Engineering Payload to Increase Expression Durability & Lower Doses
Time: 3:30 pm
day: Conference Day One
Details:
- Highlighting current developments and challenges in AAV gene therapy for Hemophilia A
- Emphasizing how engineering the FVIII protein to generate hyperfunctional mutants could enhance expression durability and improve therapeutic efficacy
- Demonstrating an efficient approach to generate hyperfunctional transgenes, enabling lower therapeutic doses and improved durability compared to firstgeneration AAV gene therapies
