Pre-Conference Workshop Day

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

Workshop A - Companion Diagnostics for AAV Gene Therapies 101
| Tuesday, August 3 | 9:00 -11:00 EDT |

When used with gene therapy, companion diagnostics (CDx) can help inform treatment decisions. In the case of gene therapies, CDx can help identify patients who are likely to benefit from therapy or those likely to experience treatment-related adverse events. These tools may facilitate the monitoring of treatment response, enabling healthcare providers to adjust therapy and achieve improved safety or effectiveness. Current regulatory guidance recommends the development of CDx assays for gene therapy and were updated to use CDx not only for safety but for efficacy as well. In light of this, the workshop will set out to cover:

  • Development and submission of companion diagnostics to evaluate pre-existing antibodies
  • Best practice for establishing a partnership with a CRO for your companion diagnostic
  • Do regulators want you to have a CDx?
  • How to navigate an increasingly fluid regulatory environment for CDx development and submission
  • How much will it cost to develop a CDx?
  • Where and how do companion diagnostics fit into the reimbursement system - who covers the cost?
Workshop A

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