Accelerating the Clinical Translation of Selective RNA-Targeting Small Molecules

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 20+ 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

    • 22+ Expert Speakers
    • 3 Days of RNA-Targeted Content
    • 2 Interactive Workshops on Functional Binder Discovery
    • Investments & Deals Panel

 

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World-Class Speaker Faculty Includes:

What to Expect

80+

Attendees

22+

Expert Speakers

18+

Data-Led Presentations

10+

Hours of Dedicated Networking

8+

Hours of Interactive Workshops & Panels

1

Global RNA-Targeted Community

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

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