Listen to Glenn Pierce, MD, PhD and Steve Pipe, MD discuss the latest gene therapy information that was presented at the ISTH 2021 Congress.
To skip to a specific question, please use the timestamps below:
- 01:25 Variability of expression between patients
- 03:00 Range of expression
- 03:40 Durability of expression
- 07:05 Can we predict which patients will do well on gene therapy, and which won’t?
- 10:44 Correlation between expression levels and bleed protection
- 12:48 Short-term safety
- 14:50 Long-term safety
- 16:39 HOPE-B hepatocellular carcinoma case