Samuele Takeshita: Scientific and Practical Aspects of Gene Therapy Access in Hemophilia and SCD
Samuele Takeshita, Head of Sales and Business Development at ELIXI International, shared a post on LinkedIn:
“A highly suggested reading to capture both the scientific and practical aspects of gene therapy access in hemophilia and sickle cell disease (SCD).
It explains how gene therapy science differs between viral vector‑based and ex vivo cell‑based approaches and their clinical implications.
In hemophilia, adeno‑associated viral (AAV) vectors deliver functional factor VIII or IX genes but can trigger inflammatory hepatotoxicity, requiring corticosteroid management.
In SCD, autologous hematopoietic stem cells are modified ex vivo (via lentiviral or CRISPR techniques) and reinfused after conditioning, providing durable functional hemoglobin but high acute toxicity risk.
Both approaches demonstrate durable efficacy, though long‑term safety data remain limited.”
Stay updated with Hemostasis Today.
-
Jul 12, 2026, 08:2210 Posts Not To Miss from ISTH 2026, Part 1
-
Jul 12, 2026, 07:58Brian O Mahony: Could NXT-007 Minimize the Need for Perioperative FVIII
-
Jul 12, 2026, 07:50Shayan Mohammadmoradi: Connecting the Next Generation of Hemostasis Researchers at ISTH 2026
-
Jul 12, 2026, 07:42Alexandra Yakusheva: ThrombInnov Debuts at ISTH 2026 with a Focus on Translational Research
-
Jul 12, 2026, 07:34Wolfgang Miesbach: ISTH 2026 Explores the Next Generation of APS Diagnostics
-
Jul 12, 2026, 07:28Sara Zalghout: ISTH 2026 Highlights Progress in Thromboinflammation Research
-
Jul 12, 2026, 07:13Ekaterina Balaian: Finding Healing Through Art at ISTH 2026
-
Jul 12, 2026, 06:59Maaike Sybil Jongen: Flow Cytometry for Platelet Phenotyping at ISTH 2026
-
Jul 12, 2026, 06:45Brian O Mahony: Advancing Concizumab Therapy for Hemophilia With Inhibitors at ISTH 2026