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A Guide to Approaching Regulatory Considerations for Lentiviral-Mediated Gene Therapies

Lookup NU author(s): Michael White, Professor Roger Whittaker, Dr Carolina Gandara de Souza, Dr Elizabeth Stoll



This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0).


Lentiviral vectors are increasingly the gene transfer tool of choice for gene or cell therapies, with multiple clinical investigations showing promise for this viral vector in terms of both safety and efficacy. The third-generation vector system is well characterized, effectively delivers genetic material and maintains long-term stable expression in target cells, delivers larger amounts of genetic material than other methods, is nonpathogenic, and does not cause an inflammatory response in the recipient. This report aims to help academic scientists and regulatory managers negotiate the governance framework to achieve successful translation of a lentiviral vector-based gene therapy. The focus is on European regulations and how they are administered in the United Kingdom, although many of the principles will be similar for other regions, including the United States. The report justifies the rationale for using third-generation lentiviral vectors to achieve gene delivery for in vivo and ex vivo applications; briefly summarizes the extant regulatory guidance for gene therapies, categorized as advanced therapeutic medicinal products (ATMPs); provides guidance on specific regulatory issues regarding gene therapies; presents an overview of the key stakeholders to be approached when pursuing clinical trials authorization for an ATMP; and includes a brief catalogue of the documentation required to submit an application for regulatory approval of a new gene therapy.

Publication metadata

Author(s): White M, Whittaker R, Gandara C, Stoll EA

Publication type: Article

Publication status: Published

Journal: Human Gene Therapy Methods

Year: 2017

Volume: 28

Issue: 4

Pages: 163-176

Print publication date: 01/08/2017

Acceptance date: 06/06/2017

Date deposited: 27/03/2018

ISSN (print): 1946-6536

ISSN (electronic): 1946-6544

Publisher: Mary Ann Liebert, Inc

URL: .

DOI: 10.1089/hgtb.2017.096


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Funder referenceFunder name
Wellcome Trust