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 PTC Splice Modulators: From a Novel Discovery to a Broad Therapeutic Platform (PTSeek)
Synopsis
- Sequence specific splice modulators have demonstrated therapeutic application for SMA and HD
- PTC has designed a discovery platform, PTSeek, that couples proprietary splicing data analysis tools with a unique library of small molecules
- Several novel series of molecules are being advanced to influence the expression of disease relevant genes through splicing modulation
- Novel splicing modulators target 5’ splice site sequences that are differentiated from the standard “GA-space” occupied by SMA and HD compounds
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
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
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
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
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
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