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8:20 am Chair’s Opening Remarks

The Third Access: Successfully Inducing Tolerance

8:30 am Checkpoint Inhibitors: Insights into Immune Tolerance

  • Jack Ragheb Senior Medical Fellow for Immunology in Global Patient Safety, Eli Lilly & Co.

Synopsis

  • Review of immune ontogeny and the establishment of immune tolerance
  • Checkpoint inhibitors and their safety profile
  • What Checkpoint inhibitors teach us about the maintenance of immune tolerance

9:00 am Exploring the Parameters by which Tolerance Induction is Determined

Synopsis

  • Antigenic tolerance would lead to less systemic side effects and quasi site specific suppression of autoimmunity
  • Evaluate the lack of reliable markers to assess successful reduction of antigenic responses in humans
  • Understanding the rules by which tolerance might be optimally induced and
    there are many unsubstantiated claims in this research space

9:30 am Translational & Clinical Development of Novel, Liver Targeted Immune Tolerance Therapies for Celiac Disease & Multiple Sclerosis

Synopsis

  • Restoration of normal immune tolerance using antigen mediated immune tolerance approaches
  • Monitoring of immune tolerance induction via multiple scientific and clinical laboratory methods

9:50 am Morning Break & Refreshments

10:50 am Immune Tolerance Induction via Allogeneic Exosomes

  • Giles Benichou Associate Professor of Surgery, Harvard Medical School, Senior Immunologist, Massachusetts General Hospital

Synopsis

  • Allograft tolerance via MHC chimerism achieved with donor exosomes
  • Restoration of tolerance in mice with ongoing EAE via mixed hematopoietic chimerism and neural stem cell therapy
  • Prevention of relapses in mice with EAE using allogeneic exosomes

11:20 am T Cell Anergy Induction by Pharmacological Agents Partially Restricting T Cell Activation

  • Garth Ringheim Director Immunology Therapeutic Area, Clarivate Analysis

Synopsis

  • Inducing antigen tolerance in T cells by modulating select T cell receptor signaling pathways
  • Functional measures in the lab and the clinic to monitor T cell activation and anergy
  • Mechanisms regulating T cell anergy and the potential for developing new drugcandidates

11:50 am Establish Tolerance in MS with Myelin-Peptide Coupled Red Blood Cells

Synopsis

  • Successful development of GMP manufacturing process for peptide coupled cells and clinical implementation
  • Clinical safety and immunological proof-of-concept data of phase Ib trial
  • Challenges and pitfalls in implementing clinical PoC in phase II development

Tolerance Induction in Parallel Fields: What Lessons can be Applied for Autoimmune Diseases

12:50 pm Antigen Specific Tolerance in Autoimmunity & in Gene Therapy

  • Lawrence Steinman Professor of Neurology, Neurological Sciences & Pediatrics, Stanford University

Synopsis

  • Description of the engineered DNA platform for tolerance
  • Pre-clinical results in gene therapy of Duchenne’s Muscular Dystrophy
  • Status of Clinical Trials in Type 1 diabetes, myasthenia gravis and neuromyelitis optica

1:00 pm Lunch & Networking

2:00 pm Antigen-Specific T & B Cell Profiles as Therapeutic Targets in Allergy & Autoimmune Disease

Synopsis

  • High-dimensional phenotyping identifies distinct antigen-specific T and B cell profiles using peripheral blood from patients with allergy and autoimmune disease
  • Deletion, deviation, anergy, and exhaustion are potential tolerogenic outcomes of therapy
  • Tracking antigen-specific T and B cells during therapy may be a surrogate for
    clinical response

2:30 pm Unraveling the Mechanism of Desensitization & Tolerance through Food Allergen-Specific Oral Immunotherapy

  • Monali Manohar Life Science Research Scientist, Stanford University School of Medicine

Synopsis

  •  Novel insights into OIT-induced changes in allergen-specific CD4+ T cells favoring desensitization and tolerance
  • The role of anti-IgE adjunct treatment in allergen-specific OIT regimen to facilitate immune changes leading to desensitization
  • Pivotal role of non-specific immune cell subsets in shaping allergen-specific immune response during OIT

What Assays do we use & when? Developing Assays for Clinical Trials

3:00 pm FlowSpot: A Single Platform Assay with Broad Versatility for Measuring Cellular & Humoral Immune Status, Responsiveness & Memory

  • Dolly Tyan Professor; Emerita of Pathology, Stanford University School of Medicine

Synopsis

  • Overview of the assay design
  • Profiling and measuring T cell and antigen specific BCR (memory B cell) responses
  • Assessing responsiveness to antigenic challenges and immunosuppressive drugs
  • Determining transplant compatibility and predicting GVHD

3:30 pm Assay Development for AKS-107, an Insulin-Fc Biologic Designed to Target Insulin-Specific B Cells in Type 1 Diabetes (T1D)

Synopsis

  • Insulin-specific B cells promote T1D pathogenesis by acting as antigen-presenting cells that activate pathogenic effector T cells
  • Clinical insulin-specific B cell assays are being developed for patient inclusion and therapeutic response monitoring
  • Overcome the funding challenges of bringing a novel T1D immunotherapeutic from research to a first-in-human clinical safety trial to enable optimal positioning for subsequent Phase II proof-of-concept trials via T1D consortia

4:00 pm Afternoon Refreshments & Networking

Exploring Clinical Opportunities to use Combination Strategies to Successfully Deliver Tolerance Inducing Therapeutics

4:30 pm Considerations for Dual Therapy Approaches in Autoimmune Disease

  • Joshua Sestak President and Chief Executive Officer, Founder, Orion BioScience

Synopsis

  • Where the need for dual therapy is greatest
  • The role of safety in pursuing multiple therapeutic approaches
  • How to best leverage safety and efficacy in dual therapy

5:00 pm Novel Applications of Localized Delivery of Combination Strategies for Modulation of Allo- & Auto-Reactivity

  • Giorgio Raimondi Associate Scientific Director for Transplant Immunology, John Hopkins School of Medicine

Synopsis

  • A novel rationale for immunomodulation via combination strategy
  • The use of “injectable hydrogels” for localized modulation of alloreactivity
  • The use of “lipid nanoparticles” for orally-based modulation of autoreactivity

5:30 pm Regulatory Perspective on Combination Therapies

Synopsis

  • Details to be confirmed

6:00 pm Panel Discussion: “Bridging the Valley of Death: Non-Profit Organizations as Enablers of Novel Commercial T1D Therapeutics from Research to Phase II”

  • David Alleva Executive Director, Akston Biosciences
  • Todd Zion President & Chief Executive Officer, Akston Biosciences
  • Ben Williams Program Officer for the Type 1 Diabetes (T1D) Program, Helmsley Charitable Trust
  • Kurt Griffin Associate Professor , University of South Dakota Sanford School of Medicine

Synopsis

  • Explore what role non-profit organizations can play in addressing the key funding acquisition problems of pre-clinical and clinical development
  • From concept to action: A non-profit funding case study
  • Evaluating the scope of current non-profit funding programs and where best to place resources along the development stage

6:30 pm Chair’s Closing Remarks