Freeline Moves Closer to Identifying Hemophilia B Gene Therapy Dose
A second cohort has been dosed with 7.7e11 vg/kg to hopefully confirm the dose for phase 3 trials.
Gene Therapies for Hemophilia Have Positive Real-World Impact, Studies Show
Presentations at ISTH 2021 revealed attributes most important to patients when considering gene therapies and where further research is needed.
Hemophilia Gene Therapy Registry Launched to Monitor Long-Term Outcomes
The World Federation of Hemophilia’s registry will include long-term safety and efficacy data in people with hemophilia treated with gene therapies.
Fidanacogene Elaparvovec Well-Tolerated in Hemophilia B
No concerning effects to liver health were observed in the phase 1/2a study.
Hemophilia B Gene Therapy AMT-060 Shows Durable Effect
Phase 3 studies are continuing in the enhanced Padua FIX variant AMT-061 for hemophilia B.
Valoctocogene Roxaparvovec’s Treatment Durability in Hemophilia A in Question
A drop in endogenous Factor VIII expression was observed from treatment to 5-year follow-up despite continued demonstration of efficacy.
Valoctocogene Roxaparvovec Efficacious in Treating Hemophilia A
Over 90% participants had an annualized bleeding rate of 0 or a lower bleed rate than baseline after week 4 after treatment.
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