Schmidt, N.A., Giblin, J., MacLachlan, T.K. et al. (9 more authors) (2022) Current global regulatory landscape for biodistribution & shedding assessment of rAAV gene therapies & recommendations of the IMI ARDAT consortium on future directions. Cell and Gene Therapy Insights, 8 (3). pp. 377-394. ISSN 2059-7800
Abstract
An understanding of the biodistribution and shedding profile of a gene therapy product following in vivo administration is an important element of the development program. Recommendations for biodistribution studies have been issued by various regulatory authorities with the most recent draft International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use guideline S12 ‘Nonclinical biodistribution considerations for gene therapy products’ released for public comment on 3 June 2021. In this paper the Innovative Medicines Initiative, Accelerating Research & Development for Advanced Therapies consortium provides an overview of the current regulatory landscape for conducting shedding and biodistribution studies and makes a call for harmonization across regions. In addition, over the last three decades, a significant body of literature on biodistribution and shedding of AAV-based gene therapies has amassed, and we describe herein the initial stages of construction of a formal database of published biodistribution www.insights.bio 377CELL & GENE THERAPY INSIGHTS and shedding data. The outputs from the database could be leveraged by Sponsors of AAV programs in regulatory submissions. This would reduce the need for unnecessary duplicative studies, streamline nonclinical development and expedite the arrival of this important class of novel medicines into clinic.
Metadata
Item Type: | Article |
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Authors/Creators: |
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Copyright, Publisher and Additional Information: | © 2022 Schmidt NA, Giblin J, MacLachlan TK, Dandapat S, Veres G, Reimer TA, Schulz M, Hatzmann E, Dahy A, LaRosa L, Azzouz M, & Mann CJ. Published by Cell and Gene Therapy Insights under Creative Commons License Deed CC BY NC ND 4.0. This is an Open Access article distributed under the terms of the Creative Commons Attribution-NonCommercial-NoDerivatives licence (https://creativecommons.org/licenses/by-nc-nd/4.0/) |
Dates: |
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Institution: | The University of Sheffield |
Academic Units: | The University of Sheffield > Faculty of Medicine, Dentistry and Health (Sheffield) > Department of Neuroscience (Sheffield) |
Funding Information: | Funder Grant number EUROPEAN COMMISSION - HORIZON 2020 945473 |
Depositing User: | Symplectic Sheffield |
Date Deposited: | 07 Mar 2023 12:23 |
Last Modified: | 07 Mar 2023 19:17 |
Status: | Published |
Publisher: | BioInsights Publishing, Ltd. |
Refereed: | Yes |
Identification Number: | 10.18609/cgti.2022.056 |
Open Archives Initiative ID (OAI ID): | oai:eprints.whiterose.ac.uk:197025 |