Explore the Agenda
8:30 am Check-In & Light Breakfast
8:50 am Chair’s Opening Remarks
Moving Beyond Traditional LNPs by Incorporating Novel Polymers & Proteins for Selective Delivery Beyond the Liver
9:00 am Payload Delivery Tool Kit: Lipids vs Polymers
- Compare lipid and polymeric nanoparticle platforms with a specific focus on extra hepatic payload delivery
- Differences in biodistribution, cellular uptake, endosomal escape, payload compatibility, repeat dosing, and translational readiness
- Highlight why extrahepatic delivery remains challenging for lipid nanoparticles and where polymeric systems may offer advantages, as well as the trade-offs involved in moving beyond clinically validated lipid platforms
9:30 am Panel Discussion: Exploring Next-Generation Nanoparticle Systems Beyond LNPs to Expand Extrahepatic Targeting, Improve Safety, & Enable Broader Therapeutic Delivery
- Where do LNPs begin to show limitations for extrahepatic delivery and which emerging delivery vehicles offer the most realistic advantages?
- How do alternative delivery platforms compare in terms of manufacturability, scalability, safety, and regulatory readiness when moving towards the clinic?
- Which delivery challenges, from immunogenicity to targeting specificity, are best addressed by exploring non-LNP platforms, and where do LNPs remain the most practical solution?
10:00 am Morning Break & Networking
Optimizing Peptide Design to Minimize Liver Toxicity & Improve Cytosolic Delivery to Enhance Delivery Efficiency & Specificity
11:00 am Overcoming Hepatic Accumulation Using a Redosable Targeted Peptide-Based Nanoparticle Platform
- AI-driven design and optimization of a peptide-based targeted RNA delivery system
- PEP-NPTM: Highly selective, best-in-class, peptide nanoparticles for extrahepatic mRNA delivery
- In vivo mRNA based CAR-T cell engineering using novel targeted peptide-based nanoparticles
11:30 am Enhancing Cytosolic Delivery of Lipid Nanoparticle Payloads Using Peptides
- Incorporating peptides in LNPs had minimal impact on particle properties and showed improved intracellular delivery of mRNA
- The presence of peptides in LNPs did not change particle biodistribution in vivo
- Peptide-LNPs showed higher mRNA delivery in most organs vs LNP control
12:00 pm Lunch & Networking
Overcoming LNP Toxicity by Incorporating PEG-Alternatives & Modifying LNP Chemistry for Safer Immune Cell Delivery
1:00 pm Evaluating PEG-Alternative LNPs for Extrahepatic Delivery in In Vivo Cell Reprogramming
- Novel Ionizable Lipid: Demonstrates enhanced T-cell tropism with reduced hepatic uptake
- In Vivo Characterization: Ongoing studies on biodistribution, safety, and toxicity profiles
- Formulation Strategy: Assessing PEG-alternatives in combination with ionizable lipids for delivery feasibility
1:30 pm Encoding Active Antigen-Specific Immune Tolerance Using mRNA-LNPs for the Treatment of Autoimmune Diseases
- Highlighting the advantages of incorporating an active immune modulator into antigen-specific tolerance strategies
- Exploring critical factors in applying mRNA-LNP technology to autoimmune conditions, including challenges and successes targeting immune cell delivery
- Presenting preclinical findings from autoimmune disease models
2:00 pm Roundtable Discussion: Discussing Targeted LNP Design to Minimize Immunogenicity, Maximize Safety, & Protect Sensitive Tissues for Extrahepatic Applications
- How can lipid chemistry and functional group modifications be leveraged to reduce toxicity and off-target effects while maintaining delivery efficiency?
- Which design strategies and targeting ligand modifications best balance tissue specificity, immunogenicity, and safety across sensitive extrahepatic targets?
- How can LNP formulations be engineered to mitigate immune recognition and anti-drug antibody formation to achieve tolerable repeat dosing?
2:30 pm Afternoon Break & Networking
Fine-Tuning LNP Formulations to Reduce Hepatic Accumulation for More Potent Oncology Therapeutics
3:30 pm Targeted Therapeutic Delivery Using Programmable RNA Architectures for Oncology Indications
- Programmable RNA architectures maintains defined structural integrity in circulation, enabling precise molecular targeting and controlled payload presentation across therapeutic modalities
- Preclinical biodistribution studies demonstrate minimal nonspecific uptake, with little to no detectable accumulation in the liver and rapid renal clearance of unbound material when target engagement does not occur
- Complementary RNA nanostructure and RNA-programmed vesicular systems can be selectively deployed based on biological constraints and uptake requirements, supporting targeted delivery beyond conventional lipid-based approaches
4:00 pm A Structural Nanomedicine Platform for Efficient Extrahepatic Delivery of Nucleic Acid Therapeutics
- Applying the discovery of spherical nucleic acids by Chad Mirkin’s laboratory at Northwestern University to multiple treatment modalities, including immuno-oncology, RNA therapeutics, non-viral gene therapy, gene editing, and in vivo CAR-T
- Improving stability and cellular uptake via precision-engineered architecture
- Avoiding hepatic accumulation and targeting disease-specific tissues
4:30 pm Enhancing Design & Characterization of LNPs to Penetrate & Treat Organ Specific Tumors
- Optimizing LNP design by leveraging targeting ligands for effective organ-specific delivery
- Overcoming physical barriers by addressing particle size limitations to improve penetration into the dense organ specific microenvironment
- Improving stability of targeted LNPs for robust delivery and treatment of oncological indications