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.
-
Mar 31, 2026, 16:05Happy National Doctors’ Day to All Physicians Making a Difference – ASH
-
Mar 31, 2026, 16:03Robert Negrin: Strengthening Collaboration with NCI to Advance Hematology Research
-
Mar 31, 2026, 16:02Jim Hoffman: Does Multiomics Suggest LC PASC Patients Require Multiple Drugs to Correct Biochemical Dysregulation?
-
Mar 31, 2026, 15:59Seema Dawood: Blister Cell in G6PD Deficiency
-
Mar 31, 2026, 15:57Ney Carter Borges: Clopidogrel vs Aspirin in Long-Term Monotherapy
-
Mar 31, 2026, 15:57Join the Webinar on Immunoglobulin Access and Stewardship in the UK and Australia – ISBT
-
Mar 31, 2026, 15:55Hind El Azzazi: Successful Defense of My Medical Doctorate on Antiphospholipid Syndrome in Men
-
Mar 31, 2026, 15:54Rucha Patil: Honored to Speak at AIIMS Nagpur ToT Workshop on Non-Factor Therapy in Hemophilia A
-
Mar 31, 2026, 15:43Deepak Sudheendra: When the Clot Is Gone, but Symptoms Remain