8:00 am Check In Opens & Morning Welcome Coffee & Light Breakfast
Expanding the Therapeutic Potential of RNAi Drugs by Exhibiting the Next Generation of Therapies to Improve Drugging of Cancer Targets
8:55 am Chair’s Opening Remarks
9:00 am Uncovering the Critical Challenges of Oncology Targeting & Finding Solutions to Improve Drug Development to Enable Acceleration Into the Clinic
Synopsis
• Explore how is the field approaching getting inside cancerous cells
• Uncover how to selectively target immune cells vs tumor cells for treatment of cancer
• Discover advantages of targeting tumor microenvironment with RNAi therapeutics
9:30 am Developing a Delivery Platform for siRNAs to Treat Pancreatic Cancer
Synopsis
• Discussing oligonucelotide delivery to tumors and metastases
• Analyze siRNA-based checkpoint inhibition for the treatment of pancreatic adenocarcinoma
• Explore siRNA delivery to immune cell subpopulations in the context of cancer
10:00 am Morning Break & Speed Networking
Synopsis
This session is your critical to get face-to-face with many of the brightest minds working in the RNAi field, and establish meaningful business relationships to pursue for the rest of the conference
Utilizing RNAi & Screening Approaches to Develop Specific Drugs for More Effective Treatment of Diseases
11:00 am Expanding the Landscape of Multiomic Spatial Analysis for RNAi-based Therapeutics Using miRNAscope & RNAscope Plus Assay
Synopsis
- Detect small RNAs in tissues with spatial and morphological context at single cell resolution
- Assess delivery and biodistribution of the therapeutic oligonucleotides
- Evaluate safety and efficacy of the therapeutic in spatial context within specific cell types
11:30 am Using RNAi to Identify & Validate Novel Targets for Cancer Drug Development
Synopsis
• RNAi knockdown is a key step in determining the best modality for drug development
• Selected Oncology targets may not require complete knockdown!
• Explore examples from the Histone methyltransferase family
12:00 pm DELiveri™: A Conjugate Discovery Platform, Empowered by Combinatorial Chemistry & AI, for ex-Hepatic Selective Delivery of Synthetic RNA
Synopsis
• The DELiveri™ platform ushers in a new era of precision in nucleic acid medicine, enabling high-throughput screening (HTS) of cell-specific targeting small molecules
• DELiveri™ uses a proprietary engineered genetic circuit that transforms screened cells into MOLECULAR RADARs
• Once coupled with DNA-encoded libraries (DELs) and AI for analysis, HTS outputs generate quantitative signals unique to cytoplasm-available entities
Achieving Safe & Efficacious siRNA Therapies by Optimizing Chemical Modifications & Identifying Off-Targets for Faster Approval
12:30 pm Lunch & Networking
1:30 pm Extended Nucleic Acid (exNA): A Platform Technology for Enhancing siRNA Efficacy & Tissue Accumulation In Vitro
Synopsis
• Metabolic stabilization of siRNA backbone for longer duration of effect in vivo
• Significantly improved oligonucleotide plasma PK profile
• The improved potency and durability open extrahepatic tissues and indications to therapeutic interventions
2:00 pm Alternatives for Reducing Number of Undefined Stereo Centres in GalNAcConjugated siRNA
Synopsis
• Regular phosphorothioate internucleotide linkages creates undefined stereo centers during solid phase synthesis
• Phosphorodithioate internucleotide linkages circumvent undefined isomer formation while retaining or increasing metabolic stability
• Synthetic RNA building blocks in key positions can reduce exonuclease mediated degradation without the need for thiolation
Leveraging Protein Conjugates for Extra-Hepatic Delivery to Broaden the Disease Landscape Currently Accessed
2:30 pm Direct Identification of siRNA Off-Target Binding with miR-eCLIP +siRNA
Synopsis
- Off-targets limit the efficacy of RNAi therapeutics
- miR-eCLIP +siRNA enables direct identification of where siRNAs bind across the transcriptome
- Learn how direct detection of siRNA binding reveals the source of RNA-Seq changes
3:00 pm Afternoon Break & Networking
4:00 pm Novel Protein Conjugate for Delivering siRNAs to Tumor Cells
Synopsis
• Discussing the development of the 4th generation technology for targeted delivery of siRNA to extrahepatic tissues
• Describing functional and development advantages of protein-siRNA conjugate (PRC) technology
• What are the most exciting therapeutic targets that take advantage of targeted siRNA delivery?
4:30 pm A Novel Platform to Discover the Right Protein for Extra-Hepatic Delivery
Synopsis
• Aro Centyrin-siRNA platform, lead characterisation and preclinical overview
• Development of a novel targeting ligand platform with optimized properties for extrahepatic siRNA Delivery
• New platform protein ligands deliver siRNA and result in selective gene knockdown in target tissues