TABLE 3.
Working Group 3: scientific priorities and implementation strategies for acquiring an actionable understanding of FVIII immunogenicity and the immunology of both the host immune response and tolerance
Co-chairs | Shannon Meeks, MD and Roland Herzog, PhD |
Charge | Basic and translational research to elucidate an actionable understanding of FVIII immunogenicity and the immunology of both the host immune response and tolerance to inform predictive models for inhibitor development and novel therapeutic targets |
Goals | Application of novel ideas, technologies and cross-disciplinary science to these studies • Consideration of the role of host cell expression of FVIII/VWF in immunogenicity as well as gene therapy as a model for FVIII immune tolerance • Identification of appropriate animal and ex vivo models for immunogen expression and peptide generation • Human biospecimens required to further research • Challenges associated with and successful models for conducting cross-disciplinary science as well as resources and partners required for success • Optimization of private-public partnerships in basic/translational research • Training models and opportunities engendered by novel cross-disciplinary science |