Understanding how the Unfolded Protein Response Can Lead to Waning Durability

Time: 1:05 pm
day: Pre-Conference Day


Participate in this collaborative session to understand the complex landscape of the unfolded protein response and discuss the latest developments in strategies to mitigate the unfolded protein response. Attend this workshop to learn about:

• Understand how the unfolded protein response can be linked to reduced durability in a Hemophila A gene therapy trial

• Understand how to utilize second generation gene therapy to specifically address the unfolded protein response

• Explore how editing the amino acid sequence of a factor VIII transgene leads to easier folding and release of the protein into the blood stream

• How to monitor the unfolded protein response in clinical trials thorough biomarkers and biopsy specimens