Day One

Deliveromics & GT AI for Cell Type Specific Ligand-LNP Design

• Deliveromics is a high-throughput screening platform that maps the delivery of hundreds of ligand-LNPs in vivo with cell type resolution
• GT AI designs both ligands and LNP formulations for cell-type specific delivery
• GT ligand LNPs crossed blood-brain barrier and transfected specific neuronal populations in mice

• How to optimize screening approaches to rapidly evaluate large libraries of LNP compositions and identify candidates with enhanced extrahepatic tropism?
• What screening approaches are most effective for selecting and testing targeting moieties to ensure high specificity, manufacturability, and reproducible performance after LNP conjugation?
• How can AI/ML and predictive models be integrated into experimental screening workflows to improve lipid and ligand selection?

• We develop a new high-throughput screening strategy that can screen LNP libraries and accurately predict delivery in vivo directly from tissue sequencing
• Our strategy can screen LNPs in organs that traditional screening methods are impractical, such as the heart, kidneys, and brain
• We use our strategy to screen a new class of lipids to identify LNPs with extrahepatic tropisms and low reactogenicity

This session is your opportunity to get face-to-face with many of the brightest minds working on extrahepatic delivery, and establish meaningful business and scientific relationships to pursue for the rest of the conference

• Introduce a SHARP platform that enables efficient nucleic acid delivery without multicomponent formulation complexity
• Demonstrate programmable organ tropism achieved through molecular-level control of amphiphilic structure, enabling selective delivery to organs such as liver, lung, spleen, and the lymphatic system etc
• Highlight the translational potential and future directions of this platform for organ-selective nucleic acid therapeutics across diverse disease indications

• Redefining the LNP Core (The “Active Fusion Core”): Introducing the Hyper-15™ Proteolipid Vehicle (PLV), a novel LNP active fusion core architecture embedded with a proprietary “Wedge & Motor” fusion engine. Unlike first-generation viral fusion proteins that rely on slow “hydrophobic insertion” loops, this platform utilizes a novel method to sterically displace membrane hydration, rendering the core mechanism insensitive to target lipid composition
• Bypassing the “Acidification Trap”: While standard LNPs lose >98% of cargo to endosomal degradation, Hyper-15™ completely bypasses the endocytic pathway. By forcing active, instantaneous fusion at the plasma membrane surface, we demonstrate the ability to achieve 100% cytosolic bioavailability of genetic payloads without requiring pH-dependent acidification or receptor upregulation
• Unlocking “Endocytically Quiet” Tissue: Presenting breakthrough efficacy data in Human Skeletal Muscle Myotubes and Adipocytes. We show how this mechanical fusion mechanism successfully transfects mature, non-dividing tissues that are impenetrable to standard LNPs, overcoming the industry’s longest-standing delivery bottleneck in metabolic and neuromuscular disease

• Overview of Stylus’ in vivo gene editing system and therapeutic potential
• Key insights on optimizing process conditions to enhance mRNA and DNA LNP formulation quality and consistency
• Advances in lipid formulation design that improve extrahepatic biodistribution and drive efficient T-cell delivery in vivo

• What are the remaining barriers for achieving efficient, organ-specific delivery to difficult targets?
• Which design strategies and technologies are most promising for enabling precise extrahepatic targeting while minimizing liver accumulation?
• What unmet therapeutic needs could next-generation extrahepatic LNPs address to expand the RNA, gene editing, and cell therapy landscape?

As extrahepatic targeting for LNP-mediated therapeutics continue to progress from strength to strength, it is more important than ever to collaborate and learn with your peers. Join this dedicated session to share your latest data and have the first look on what your peers are working on!

• Introducing key strategies to achieve immune-cell specific and organ-specific delivery using nanoparticles
• Examining how LNP design choices shape immune cell delivery and modulation
• Beyond CAR-T: Investigating in vivo CAR approaches for additional immune cell types

• RNA-based therapy that controls the cGAS-STING pathway in vivo
• cGAS-STING pathway activation in T-cells to create an intracellular factory of apoptosis-inducing signals
• Delivery of mRNA to activated T-cells mediated by targeting moieties on LNPs