8:30 am Morning Coffee & Light Breakfast
8:50 am Chair’s Opening Remarks
Delving into Novel Delivery Platforms & Strategies for Successful Delivery to the Liver & Beyond to Fast-Track More Effective Drugs through the Clinic
9:00 am GalAhead™: A Proprietary GalNAc-RNAi Therapeutic Platform to Downregulate Single & Multiple Genes
Synopsis
• Introduction to GalAhead, Sirnaomics’ GalNAc-RNAi therapeutic platform
• Validation of technology in vivo and in vitro
• Progress report on GalAhead-based programs
9:30 am Expert Panel Discussion: The Right Delivery for the Right Therapeutic Area
Synopsis
The major challenge to harnessing RNAi for the treatment of disease is delivery of functional nucleic acid to the target tissues, cells, and subcellular compartments. This panel will discuss challenges and solutions they have found in their specific areas of interest.
10:30 am Morning Networking Break & Scientific Poster Session
11:30 am In Vitro & In Vivo Lipid Nanocrystal (LNC) Delivery of RNAi Oligonucleotides
Synopsis
• Lipid nanocrystals (LNCs) – what they are and what they can do
• Experience to date; recent in vitro and in vivo data with small RNAi oligos
• Future directions and opportunities
12:00 pm Unprecedented, Tissue- & Modality-Agnostic Potency & Durability Improvement by Targeted Endosomal Escape In Vitro & In Vivo: an Update on Sapreme’s Unique Endosomal Escape Enhancer Platform
Synopsis
• Efficient intracellular delivery of oligonucleotides remains a bottleneck in hepatic and especially extrahepatic tissues
• Sapreme’s endosomal escape enhancers allow for highly efficient and tissue specific intracellular delivery, using existing targeting ligands and payloads
• We will share data demonstrating in vivo liver, muscle and solid tumor delivery of oligonucleotides, allowing strong dose reductions whilst maintaining durability of effect
12:30 pm Lunch & Networking
Illuminating Optimal Strategies for Faster Development of Novel RNAi Platforms to Enable Arrival to Patients Sooner
1:30 pm Dry Powder Formulations for Pioneering Local siRNA Delivery to the Lung
Synopsis
• Nano-Embedded Microparticles (NEMs) offer a breakthrough in inhalable RNA therapeutics, ensuring optimal lung- delivery and enhanced compliance.
• Adopting a cost-effective, scalable, and continuous spray-drying technique ensures particles optimized for deep lung delivery.
• RNAi-based dry powder treatment against type 2 asthmatic disease demonstrates remarkable efficacy without any toxic effects in vivo.
2:00 pm Expert Panel Discussion: Reviewing Production Strategies for New Delivery Platforms to Optimize Manufacturability to Enable Scale Up for the Clinic
Synopsis
• Are peptide conjugates easier to produce on a larger scale compared to antibody conjugates?
• How to ensure quality is maintained through the process?
• How is the process being kept cost efficient?
3:00 pm Afternoon Break & Networking
Navigating Critical Manufacturing Challenges to Improve Feasibility of RNAi Therapeutics & Allow Quicker Production
3:30 pm Manufacturing Approaches to Allow Robust Production of siRNA for Faster Approval
Synopsis
• Phase-appropriate process development to balance the speed with reliability of clinical supply
• Sound technical understanding and platform knowledge complimented by rational strategy for rapid development
• Judicious implementation of new technologies to properly manage clinical/commercial demand
4:00 pm Preparation for Transition from Early to Late-Stage Manufacturing
Synopsis
• Discussion around early mistakes in API and drug product development and how to correct them
• Demonstration of the criticality of non-release testing on product understanding
• Use of market analysis in you late-stage planning for scale and annual production cycle