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In-Vivo GMO Gene Therapy Medicinal Products – Pharmacy Institutional Readiness Guidance

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Topics: Advanced therapy medicinal productsClinical trialsGuidance

This document outlines key areas for focus of pharmacy expertise prior to an organisation implementing In-Vivo Genetically Modified Organism (GMO) Gene Therapy.

About

In-vivo GMO Gene Therapies are ATMPs which are handled and manipulated by Pharmacies. In-vivo GMO gene therapy directs the GTMP into a specific tissue in the body and the GMO is then taken up by individual cells, or (more usually) it is administered intravenously (IV)

V2 of this guidance covers genome modifying gene therapy only.

Purpose

The purpose of this document is to outline the key areas where chief pharmacists should focus pharmaceutical expertise prior to an organisation implementing any in-vivo (virus based) Gene Therapy Medicinal Product (GTMP) e.g. IMP for Clinical Trials or Marketed ATMPs.

This document presents a flow diagram outlining a stepwise approach to implementing in-vivo (virus based) GTMP. It is followed by checklists which relate to the various steps presented in the diagram.

Attachments

Update history

  1. Updated to cover GMO gene therapy only. Non GMO gene therapy guidance will follow.
  2. Published

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