develop Safe, Bioavailable, and Clinically Effective Therapies
RNA-targeting small molecules are rapidly emerging as a powerful therapeutic reality. From modulating splicing to directly binding RNAs, these molecules offer a revolutionary approach to treating previously undruggable diseases across neurodegenerative, cancer and rare disorders. In 2025 alone, the field has seen major collaborations, including Wayfinder Biosciences with Daiichi Sankyo, Ono Pharmaceutical with Reborna Biosciences, and xFOREST with Astellas Pharma—signaling growing momentum and industry confidence.
Now in its 8th year, the RNA-Targeted Drug Discovery & Development Summit returns as the only dedicated industry forum addressing the unique challenges of RNA-targeted drug development across splice modulators, covalent drugs, proximity inducers and direct RNA binders.
Join 25+ world-class speakers from leaders like PTC Therapeutics, ReviR Therapeutics, Remix Therapeutics, Rgenta Therapeutics, Arrakis Therapeutics and more this December to gain the connections, tools and scientific firepower needed to bring your RNA-targeted therapeutics to clinic and beyond.
Explore the Full Event Guide
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- 22+ Expert Speakers
- 3 Days of RNA-Targeted Content
- 2 Interactive Workshops on Functional Binder Discovery
- Investments & Deals Panel
World-Class Speaker Faculty Includes
Attend as a team. Leave with a strategy.
Align Strategy Across RNA Drug Discovery
Unite cross-functional teams around shared challenges—from early target validation and RNA structure elucidation to novel binder chemistry—for a more integrated approach to RNA-targeted drug development.
Tackle Cross-Departmental Challenges in One Place
Bring together Directors and Scientists from discovery biology, chemistry, structural biology, pharmacology, and more to break down organizational silos and address interconnected pain points across strategy and technical teams.
Benchmark Against Industry Innovators
Learn directly from senior leaders at companies like PTC Therapeutics, Remix Therapeutics, Revir Therapeutics and more to see what’s working across the most complex RNA-targeted programs—then adapt those strategies for your own pipeline.
Empower Your Team with Pharma & VC Insights
From insights on investment trends and recent deals to what large pharma and VCs look for in a biotech, equip your team with practical tactics to attract collaborators and funding to accelerate RNA drug discovery and development.
Maximize Value with Group Savings
Secure up to 20% off your pass with team discounts—and ensure your entire team walks away aligned, inspired, and ready to deliver on your RNA-targeted drug discovery goals.
'' This conference is a unique opportunity for experts from academia, biotech, and pharma to come together and discuss specific therapeutic modalities. It helps me broaden my knowledge in the field and creates more opportunities for collaboration ''
Akihiro Furukawa, Director, Research & Technology Search & Evaluation, Global Business Development
Daiichi Sankyo
Top Testimonials
'' The 8th RNA-Targeted Drug Discovery & Development Summit offers a unique opportunity to engage with leading experts and innovators who are driving progress in this rapidly evolving field. As RNA-targeted therapeutics advance, the summit serves as a vital platform to exchange cutting-edge insights, foster collaboration across academia and industry, and accelerate breakthroughs. I look forward to contributing to the dialogue and helping to advance the next generation of RNA-targeted drugs. ''
Ella Czarina Morishita, Chief Scientific Officer,
Veritas In Silico
'' The RNA-Targeted Drug Discovery & Development Summit has evolved into a valuable touchbase for scientists spanning diverse areas of drug discovery and biotech. It serves as a forum for connection and discussion, as the field aggressively pursues novel therapies for diseases of unmet medical need ''
Christopher Trotta, Senior Vice President, Discovery Biology
PTC Therapeutics
View the 2025 Blind Delegate List
Who is confirmed to attend this December?
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