Coming 2019

Day One
Wednesday April 25, 2018

Day Two
Thursday April 26, 2018

Morning Coffee & Networking

Chair’s Opening Remarks

  • Amy S. Rosenberg Supervisory Medical Officer and Division Director, Office of Biotechnology Products, CDER/FDA

Surging Translational Potentials of Pre-Clinical Antigen-Specific Therapies

Pathology of Human Type 1 Diabetes and New Antigenic Interventions

  • Matthias von Herrath Professor at the La Jolla Institute for Allergy and Immunology; VP & Head of Type 1 Diabetes R&D Center Seattle, Novo Nordisk


  • Type 1 Diabetes might be relapsing remitting in some patients, therefore continuous treatment like an antigenic tolerance regimen might be needed
  • We have found many published antigenic interventions in mouse models not to be robust enough
  • Continuous treatment with antigen and suitable response modifiers might be needed for establishing beta cell specific tolerance. Combination treatments might also be needed

Case Study: Non-Clinical Assessments of Immunogenicity

  • Wojciech Jankowski Research Reviewer, Center for Biologics Evaluation & Research, CBER/FDA


  • The importance of immunogenicity during drug development and overview of regulatory Guidance Documents and White Papers that are available
  • Two important concepts in immunogenicity risk assessment (with examples): (i) The ability to distinguish between validating a specific method and predicting clinical immunogenicity. (ii) Estimating the probability of anti-drug development vs. the consequences if such antibodies are generated
  • Engineered protein therapeutics have neo-sequences introduced into them. How can one assess whether these may be neo-epitopes? If so, how does one mitigate the risk prior to starting clinical trials?

Case Study: Nanoparticles for the Induction of Antigen-Specific Tolerance


  • Mechanisms that control tolerogenic DCs
  • Nanoparticles for the induction of tolerance
  • Translational potential

Case Study: Targeting Insulin-Specific B Cells To Prevent Type 1 Diabetes

  • Todd Zion President & CEO, Akston Biosciences


  • Akston has developed a novel, modified insulin capable of deleting high affinity insulinspecific B cells and disrupting insulin-specific antigen presentation, while leaving the rest of the B cell repertoire untouched
  • The lead candidate, AKS-107, prevents the development of Type 1 Diabetes in nonobese diabetic (NOD) mice with lasting effects after cessation of dosing. AKS-107- treated NOD mice confer disease protection after adoptive lymphocyte transfer
  • Studies in non-human primates demonstrate the safety of AKS-107 and its long serum half-life supporting q2w dosing. The compound is now being developed under good manufacturing practice (GMP) to support first-in-human clinical trials

Case Study: Harnessing Natural Tolerance Pathways with Targeted Delivery Technologies Induces Robust Antigen-Specific Tolerance


  • Mimicking physiological tolerance mechanisms by way of active targeting domains induces antigen-specific T cell tolerance
  • Our technologies are translational, in that the mechanisms induced are consistent with unmet clinical needs (T cell deletion and regulation), and our molecules are developable
  • Discuss potential approaches to de-risk MoA’s in higher-order species, and consider their value

Morning Refreshments & Networking

Optimizing Clinical Development & Paving the Future of Antigen-Specific Immune Tolerance Therapies

Immune Tolerance Induction: Clinical Urgency as a Factor in Dictating Approach

  • Amy S. Rosenberg Supervisory Medical Officer and Division Director, Office of Biotechnology Products, CDER/FDA


  • Rapid induction of tolerance may require non-antigen specific approaches
  • Prophylactic tolerance induction requires less duration and intensity of immune suppression
  • Rapid induction of antigen-specific immune tolerance requires consideration of combination therapies

Case Study: Pre-clinical & Clinical Development of Tolerogenic Nanoparticles to Mitigate Immunogenicity Against Biologic Drugs


  • We have developed biodegradable Synthetic Vaccine Particles containing rapamycin (SVP-Rapamycin) that induce tolerogenic dendritic cells and antigen-specific regulatory T cells
  • SVP-Rapamycin has been shown to mitigate immunogenicity against a wide variety of biologic drugs, including adalimumab in a model of inflammatory arthritis, coagulation Factor VIII in haemophilia A mice, acid alpha glucosidase in a model of Pompe Disease, immunotoxin in a model of mesothelioma, pegylated uricase in uricase deficient mice, and AAV gene therapy vectors
  • SVP-Rapamycin is currently in Phase 2 clinical trials with pegsiticase, a pegylated uricase enzyme, in patients with symptomatic gout and hyperuricemia

Case Study: Antigen- Specific Tolerance for Type 1 Diabetes, Neuromyelitis Optica, Myasthenia Gravis and Protein Replacement Therapy

  • Lawrence Steinman Professor at Stanford University & Executive Chairman of the Board, Tolerion


  • Clinical Efficacy in Phase 2a trial of type 1 diabetes when tolerizing to proinsulin in type 1 diabetes
  • Promising pre-clinical results in neuromyelitis optica, myasthenia gravis
  • Promising pre-clinical results in gene therapy of Duchenne Muscular Dystrophy

Networking Lunch

Commercialization: Future Proofing Immune Tolerance Therapies for Patients

Panel Discussion: How to Develop Transferable and Durable Antigen-Specific Immune Tolerance Therapies?


  • How to deal with the complex human immune system?
  • How to select precise subpopulations and build expandability to entire disease target?
  • How to ensure safety and efficacy?

Panel Discussion: How to Develop Commercially Viable Antigen-Specific Immune Tolerance Therapies?

  • Daniel Rotrosen Director, Division of Allergy, Immunology & Transplantation, NIAID/NIH
  • Lachy McLean VP, Head of Clinical & Translational Sciences, Immunology, Takeda Pharmaceuticals
  • Arpita Maiti Senior Director, External Science & Innovation, Inflammation, Immunology and Microbiome, Pfizer
  • Matthias von Herrath Professor at the La Jolla Institute for Allergy and Immunology; VP & Head of Type 1 Diabetes R&D Center Seattle, Novo Nordisk


  • What learnings can be leveraged from other non-traditional modalities to develop antigen-specific tolerogenic therapies?
  • How to anticipate chemistry, manufacturing, and controls (CMC) hurdles?
  • What are some of the advantages of the these therapies in the context of manufacturing?

Chair’s Closing Remarks & End of the Summit, Followed by Afternoon Refreshments & Networking

  • Amy S. Rosenberg Supervisory Medical Officer and Division Director, Office of Biotechnology Products, CDER/FDA