7:30 am Morning Registration & Coffee
8:00 am Developing LNP/mRNA Therapeutics to Achieve Antigen-Specific Immune Tolerance
Synopsis
• Highlighting the unique benefits of using a Multi-Cargo LNP approach to deliver autoantigens and modulate the immune system
• Discussing the biodistribution and mechanism of LNP/mRNA in inducing immune tolerance
• Showcasing the therapeutic potential of LNP/mRNA technology in type 1 diabetes and other autoimmune diseases
8:20 am Chair’s Opening Remarks
Cell Therapy & mRNA Technologies for Enhanced Tissue Specificity in Inducing Immune Tolerance
8:30 am Harnessing Antigen Specific CAR-Treg Cells for Enhanced Specificity Autoimmune Diseases
Synopsis
• Engineering CAR-Treg cells that recognize self-antigen specific to the site of inflammation
• CAR-Tregs to enhance localization and the potency of Treg therapies to treat inflammatory bowel disease
• Discuss the potential advantages of antigen-specific CAR-Treg cells compared to Polyclonal Treg therapies
9:00 am New insights into the MOA for systemic tolerance induction with IM administered SNAP-TI nanoparticles and updates on the clinical candidate VTP-1000
9:30 am LNP-MRNA Strategies to Restore Immune Homeostasis in Autoimmune Indications
Synopsis
• Antigen discovery and selection for autoimmune diseases
• The potential for mRNA therapeutics to restore immune homeostasis
• Preclinical animal studies demonstrating safety and tolerability for mRNA medicines
10:00 am Morning Refreshments & Networking
Nanoparticle Technologies for Enhanced Tissue Specificity in Inducing Immune Tolerance
10:30 am Pioneering the Use of Nanoparticles to Enable Disease-Specific Immune Modulation
Synopsis
• Uncovering the biologic and chemical components of targeted nanoparticles for disease specific effects
• Engineering nanoparticles to bind to specific autoantigen-experienced T-cells
• Outlining the advantages of tissue-specific targeting for a larger expansion of disease specific Tregs
11:00 am Showcasing the Antigen-Specific CNP Nanoparticle Treatment Regulates CD8+ T Cells in a Model of Type 1 Diabetes
Synopsis
• Developing nanoparticles that bind to monocytes and enhance uptake
• Ensuring optimal, targeted delivery of ASIT therapies through nanoparticles
• Harnessing COUR’s nanoparticle technology to reprogram the immune system for a breakthrough approach to treating autoimmune disease
11:30 am Exploring the Nanodisc Platform for the Induction of Antigen-Specific Immune Tolerance
Synopsis
• Developing a proprietary technology that can effectively stimulate dendritic cells to orchestrate immune responses
• Manipulating dendritic cells to induce either immune activation or tolerance, depending on the stimulation they receive
• Efficiently delivers antigens to lymph nodes, where dendritic cells are concentrated, enhancing the immune response
12:00 pm Lunch & Networking
Demonstrating Tolerance Induction in Preclinical Models to Propel Immune Tolerance Therapies from Bench to Bedside
1:00 pm Panel Discussion: Practical Approaches for Creating a Compelling Preclinical Data Package for IND Submission to Move ASIT Therapies from the Lab to the Clinic
Synopsis
• Highlighting the importance of robust clinical data to secure funding, partnerships, and regulatory buy-in in a new area with limited proof-of-concept studies
• Turbocharging the development of humanized mouse models and organoids to accurately replicate human autoimmune diseases for increased preclinical data
• Evaluating strategies for first in-human trials: when and where should the first patient be dosed?
1:30 pm Antigen-Specific Cell targeting for Antigen Discovery and Cell Reprogramming
Synopsis
• Understanding the targets of T and B cells is key to determining the mechanism of disease
• Targeting viral vectors via pMHC-TCR interactions can provide a means of decoding immunity as well as reprogramming cells for desirable function
2:00 pm Validating the Safety & Efficacy of Antigen Specific Immune Tolerance Therapies Using In Vitro & In Vivo Assays
Synopsis
• Evaluating the ability of APCs to present the target antigen to T-cells
• Discussing the advantages and disadvantages of animal models to study the autoimmune diseases
• Establishing a correlation between in vitro and in vivo findings to identify consistent trends and patterns to validate the efficacy
2:30 pm Afternoon Break & Networking
Future Directions: What’s Next in Antigen Specific Immune Tolerance?
3:00 pm Panel Discussion: Tissue Vs Antigen Specificity: Considering Larger Patient Populations & More Complex Diseases
Synopsis
• Examining the instances in which a tissue-specific response might be superior to an antigen specific-immune response
• Harnessing the epitope-spreading phenomena to develop ASIT therapies for diseases with unknown antigens
• Evaluating the risks of tissue-specific approaches being immunosuppressive rather than resetting the immune system
3:30 pm Panel Discussion: Future Directions in Tolerizing Therapies
Synopsis
• Creating a convincing preclinical data package to ignite regulatory excitement and push more ASIT therapies toward the clinic
• Strategies for determining clinical endpoints by phase of development – are surrogate endpoints the way forward?
• Assessing optimal modalities and disease areas for future development – are cell therapies the future of ASIT? Will
modulating the microbiome for IBD and Colitis be the next therapeutic area to tackle with ASIT therapies?