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.”
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