The Landscape of Early Clinical Gene Therapies outside of Oncology.

Autor:	Rittié L; GSK R&D Cell and Gene Therapy Discovery Research, UP1410, 1250 S. Collegeville Road, Collegeville, PA 19426, USA. Electronic address: laure.x.rittie@gsk.com., Athanasopoulos T; GSK R&D Cell and Gene Therapy Discovery Research, 6F, Gunnels Wood Road, Stevenage, Herts SG1 2NY, UK., Calero-Garcia M; GSK R&D Cell and Gene Therapy Discovery Research, 6F, Gunnels Wood Road, Stevenage, Herts SG1 2NY, UK., Davies ML; GSK R&D Cell and Gene Therapy Discovery Research, 6F, Gunnels Wood Road, Stevenage, Herts SG1 2NY, UK., Dow DJ; GSK R&D Cell and Gene Therapy Discovery Research, 6F, Gunnels Wood Road, Stevenage, Herts SG1 2NY, UK., Howe SJ; GSK R&D Cell and Gene Therapy Process Research, 6F, Gunnels Wood Road, Stevenage, Herts SG1 2NY, UK., Morrison A; GSK R&D Worldwide Business Development, 5G104, Gunnels Wood Road, Stevenage, Herts SG1 2NY, UK., Ricciardelli I; GSK R&D Cell and Gene Therapy Discovery Research, 6F, Gunnels Wood Road, Stevenage, Herts SG1 2NY, UK., Saudemont A; GSK R&D Cell and Gene Therapy Discovery Research, 6F, Gunnels Wood Road, Stevenage, Herts SG1 2NY, UK., Jespers L; GSK R&D Cell and Gene Therapy Discovery Research, 6F, Gunnels Wood Road, Stevenage, Herts SG1 2NY, UK., Clay TM; GSK R&D Cell and Gene Therapy Discovery Research, UP1410, 1250 S. Collegeville Road, Collegeville, PA 19426, USA.
Jazyk:	angličtina
Zdroj:	Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2019 Oct 02; Vol. 27 (10), pp. 1706-1717. Date of Electronic Publication: 2019 Sep 06.
DOI:	10.1016/j.ymthe.2019.09.002
Abstrakt:	The field of cell and gene therapy (GT) is expanding rapidly and there is undoubtedly a wave of enthusiasm and anticipation for what these treatments could achieve next. Here we assessed the worldwide landscape of GT assets currently in early clinical development (clinical trial phase 1/2 or about to enter clinical trial). We included all gene therapies, i.e., strategies that modify an individual's protein make-up by introducing exogenous nucleic acid or nucleic acid modifiers, regardless of delivery. Unmodified cell therapies, oncology therapies (reviewed elsewhere), and vaccine programs (distinct therapeutic strategy) were not included. Using a December 31, 2018 cutoff date, we identified 336 gene therapies being developed for 138 different indications covering 165 genetic targets. In all, we found that the early clinical GT landscape comprises a very disparate group of drug candidates in terms of indications, organizations, and delivery methods. We also highlight interesting trends, revealing the evolution of the field toward in vivo therapies and adeno-associated virus vector-based delivery systems. It will be interesting to witness what proportion of this current list effectively translates into new medicines. (Copyright © 2019 The American Society of Gene and Cell Therapy. All rights reserved.)
Databáze:	MEDLINE
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