9:00 am Morning Coffee
9:25 am Chair’s Opening Remarks
Spotlighting Novel Approaches of Direct RNA Binders to Treat Disease with Unmet Need
9:30 am RNA Degradation – Novel Approaches to Targeting RNA with Small Molecules
Synopsis
- Is RNA degradation approach the future of RNA-targeted drug discovery?
- Identifying small molecules that bind to RNA and understanding their functionality as part of their drug design
- What are the current tools being used to study knock-down effects?
10:00 am Allele-Selective Targeting of HTT RNA with Small Molecules
Synopsis
- Selection of the RNA target by leveraging structural tools
- Development of methods for screening and characterizing RNA-binding small molecules
- Identification of small molecules that interact with HTT RNA
10:30 am Morning Break & Scientific Poster Session
11:30 am AI in mRNA Biology: Small Molecule Drug & Target Discovery
Synopsis
- Visualizing the complete mRNA life cycle from multiple disease models using highcontent mRNA imaging technologies
- Decoding the mRNA biology underlying disease phenotypes with AI technology using mRNA image neural networks, knowledge graph, large language model, and its copilot
- Examples and case studies in oncology
Turbocharge Screening Tools & AI to Improve Translatability from In Vitro to In Vivo
12:00 pm Magnetic Force Spectroscopy – a Novel Approach to Improve the Hit-to-Lead & Lead Optimization Phases of RNA-Targeted Small-Molecule Drug Discovery
Synopsis
- Overview of MAGNA technology which employs magnetic force spectroscopy to analyze biomolecular interactions with single molecule resolution
- Acquisition of kinetic and structural information about small molecule binding to RNA targets
12:30 pm Lunch & Networking Break
Successfully Develop Highly Specific Small Molecule Inhibitors in Oncology for Advanced Translation
1:30 pm Discussing Novel Best-in-Class Inhibitors of RNA Modifying Enzymes in Oncology for Preclinical Progression
Synopsis
- Targeting different RNA modifying enzymes using in-house established discovery platform to identify and develop novel highly specific small molecule inhibitors for advanced translation and progression into clinic
- Exploring case studies of preclinical programs of the best, first in class molecules in oncology with the goal of providing novel therapeutic approaches for patients with unmet medical need
2:00 pm Roundtable: Discussing the Different Strategies & Approaches of Translating Binding & Functionality Effects to the Clinic
Synopsis
- Understanding tonality and the evolution of novel small molecule inhibitors for improved efficacy in in vivo models
- What are the current strategies and approaches to successfully translate functionality into the clinic and how can these be improved?
3:00 pm DHX9 Inhibitors as Novel Therapeutic Modalities in MSI-High Cancers
Synopsis
- DHX9 is an RNA helicase with important roles in replication, transcription, translation, RNA splicing and RNA processing
- Microsatellite instable high (MSI-high) cancer cell lines are selectively dependent on DHX9 activity
- Potent inhibitors of DHX9 have been identified that show antiproliferative activity in MSIhigh cancer models in vitro and in vivo