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Andjelka Stones: Key Findings on Hemophilia A Gene Therapy
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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