July 11th 2022
A second cohort has been dosed with 7.7e11 vg/kg to hopefully confirm the dose for phase 3 trials.
August 25th 2021
Presentations at ISTH 2021 revealed attributes most important to patients when considering gene therapies and where further research is needed.
August 12th 2021
The World Federation of Hemophilia’s registry will include long-term safety and efficacy data in people with hemophilia treated with gene therapies.
August 6th 2021
No concerning effects to liver health were observed in the phase 1/2a study.
August 4th 2021
Phase 3 studies are continuing in the enhanced Padua FIX variant AMT-061 for hemophilia B.
July 27th 2021
A drop in endogenous Factor VIII expression was observed from treatment to 5-year follow-up despite continued demonstration of efficacy.
July 20th 2021
Over 90% participants had an annualized bleeding rate of 0 or a lower bleed rate than baseline after week 4 after treatment.