Andjelka Stones/Pinterest
Jun 5, 2026, 15:53
Andjelka Stones: Key Findings on Hemophilia A Gene Therapy
Andjelka Stones, Former Distinguished Adjunct Professor at NorthCentral University, shared a post on LinkedIn:
“Hemophilia A Gene Therapy
Gene therapy for hemophilia A: 4‑year data from the Phase 3 GENEr8‑1 trial are out.
Key findings (n=134):
- Mean treated bleed rate 82.6% from baseline
- Factor VIII infusion rate 95.5%
- 73.6% of patients had zero treated bleeds in year 4
- Mean FVIII activity at year 4: 16.1 IU/dL
- Only 18% of patients resumed prophylaxis
No new safety signals were observed. Health‑related quality of life remained improved.
This is the longest and largest Phase 3 follow‑up of a gene therapy in hemophilia.
Durable benefit after a single infusion.
Full citation in my archive.”
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