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S12 – Nonclinical Biodistribution Considerations for Gene Therapy Products

Guideline number: S12
Status: Implementation (Step 5)
Adopted (Step 4): 14 March 2023

This topic was endorsed by the ICH Assembly in June 2019.

The ICH S12 Guideline reached Step 4 of the ICH process on 14 March 2023.

The ICH S12 Guideline:

  • Provides harmonised recommendations for the conduct of nonclinical biodistribution (BD) studies in the development of gene therapy (GT) products;
  • Includes recommendations to facilitate the development of GT products while avoiding unnecessary use of animals, in accordance with the 3Rs (reduce/refine/replace) principles;
  • Makes recommendations for the overall design of nonclinical BD assessments.

The ICH Harmonised Guideline was finalised under Step 4 in July 1997. This document covers the preclinical safety testing requirements for biotechnological products. It addresses the use of animal models of disease, determination of when genotoxicity assays and carcinogenicity studies should be performed, and the impact of antibody formation on duration of toxicology studies.

An addendum was proposed to provide clarification on S6 and an update of the following topics discussed in the original ICH S6 Guideline: species selection, study design, immunogenicity, reproductive and developmental toxicity and assessment of carcinogenic potential. The Harmonised addendum provides further complementary guidance to the S6 Guideline and helps to define the current recommendations and reduce the likelihood that substantial differences will exist among regions.

The addendum reached Step 4 of the harmonisation process in June 2011 and was integrated as part II in the core Guideline that was then renamed S6(R1).

Guideline
WG Presentations/Trainings

Annexes

No results found.

Guideline implementation

ANMAT, Argentina

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Not yet implemented;
ANVISA, Brazil

-

In the process of implementation;
EC, Europe

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Implemented; Date: 30 September 2023; Reference: EMA/CHMP/ICH/318372/2021
EDA, Egypt

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Implemented; Date: 25 September 2024; Reference: Regulatory Guide for mechanisms, procedures and rules of implementing the Decree of Egyptian Drug Authority No.343 of 2021
FDA, United States

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Implemented; Date: 4 May 2023; Reference: 88 pg. 28565-28566
HSA, Singapore

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In the process of implementation;
Health Canada, Canada

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Implemented; Date: 21 July 2023; Reference: File #: 20-109241-111
JFDA, Jordan

-

Not yet implemented;
MFDS, Republic of Korea

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Implemented; Date: 3 September 2024; Reference: Guideline on Nonclinical Biodistribution Considerations for Gene Therapy Products [Guideline-1366-01]
MHLW/PMDA, Japan

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Implemented; Date: 23 October 2023; Reference: PSB/MDED Notification No. 1023-1
MHRA, UK

-

Not yet implemented;
NAFDAC, Nigeria

-

Not yet implemented;
NMPA, China

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Implemented; Date: 5 September 2023; Reference: NMPA, China Announcement No. 115 (2023)
SAHPRA, South Africa

-

In the process of implementation;
SFDA, Saudi Arabia

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Implemented; Date: 7 January 2024;
Swissmedic, Switzerland

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Implemented; Date: 14 March 2023;
TFDA, Chinese Taipei

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Implemented; Date: 19 June 2025; Reference: Public Announcement for ” List for ICH Guidelines Adopted.”
TITCK, Türkiye

-

Not yet implemented;

Q+A

Q+A Status: Implementation (Step 5)