Heghine Khachatryan: Navigating the New Era of Hemophilia Gene Therapy
Heghine Khachatryan, Editor-in-Chief of Hemostasis Today, Head of Hemophilia and Thrombosis Center at Yeolyan Hematology and Oncology Center, shared a post on LinkedIn about a recent article by Pascal Hoffmann et al, published in Journal of Thrombosis and Haemostasis.
“A timely and highly relevant overview on gene therapy in hemophilia.
As we move from substitution therapy toward potential functional cure, the key challenge is no longer innovation itself—but how we implement it safely, equitably, and sustainably in real-world practice.
Important questions remain:
patient selection, long-term safety, durability of expression, and global accessibility.
This article is a valuable step toward structured, evidence-based navigation of this new era.”
Journal of Thrombosis and Haemostasis (JTH) shared a post on LinkedIn:
“Overview of gene therapy for hemophilia: questions and answers to navigate the innovation
Gene therapy transforms hemophilia treatment through potentially one-time interventions targeting genetic deficiencies, achieving sustained factor expression and reduced bleeding with AAV-based approaches, though immunosuppressive management remains necessary for post-infusion complications.”
Title: Overview of gene therapy for hemophilia: questions and answers to navigate the innovation
Authors: Pascal Hoffmann, Niccolò Bitto, Vincenzo La Mura, Wolfgang Miesbach
Read the Full Article on Journal of Thrombosis and Haemostasis
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