Collaborative Workshop hosted by the Centers of Excellence in Regulatory Science and Innovation (CERSIs) and the Food and Drug Administration (FDA)

Full Agenda

Wednesday, October 3, 2018: 8:30 a.m. - 5:00 p.m.
Thursday, October 4, 2018: 8:30 a.m. - 5:00 p.m.

FDA's White Oak Campus
10903 New Hampshire Avenue
Building #31 - The Great Room
Silver Spring, MD 20903
PDF iconView the full event agenda online.
Goals and Objectives: 
Protein therapeutics have continued to gain an increasing share of the pharmaceutical market and now provide medical interventions for some of the most complex and intractable diseases. Immunogenicity, the propensity of a therapeutic protein to induce immune responses, principally by development of antibodies, may affect safety and/or efficacy, and is thus an important concern in the development and regulation of protein therapeutics.  Patients, regulators and the biopharmaceutical industry are all affected by immunogenicity.  Recently, both the US Food and Drug Administration and the European Medicines Agency have released Guidance Documents for industry focusing on immunogenicity.
This timely public workshop will present advances in the development of technological tools for predictions of immunogenicity and discussions on how to engage relevant stakeholders in application of these tools during drug development.
The specific objectives of this workshop are to:
  • Communicate to stakeholders the “state of the science” regarding technological approaches for prediction of immunogenicity including non-clinical (in silico) studies for detection of neo-antigens, the use of novel animal models, and strategies to de-immunize protein molecules. 
  • Explore strategies for choosing appropriate tools and interpreting the results since the novel tools and information available in this field are complex and not amenable to simple prescriptive approaches during drug development.


For speakers who have given permission, the workshop slides are posted below on this webpage (see hyperlinks). For speakers who have given permission, the workshop webcast recordings will be posted in the near future on this webpage.


Wednesday, October 3rd



8:30 - 8:45 a.m.

About the FDA and M-CERSI Partnership
Welcome & Session 1 Recording

G. Caleb Alexander, M.D. (Johns Hopkins University)

James Polli, Ph.D.
(University of Maryland School of Pharmacy)

8:45 - 9:00 a.m.

Opening Remarks from the FDA Center for Drug Evaluation and Research (CDER)

Patrizia Cavazzoni, M.D. (CDER)

9:00 - 9:15 a.m.

Opening Remarks from the FDA Center for Biologics Evaluation and Research (CBER)

Carolyn Wilson, Ph.D. (CBER)

Session 1
Advances in understanding the biology of T- & B-cell responses as it applies to the immunogenicity of therapeutic proteins
Moderator: Amit Golding, M.D., Ph.D. (University of Maryland School of Pharmacy)

9:15 - 9:20 a.m.


Dr. Amit Golding

9:20 - 10:00 a.m.

Interplay of Cells involved in Therapeutic Agent Immunogenicity

Robert G. Hamilton, Ph.D. (Johns Hopkins University School of Medicine)

10:00 - 10:25 a.m.

Coffee Break


Session 2
New analytical techniques and improvements in existing technologies to predict immune response to therapeutic proteins
Session 2 Recording
Moderator: Daniela Verthelyi, M.D., Ph.D. (OPQ/CDER/ FDA)

10:30 - 11:00 a.m.

MHC binding and immunogenicity of eluted ligands - benchmarking and predictions

Alessandro Sette, Dr. Biol. Sci. (La Jolla Institute for Allergy and Immunology)

11:00 - 11:30 a.m.

Use of risk assessment tools during preclinical development to drive a clinical immunogenicity strategy

Vibha Jawa, Ph.D. (Merck and Co. USA)

11:30 - 12:00 p.m.

The ABIRISK integrated approach to identify and evaluate predictive markers of immunogenicity

Sophie Tourdot, Ph.D. (Pfizer Inc.)

12:00 - 1:10 p.m.



Session 3
The human cost and the economic burden: How immunogenicity affects patients and the economic burden on the healthcare system
Sessions 3 and 4 Recording
Moderator: Frank F. Weichold, M.D., Ph.D. (FDA)

1:10 - 1:15 p.m.


Dr. Frank F. Weichold

1:15 - 1:45 p.m.

Immunogenicity economic impacts from gene therapy development to biosimilar markets: inconvenient biology truths for all

Mark Trusheim (MIT)

1:45 - 2:15 p.m.

A patient-centered approach to understanding the burden of inhibitors

Mark W. Skinner, J.D. (Institute for Policy Advancement Ltd)
Session 4
Mathematical models that can integrate seemingly disparate measurements related to immunogenicity
Moderator: Million A. Tegenge, RPh, Ph.D. (CBER/FDA)

2:15 - 2:20 p.m.


Dr. Million A. Tegenge

2:20 - 2:50 p.m.

The development of an Immunogenicity Simulator through a quantitative systems pharmacology consortium approach

Piet H. van der Graaf, Pharm.D., Ph.D. (Certara QSP)

Andrzej M. Kierzek, Ph.D. (Certara QSP)

2:50 - 3:20 p.m.

Do differences make a difference: human immune responsiveness and single-cell variations

John Tsang, Ph.D. (NIH)

3:20 - 3:40 p.m.



Session 5
Panel Discussion with Day 1 Speakers
Session 5 Recording
Moderator: Zuben E. Sauna, Ph.D. (CBER)

3:40 - 4:45 p.m.

Expert Panel

Dan Sikkema, Robert Hamilton, Alessandro Sette, Vibha Jawa, Sophie Tourdot, Mark Trusheim, Mark Skinner, Piet van der Graff, John Tsang

4:45 - 5:00 p.m.

Summary of Day 1 & Close

Dr. Zuben E. Sauna




Thursday, October 4th


Session 6
Big data and immunogenicity: How “omics” workflows can be used to generate and analyze large data sets as they relate to immunogenicity
Welcome & Session 6 Recording
Moderator: Joseph McGill (FDA/OMPT/CBER/OTAT/DPPT/HB)

8:35 - 8:40 a.m.


Joseph McGill

8:40 - 9:10 a.m.

Tonsil organoid cultures as a model system to study human adaptive immune responses in vitro

Mark M. Davis, Ph.D. (Stanford)

9:10 - 9:40 a.m.

Massively parallel screening of natively paired human antibody repertoires for antibody response analysis

Brandon J. DeKosky, Ph.D. (University of Kansas)

9:40 - 10:10 a.m.

Lessons Learned from the MHC II Immunopeptidome

Laura Santambrogio, M.D., Ph.D. (Albert Einstein College of Medicine)

10:10 - 10:25 a.m.



Session 7
Identification and application of biomarkers for predicting immunogenicity
Sessions 7 & 8 Recording
Moderator: João A. Pedras-Vasconcelos, Ph.D. (FDA/CDER/OBP/DBRR3)

10:25 - 10:30 a.m.


Dr. João A. Pedras-Vasconcelos

10:30 - 11:00 a.m.

De-risking Protein Therapeutics: Should You Delete that T cell Epitope or Keep it? And Why

Annie De Groot, M.D. (University of Rhode Island)

11:00 - 11:30 a.m.

Amplitude and peptidespecificity of CD4 T-cell response to therapeutic antibodies in healthy donors and in patients

Bernard Maillere, Ph.D. (University Paris-Saclay, France)

Session 8
Update NHLBI State of the Science Workshop: Inhibitors in Hemophilia

Moderator: Basil Golding, M.D. (DPPT, OTAT/CBER/FDA)

11:30 - 11:35 a.m.


Dr. Basil Golding

11:35 - 12:05 p.m.

Factor VIII Inhibitors: Generating a National Blueprint for Future Research

Donna DiMichele, M.D. (NIH)

12:05 - 1:05 p.m.



Session 9
The Holy Grail: Deimmunizing protein therapeutics
Session 9 Recording
Moderator: Amy S. Rosenberg, M.D. (CDER)

1:05 - 1:10 p.m.


Dr. Amy S. Rosenberg

1:10 - 1:40 p.m.

Recombinant Immunotoxins: Efficacy and Immunogenicity

Ira Pastan, M.D.

1:40 - 2:10 p.m.

Computationally-driven deimmunization of therapeutic proteins

Chris Bailey-Kellogg, Ph.D. (Stealth Biologics, LLC)

2:10 - 2:40 p.m.

Toward de novo design of immune silent protein and peptide therapeutics

Lance J. Stewart, Ph.D. (University of Washington)

2:40 - 3:00 p.m.



Session 10
Panel Discussion with Day 2 Speakers
Panel Discussion Recording


3:00 - 4:45 p.m.

Expert Panel

Mark Davis, Brandon DeKosky, Laura Santambrogio, Anne DeGroot, Bernard Maillere, Ira Pastan, Chris Bailey-Kellogg, Lance Stewart, Donna DiMichele

4:45 - 4:55 p.m.

Closing Remarks

Dr. Amy S. Rosenberg 

4:55 - 5:00 p.m.

Acknowledgements & Wrap-up Declaring the workshop closed

Dr. Zuben E. Sauna


** Collaborative Workshop hosted by:

Centers of Excellence in Regulatory Science and Innovation (CERSIs):
Johns Hopkins University
University of Maryland
The Food and Drug Administration:
Center for Drug Evaluation and Research (CDER)
Center for Biologics Evaluation and Research (CBER)
Office of Chief Scientist, Office of Regulatory Science and Innovation (OCS/ORSI)