Identifying and Establishing Biomarkers for Complex Rare Diseases

Time: 1:00 pm - 4:00 pm
day: Workshop H


Establishing endpoints in gene therapy is always top of mind when designing clinical trials, for which biomarkers can be a highly useful indicator. This workshop will delve into how to better identify appropriate biomarkers in different indications which could be used to define clinical endpoints that demonstrate efficacy and safety.

Attend this workshop to learn about:

  • Validating biomarkers to contribute to defining clinical endpoints
  • Balancing standard biomarkers and novel ones – time versus effectiveness
  • Predicting real-time efficacy with the patient journey at the centre