8:25 am Chair’s Opening Remarks
Harnessing Autoantibody Signatures and Mapping Out the B&T Cell-Mediated Autoimmune Disease Landscape
9:00 am Systemic vs. Organ-Specific Autoimmune Disease: Unique Challenges & Opportunities
Synopsis
- Overview of systemic vs. organ-specific autoimmunity using SLE and stage 2 T1D as examples, relating to “mainstream” vs. rare autoimmune disease states
- Central and peripheral tolerance mechanisms of these diseases
- Exploiting the disease process – successes and remaining challenges
9:30 am Panel Discussion:Mapping the Therapeutic Target Landscape for B-Cell Mediated Autoimmune Disease & T-Cell Dependent Autoantibody Responses
Synopsis
- Assessing the depletion of B-cells with anti-CD20 monoclonal antibodies
- Exploring how to best target CD8 T suppressor cells
- Discussing the various autoantigen specific B cell depleting and modulating therapies (CAART, BTK inhibitors, anti-CD20), targeting T-cell co-stimulatory pathways (CD40/CD40L, CD28/CTLA-4, OX40, ICOS, PD-1), and subduing autoantibody (FcRn inhibitors) approaches
10:00 am T Cell Biomarkers in Autoimmunity for Target Discovery & Treatment Monitoring
Synopsis
- Use T-cell repertoire to determine HLA type for high-risk alleles
- Monitor-T cell response to immune modifying therapies as a biomarker of activity
- Map T-cell clones to self-antigens to identify disease targets and track antigenspecific response to therapy
10:30 am
Speed Networking & Morning Break
Synopsis
This session is the ideal opportunity to network and forge meaningful business relationships with many of the brightest minds working in the B & T cell field, face to-face for the first time
11:00 am The InhiBETTM Platform: Bromodomain and End Terminal (BET) Inhibitors in Autoimmune Disease
Synopsis
- Review BET protein antagonism and MoA in autoimmune disease, what have we learned?
- Explore drug design and disposition strategies to optimize benefit/risk of BET inhibitors
- Introducing VYN201, a highly potent BD1/BD2 BET protein inhibitor, for locallyacting treatment of major immuno-inflammatory
Highlighting T-Cell Interactions & Nanoparticle-Based Interactions for Immune Tolerance
11:30 am Precision Medicine for Autoimmunity – Mechanism of Action for CAAR T cell Therapy and Applications to B Cell-Mediated Autoimmune Diseases
Synopsis
- Review mechanism of action for the CAAR T platform that is designed to recognize and eliminate only the B cells that cause the autoimmune disease in patients
- Discuss progress in DesCAARTes trial of DSG3-CAART and preclinical development for select pipeline programs
- Share rationale for indication prioritization and along with preclinical development path for new product candidates
12:00 pm A Novel NanoDisc Platform for Induction of Antigen-Specific Immune Tolerance
Synopsis
- Examine how subcutaneous administration of NanoDisc targets multiple lymphoid tissues and immune system organs
- Discover how NanoDisc carrying autoantigens induce antigen-specific T-Regs and Tr1 cells, while reducing autoreactive IgG response
- Learn how NanoDisc therapy exerts robust efficacy in preclinical models of Multiple Sclerosis and Type 1 Diabetes
12:30 pm Manipulation of Apoptosis for Induction of Antigen-specific T-regs to Treat Autoimmunity
Synopsis
- Inducing specific immunotherapy to autoimmunity without compromising overall immune responses
- Explore how to induce autoantigen-specific Tregs in animals, and ultimately in patients with established autoimmune disease
- Learn about the manipulation of the apoptosis of immune cells with antibodies and nanoparticles together with administration of autoantigens
1:00 pm
Lunch & Networking
2:00 pm Synergistic activity of IL-2 mutein with tolerogenic ImmTOR nanoparticles with leads to massive expansion of antigen-specific Tregs
Synopsis
- Engineered IL-2 molecules selective for the high affinity IL-2 receptor expressedon Tregs have been shown to non-selectively expand Tregs in vivo and are being developed for the treatment of autoimmune diseases
- We have developed tolerogenic nanoparticles that have been shown to induce antigen-specific immune tolerance and are in Phase 3 clinical trials in
combination with a highly immunogenic fungal-derived uricase enzyme for the treatment of uncontrolled gout - Here we demonstrate that ImmTOR combined with a Treg-selective IL-2 molecule shows profound synergistic activity in animal studies resulting in massive expansion of antigen-specific Tregs
2:30 pm A nanomedicine approach to treat autoimmune disease leverages a natural differentiation pathway of T regulatory-1 (TR1) cells.
Synopsis
- T-follicular helper (TFH) cells can serve as in vivo precursors of TR1 cells in humans and in mice
- Complete differentiation of TR1 effector function requires the transcription factor Blimp-1 (Prdm1)
- Navacims, a pMHC-II nanomedicine platform induce TFH expansion and differentiation to immunomodulatory TR1 cells
3:00 pm A nanoparticle platform for multiple immune tolerance therapies
Synopsis
- Broad therapeutic potential of our proprietary Topas Particle Conjugates delivering peptide antigens to special liver cells
- Diverse mechanism-of-action through different immune pathways to rebalance the immune system
- Pemphigus vulgaris and Celiac disease as strong clinical models
3:30 pm
Afternoon Break
Comparing the Efficacy of Small Molecule Targeting on Signaling Pathways & their Impact on Autoimmune Diseases
4:00 pm Investigating GB7208: An Oral, CNS-Penetrant, Irreversible BTK inhibitor for the Treatment of Neuroinflammatory Diseases
Synopsis
- Learn how BTK inhibitors are actively being explored in inflammatory diseases
- Discover how they are characterized by modest selectivity and/or limited central nervous system (CNS) penetrance
- Assess GB7208, an orally available, selective, irreversible small molecule BTK inhibitor which has been selected based on its potency, specificity, and high brain penetrance in preclinical models
4:30 pm Exploring NX-5948: A CNS Penetrant Selective Degrader of BTK that Significantly Reduces Inflammation in Mouse Models of Autoimmune Disease
Synopsis
- Uncover NX-5948, a highly potent, CNS penetrant, targeted protein degrader of BTK in Phase 1 clinical development for B cell malignancies
- Evaluate how daily oral administration of NX-5948 provides efficacy in multiple preclinical models of autoimmune disease
- Learn how NX-5948 preclinical data supports further exploration of NX-5948 to treat autoimmune diseases
5:00 pm Cell Therapy Approaches for the Treatment of Autoimmune Diseases
Synopsis
- Discovering how cell therapy approaches offer the potential to transform the care of patients with refractory autoimmune diseases
- Review how cells can be engineered to either delete (e.g. car t approaches) or suppress the autoreactive immune system (e.g. Tregs approaches)
- Exploring how the background of patients with autoimmune disease differs from oncology patients, and how this influences their approach when being introduced in the clinic