Conference Day One

7:45 am Check-in & Morning Coffee

8:45 am Opening Remarks

Synopsis

• Highlight past and ongoing efforts to develop RNA-targeted therapeutics

• Explore the history and advancement of splicing modifiers as the cornerstone in RNA-targeted discovery

• Provide a meeting roadmap, showcasing the exciting science that stems from foundational clinical successes in RNA-targeted therapeutic development

Advancing RNA-Targeted Therapeutics to Unlock Therapeutic Effect & Accelerate Translation Across Disease Areas

9:00 am Building Mechanism-Driven Screening Platforms to Accelerate Functional Hit Discovery & Advance RNA-Targeted Drug Programs

  • Matt Woll Senior Vice President of Chemistry, PTC Therapeutics

Synopsis

• Segmenting complex RNA sequences into biologically relevant motifs to enable structure-guided screening and improve hit quality

• Deploying in-house biosensors with quantitative fluorescent readouts to identify highaffinity binders and prioritize functional RNA structures

• Integrating biochemical screening with cellular validation to refine structure selection and increase the likelihood of phenotypic response

9:30 am Drugging RNA–Protein Complexes with Small Molecules to Enable RNA Splice Modulation & Degradation

Synopsis

• REM-422 is being developed by Remix Therapeutics for the treatment of ACC and AML/HRMDS

• REMaster™ platform identifies compounds that address undruggable high unmet medical need targets

• Next generation Remix drug discovery programs are enabled by a suite of biophysical assays and expand the scope of pharmacologically tractable splice modulator modalities

10:00 am Morning Break & Speed Networking

Synopsis

This networking session is your opportunity to get face-to-face with many of the brightest minds working with RNA-targeted therapeutics to establish meaningful connections to pursue for the rest of the conference

11:00 am Accelerating Splicing Modulation Therapies for Neurodegeneration with the VoyagR Platform

  • Peng Yue Co-Founder & Chief Executive Officer, ReviR Therapeutics

Synopsis

• Finalizing development of a lead candidate targeting HTT and PMS1 splicing with support from CIRM for IND-enabling studies in Huntington’s disease to accelerate clinical readiness and unlock therapeutic potential

• Accelerating preclinical development with refined single-molecule chemistry and new functional datasets to enhance compound validation and improve translational confidence in ReviR’s lead asset

• Expanding ReviR’s pipeline with additional splicing-modulation programs in spinal muscular atrophy and Charcot-Marie-Tooth disease to broaden therapeutic reach and address unmet needs in neurodegeneration

11:30 am Presentation Details to be Revealed

12:00 pm Lunch Break & Networking

Expanding Chemical & Mechanistic Diversity to Overcome Design Constraints & Enable Orally Available RNA Therapeutics

1:00 pm Panel Discussion Increasing Chemical Diversity in RNA-Targeted Splice Modulator Small Molecules to Expand the Target Space & Address Previously Undruggable Diseases

  • Matt Woll Senior Vice President of Chemistry, PTC Therapeutics
  • Dominic Reynolds CSO, Remix Therapeutics
  • Peng Yue Co-Founder & Chief Executive Officer, ReviR Therapeutics
  • Perla Breccia Senior Director of Chemistry, Astex Pharmaceuticals
  • Shunichi Kashida Co-Founder, Representative Director, President & CEO, xFOREST Therapeutics Co. Ltd.
  • Thomas Wynn Director of Medicinal Chemistry, Rgenta Therapeutics Inc.

Synopsis

• How can expanding the target space beyond the common splice modulators unlock new opportunities for targeting novel disease areas

• What strategies are enabling the design of chemically diverse scaffolds to overcome current limitations in the ligand design space

• Are we overdependent on nonsense-mediated decay and can reducing this reliance drive broaden therapeutic applicability of splice modulators

• What lessons can be drawn from recent investments and platform development to guide future innovation in RNA-targeted small molecules

2:00 pm Breaking the Dimensional Limits of RNA–Ligand Discovery: High- Throughput Insights into Structure, Selectivity, & Function

  • Shunichi Kashida Co-Founder, Representative Director, President & CEO, xFOREST Therapeutics Co. Ltd.

Synopsis

• Characterizing RNA motifs with both ligandable pockets & functional elements to improve druggability & achieve mechanism-based targeting

• High-resolution quantification of RNA–ligand binding enables SAR-guided optimization from submicromolar hits to nanomolar binders

• Interaction big data-driven delineation of novel chemical space to inform future design of selective drug-like RNA binders

2:30 pm Presentation Details to be Revealed

  • Karen Wu Chief Executive Officer & Co-Founder, Lucerna, Inc.

2:40 pm Afternoon Networking Break & Poster Session

Synopsis

Contribute to the conversation & showcase your groundbreaking research in RNA-targeted drug discovery and development. To present a poster, register your place & submit an abstract highlighting your latest discovery.

Harnessing RNA Splice Modulators to Target Structured Motifs & Employ Nonsense-Mediated Decay Against Nucelotide Repeat Disorders

3:45 pm Targeting Pathological RNA in Myotonic Dystrophy (DM1): Advancing a Small Molecule with Intrinsic Activity to Treat a Systemic Disease

Synopsis

• Harnessing structure-based drug design to develop rSMs to normalize RNA splicing and relieve myotonia

• Optimizing the pharmacological properties of an rSM with systemic biodistribution and functional activity in disease-relevant models

• Demonstrating molecular correction and phenotypic rescue to support the translational potential of a first-in-class small molecule against DM1

4:15 pm Optimizing Chemical Matter Against RNA to Improve Therapeutics: Lessons from Rgenta’s RNA-Targeted Platform

  • Thomas Wynn Director of Medicinal Chemistry, Rgenta Therapeutics Inc.

Synopsis

• Introducing Rgenta’s RNA-targeting small molecule platform and its approach to addressing disease-relevant RNA targets

• Highlighting case studies from lead programs in ACC & Huntington’s disease to showcase platform-driven success in drug discovery

• Sharing insights on generating and refining small molecules as RNA splice modulators, including strategies for modeling splice sites

4:45 pm Chair’s Closing Remarks

  • Perla Breccia Senior Director of Chemistry, Astex Pharmaceuticals

5:00 pm End of Conference Day One