International Society on Thrombosis and Haemostasis (ISTH)

A second cohort has been dosed with 7.7e11 vg/kg to hopefully confirm the dose for phase 3 trials.

Presentations at ISTH 2021 revealed attributes most important to patients when considering gene therapies and where further research is needed.

The World Federation of Hemophilia’s registry will include long-term safety and efficacy data in people with hemophilia treated with gene therapies.

No concerning effects to liver health were observed in the phase 1/2a study.

Phase 3 studies are continuing in the enhanced Padua FIX variant AMT-061 for hemophilia B.

A drop in endogenous Factor VIII expression was observed from treatment to 5-year follow-up despite continued demonstration of efficacy.

Over 90% participants had an annualized bleeding rate of 0 or a lower bleed rate than baseline after week 4 after treatment.