Pre-Conference Workshop Day

Tuesday, August 3 2021 | 9am - 4pm EDT

Workshop B - Predicting Immune Response to Gene Therapy Delivery
| Tuesday, August 3 | 11:30 -1:30 EDT |

Predicting the immune response of patients after delivering a gene therapy product stands as one of the greatest challenges drug developers continue to try and tackle. Limited pre-clinical models mean prediction of immune response prior to treatment in an accurate manner is particularly challenging. This workshop will explore:

  • Whether we can predict immune response in patients prior to treatment, based on their cellular reactivity
  • Employing in vitro and in silico immunogenicity tools to predict immune response
  • Developing pre-clinical models that effectively predict human immune respone to vectors and transgenes
  • How best to integrate prediction technologies and identify the most appropriate prediction tool for each stage of drug development
  • A combination approach: using immunogenicity prediction results alongside other immune related factors in order to better inform your prediction
  • Creating animal models that can effectively repeat the effects of capsid structure, DNA conformation, and immunomodulatory regimens on capsid antigen presentation and T-cell responses
workshop B

Workshop C - Exploring the Role of Complement Activation in Immunogenicity & Complement Specific Mitigation Strategies
| Tuesday, August 3 | 2:00 - 4:00 EDT |

A major obstacle to using recombinant adenoviral vectors in gene therapy is the natural ability of the human adenovirus to activate the classical and alternate complement pathways. These innate immune responses contribute to hepatic adenoviral uptake following systemic delivery and enhance the humoral immune responses associated with adenoviral infection. Consequently this workshop will set out to explore:

  • Current understanding of complements role in immune response to AAV gene therapy delivery
  • Complement mediate immunotoxicities with higher vector doses
  • Complement specific mitigation strategies for immunogenicity modulation
workshop C