Conference Agenda
Wednesday, December 3, 2025
* All timings shown in EST
8:30 am Digital Networking Room Opens
8:55 am Chair’s Opening Remarks
From Bench to Bedside: Immunogenicity Risk Assessment & Regulatory Considerations for LNP-Delivered Modalities
9:00 am Quality, Safety & Efficacy: Building a Phase-Appropriate Foundation for Regulatory Success in LNP Development
Synopsis
- Understanding how quality, safety, and efficacy data requirements evolve from early clinical trials to marketing authorization, and why foundational thinking should start early for safety and approval
- Reframing regulatory frameworks as protective tools for both patients and developers, mitigating legal risk while guiding better science
- Identifying and addressing data gaps early on to ensure a comprehensive understanding of product behavior and de-risk development decisions to ensure smoother approval
9:25 am Immunogenicity Risk Assessment of LNP-Delivered Nucleic Acid Modalities in Therapeutic Applications
Synopsis
- Discussing how these therapies can trigger unwanted innate and adaptive immune responses, potentially impacting treatment efficacy and patient safety
- Describing strategies for product design adaptations during early development stages to minimize immunogenicity
- Outlining recommendations for defining an Immunogenicity Risk Assessment based clinical testing strategy driven by immunogenicity consequences, including streamlined monitoring for minimal impact scenarios and comprehensive testing protocols when safety concerns are anticipated or arise
9:50 am Q&A Session
Synopsis
Join our speakers for an interactive Q&A session, where they’ll come together to answer your questions, share further insights, and expand on key themes from their presentations.
10:10 am Morning Break & Virtual Networking
Synopsis
This session is the ideal opportunity to connect with many of the brightest minds working in the LNP around the world and establish meaningful business relationships, through randomly allocated, one-to-one meetings
Accelerating the Development of Safer, Next-Generation Lipid Nanoparticles Through Artificial Intelligence & Preclinical Models
10:55 am Designing Safer LNPs With Machine Learning: Reducing Immunogenicity & Toxicity Through Predictive Modeling
Synopsis
- Discussing how machine learning models are being trained to predict innate immune activation, cytokine profiles, and complement responses based on LNP composition to optimize lipid and ligand selection early in the design process
- Using algorithm-driven screening to identify novel lipid structures with minimal immune stimulation and improved biocompatibility
- Applying machine learning to triage formulations prior to testing, enabling earlier identification of low-risk candidates and safer design iterations
11:25 am Bridging the Translational Gap in Extrapolating LNP Safety & Immunogenicity in Preclinical Models
Synopsis
- Exploring how rodents, non-human primates, and humans differ in complement activation, cytokine responses, and anti-PEG antibody formation to determine immunogenicity
- Discussing the limitations of rodent and monkey data in forecasting human immunogenicity and toxicity, with examples from mRNA vaccines and therapeutic LNP studies
- Exploring strategies for dose scaling, biomarker selection, and aligning immune endpoints across species to improve predictive accuracy and reduce clinical surprises
11:50 am Q&A Session
Synopsis
Join our speakers for an interactive Q&A session, where they’ll come together to answer your questions, share further insights, and expand on key themes from their presentations.
12:10 pm Lunch Break
Improving First-Generation LNPs & Advancing the Development of Safer Second-Generation Degradable LNPs
1:10 pm Impact of PEGylation on Repeat Dosing: Balancing Immune Responses & Therapeutic Efficacy in LNP Delivery
Synopsis
- Outlining how Orna Therapeutic’s LNP therapeutic programs focusing on linking safety assessment and immune response evaluation
- Exploring immunogenicity assessment strategies including anti-particle and anti-transgene assays, plus cytokine/chemokine biomarker analysis
- Advancing toxicology considerations for next-generation LNPs and how integrated safety–immunogenicity data informs safer design decisions.
1:50 pm Immunological Assessment in in Vitro Models for the Development of Safer LNPs
Synopsis
- Exploring the safety implications of introducing targeting ligands into LNP systems, including antibodies and peptides as a fifth component beyond the traditional lipid architecture
- Discussing innate immune activation, complement responses, and cytokine profiles from ligand-functionalized LNPs
- Assessing potential off-target effects and unintended biodistribution caused by ligand incorporation to minimize systemic toxicity
2:15 pm Q&A Session
Synopsis
Join our speakers for an interactive Q&A session, where they’ll come together to answer your questions, share further insights, and expand on key themes from their presentations.
2:30 pm Afternoon Break
Delivering Lipid Nanoparticles to Extrahepatic Tissues by Redesigning Formulations for Selective & Targeted Therapeutic Distribution
3:00 pm Overcoming Immunogenicity Challenges in Extrahepatic mRNA-LNP Delivery
Synopsis
- Discuss why efforts to go beyond the liver can heighten immune risk and how to design smarter LNPs that minimize it
- Review approaches to control innate immune responses when directing LNPs to organs like the heart, lung, or spleen
- Discover how administration methods and LNP composition influence immune profiles in non-hepatic targeting
3:25 pm Developing Preclinical Safety Assessment of mRNA-LNP Therapeutics for Solid Tumors
Synopsis
- Presenting key preclinical safety findings with LNP formulations
- Exploring applications in oncology, with a focus on solid tumors
- Discussing translational considerations for advancing mRNA-LNP therapeutics
3:50 pm Q&A Session
Synopsis
Join our speakers for an interactive Q&A session, where they’ll come together to answer your questions, share further insights, and expand on key themes from their presentations.
Advancing Dose Optimization in LNP Therapeutics: Addressing Repeat Administration, Cumulative Toxicity & Maximum Tolerated Dose
4:00 pm Fireside Discussion: Overcoming Barriers in Repeat Dosing & Dose Escalation of LNP Therapeutics
Synopsis
- Exploring how PEGylation impacts repeat dosing, including the development of anti-PEG antibodies, their effect on clearance and efficacy, and strategies to optimize PEG amount, chain length, and alternatives to minimize immune responses
- Discussing non-clinical and clinical biomarkers that inform on cumulative toxicity, highlighting translational markers that correlate with human outcomes and guide safety margins, dose selection, escalation decisions, and long-term monitoring
- Reviewing strategies to safely increase overall dose in LNP therapeutics by mitigating key toxicities, modifying LNP composition or administration routes, and applying real-time assessment of immunogenicity and inflammation to balance efficacy with immunotoxicity management