Conference Day One
Wednesday, June 25
8:00 am Light Breakfast & Check-In
8:55 am Chair’s Opening Remarks
Unlocking Extra-Hepatic Delivery Through Novel Delivery Systems to Reach Hard-to-Target Organs & Deliver Life Changing Cures for Novel Diseases
9:00 am PANEL DISCUSSION: Exploring the Future of Extra-Hepatic Delivery Systems: Overcoming Barriers & Meeting Unmet Needs Across Therapeutic Modalities
Synopsis
- What are the key strategies for designing next-gen delivery systems that can target extra-hepatic tissues and avoid accumulation in the liver to enable effective RNA therapeutics for a broader range of diseases?
- What challenges are we facing in achieving efficient extra-hepatic delivery, and what is a realistic timeline for consistently overcoming barriers to target specific organs beyond the liver, such as the brain or lungs?
- What are the most pressing unmet needs that new systems can fill to make space in the crowded ecosystem of RNA
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 next generation lipid delivery vehicles to establish meaningful connections to pursue for the rest of the conference
Overcoming Liver Accumulation to Successfully Target Extra-Hepatic Organs & Increase the Therapeutic Efficiency of Nucleic Acid Drugs
11:15 am Breathing New Life into Pulmonary Disease Patients with Lipid-Based Inhalation Systems for Effective Lung Targeting
Synopsis
- Tackling critical obstacles including drug stability during aerosolization, crossing mucosal barriers and precise cell-type targeting to maximize the potency of pulmonary delivery
- Designing an inhalation system with enhanced stability and reduced immunogenicity to improve patient outcomes with a safer therapeutic for pulmonary diseases
- Overcoming particle deposition and uneven therapeutic distribution in the respiratory tract to enhance the effectiveness of inhaled lung-targeted drugs
11:45 am Revolutionizing LNP Targeting with an Adaptable Antibody Technology for Precise Delivery
Synopsis
- Developing a flexible antibody-peptide tag platform that enables LNP targeting across diverse cell types, unlocking novel delivery opportunities in previously unaddressed areas
- Showcasing data to highlight the adaptability and precision of the system for targeted delivery applications
- Presenting cutting-edge innovations in antibody binding and tag attachment techniques that drive efficiency, scalability, and specificity in LNP-based therapeutics
12:15 pm Lunch Break & Networking
1:15 pm Bispecific Antibody-Mediated Targeting of Lipid Nanoparticles for Precision RNA Delivery
Synopsis
- Implementing innovative LNP targeting strategies using bispecific antibodies to confer cell specificity, enhancing RNA uptake while minimizing off-target effects, thereby optmizing therapeutic efficacy
- Validating cell-specific RNA delivery through functional assays and in vivo models, confirming the potential of bispecific antibody-guided LNPs for next-generation RNA therapeutics
- Engineering lipid nanoparticle (LNP) formulations for efficient and selective RNA delivery, achieving 100% targeting of human T cells in vitro and germinal center B cells in vivo, demonstrating precise immune cell modulation
1:45 pm Supercharging TIL Therapy with LNP-Based Neoantigen Vaccines: A New Era in Cancer Immunotherapy
Synopsis
- Developing cutting-edge lipid-based delivery systems designed to specifically activate T-cells through polyepitope vaccines designed to enhance immune responses against common malignancies
- Providing ‘Signal 1’ — precise T cell receptor binding — to trigger T cell proliferation, persistence, and tumor penetration to ensure effective cytotoxic activity against cancer cells
- Accelerating the time from bench to bedside for personalized T-cell therapeutics through rapid formulation techniques, enabling a swift immune response and enhancing therapeutic potency for cancer patient
2:15 pm Afternoon Networking Break & Poster Session
Synopsis
As new next-gen delivery systems are revealed almost every month, it is more important than ever to collaborate and learn from your peers. Get feedback on your latest data and have the first look into what your peers are working on!
Adapting Lipid Systems for Larger Payloads to Transform Gene-Editing Delivery & Aid the Fight Against Genetic Diseases
3:15 pm Unlocking CNS Delivery: A Lipid Nanoparticle Platform Inspired by Natural Brain Chemistry
Synopsis
- Developing unique lipid nanoparticles optimized for CNS applications to enable precise delivery of genetic medicines (such as RNA therapeutics and CRISPR gene editing) for neurodegenerative diseases
- Showcasing in vitro gene editing data and in vivo biodistribution insights to highlight targeted delivery to specific brain regions and cell types
- Huntington’s Disease and MSH3 as clinical targets for translational application of the platform
3:45 pm Development of Targeted LNP for Efficient In Vivo Editing of Hematopoietic Stem Cells
Synopsis
- Designing LNP composition to de-target from liver and redistributed to target organs of interest
- Developing screening platform to select targeting ligands that maximize the internalization of cell-surface targets while maintaining cellular specificity for HSCs
- Conjugating the improved targeting ligands on the optimal liver de-targeting base LNP achieved efficient editing of HSPCs in humanized mice as well as efficient editing in HSCs in NHPs after a single dose