Advancing ex vivo gene therapy: the role of lentiviral vectors and organizational collaboration.

Authors

Yuri Sano, Yu Nishida, Kota Kodama, Shuto Miyashita, Shintaro Sengoku

Published in

Drug discovery today. Pages 104510. Oct 24, 2025. Epub Oct 24, 2025.

Abstract

Gene therapy is emerging as a promising treatment. Ex vivo gene therapy modifies patient cells outside the body before reintroducing them. It targets diseases, such as rare disorders and cancer, but involves complex steps, including cell collection, custom vector production, and gene transfer. Considering these challenges, we examined the technical and organizational requirements for the widespread application of ex vivo gene therapy. Lentiviral vectors have become the de facto standard for gene transfer. Unlike in vivo therapy, multi-institutional collaboration drives development, supported by approved drug cases. Success relies on combining academic and biotech research, pharmaceutical acquisition, and Contract Development and Manufacturing Organization (CDMO) manufacturing expertise. More efficient, cost-effective treatment systems are essential for further industrial growth and broader adoption.

PMID:
41201621
Bibliographic data and abstract were imported from PubMed on 07 Nov 2025.

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