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CAR T From Within - Vilcot, Brugère, Fuentealba, Houot

HemaSphere Journal

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CAR T From Within - Vilcot, Brugère, Fuentealba, Houot

10 просмотров · 8 дн. назад
HemaSphere Journal
194 подписчика
10 просмотров · 8 дн. назад
Could making CAR T cells directly inside the patient change how treatment is delivered? Scientific Editor Dr Charles De Bock joins Prof Roch Houot, Dr Jaime Fuentealba, François Vilcot, PharmD, and Carole-Anne Brugère, PharmD, to explore the promise and challenges of in vivo CAR therapy. They compare delivery approaches, examine safety and targeting, and discuss potential gains in access alongside uncertainty over costs. Listen for a multidisciplinary perspective on what comes next. Read the referenced Review Article here: https://bit.ly/4d1mfjP Key topics • How in vivo CAR therapy differs from conventional CAR T manufacturing • Lentiviral vectors and lipid nanoparticles: targeting, persistence, and safety, including the need for long-term monitoring • Clinical translation, scalability, and why simpler delivery may not mean lower prices • Europe’s role in development and lessons from writing a multidisciplinary review Timestamps 00:00 — Why in vivo CAR is attracting attention now 02:37 — What it means to manufacture CAR T cells inside the patient 05:29 — Three layers of specificity for lentiviral delivery 10:08 — Why modern lentiviral vectors are safer than early gene therapy vectors 14:41 — How lipid nanoparticles can target T cells and avoid the liver 16:26 — Whether transient CAR expression can still be therapeutic 19:10 — Choosing between lentiviral vectors and lipid nanoparticles 22:53 — The hardest unsolved problem: balancing efficacy, safety, and scalability 23:18 — Site-specific gene editing and TRAC locus insertion 26:21 — Industry momentum and whether in vivo CAR can lower cost 28:40 — Europe’s opportunity in clinical translation 31:23 — What to expect from upcoming ASH and other conference data 35:05 — How the multidisciplinary review was scoped and organized 37:09 — Why figures were designed early in the writing process 39:57 — Advice for PhD students writing a review in a fast-moving field