{
  "url": "https://openlegislation.uk/eu-directive-2001-83/annex-I/part-IV/division-4/division-4.2/division-4.2.1",
  "json_url": "https://openlegislation.uk/eu-directive-2001-83/annex-I/part-IV/division-4/division-4.2/division-4.2.1.json",
  "legislation": "eudr/2001/83",
  "legislation_title": "Directive 2001/83/EC of the European Parliament and of the Council of 6 November 2001 on the Community code relating to medicinal products for human use",
  "legislation_url": "https://openlegislation.uk/eu-directive-2001-83",
  "path": "annex/I/part/IV/division/4/division/4.2/division/4.2.1",
  "kind": "division",
  "number": "4.2.1",
  "heading": "Pharmacology",
  "text": "(a) In vitro and in vivo studies of actions relating to the proposed therapeutic use (i.e. pharmacodynamic ‘ proof of concept ’ studies) shall be provided using models and relevant animal species designed to show that the nucleic acid sequence reaches its intended target (target organ or cells) and provides its intended function (level of expression and functional activity). The duration of the nucleic acid sequence function and the proposed dosing regimen in the clinical studies shall be provided.\n(b) Target selectivity: When the gene therapy medicinal product is intended to have a selective or target-restricted functionality, studies to confirm the specificity and duration of functionality and activity in target cells and tissues shall be provided.",
  "extent": "E+W+S+N.I.",
  "status": null,
  "version": "current",
  "annotations": [
    {
      "type": "F",
      "label": "F1",
      "text": "Substituted by Commission directive 2003/63/EC of 25 June 2003 amending Directive 2001/83/EC of the European Parliament and of the Council on the Community code relating to medicinal products for human use (Text with EEA relevance)."
    },
    {
      "type": "F",
      "label": "F2",
      "text": "Substituted by Commission Directive 2009/120/EC of 14 September 2009 amending Directive 2001/83/EC of the European Parliament and of the Council on the Community code relating to medicinal products for human use as regards advanced therapy medicinal products (Text with EEA relevance)."
    }
  ],
  "outstanding_effects": [],
  "provenance": {
    "source": "legislation.gov.uk",
    "source_url": "https://www.legislation.gov.uk/eudr/2001/83/annex/I/part/IV/division/4/division/4.2/division/4.2.1",
    "retrieved_at": "2026-11-01T15:22:59.083752+00:00"
  },
  "licence": "Open Government Licence v3.0",
  "licence_url": "https://www.nationalarchives.gov.uk/doc/open-government-licence/version/3/",
  "attribution": "Contains public sector information licensed under the Open Government Licence v3.0. Source: legislation.gov.uk, The National Archives."
}