The first stop for professional medicines advice

Ex-Vivo (cell based) GMO Gene Therapy Medicinal Products – Pharmacy Institutional Readiness Guidance

Last updated See all updates
Topics: Advanced therapy medicinal productsClinical trialsGuidance

This document outlines key areas for focus of pharmacy expertise prior to an organisation implementing Ex-Vivo genetically modified organism (GMO) Gene Therapy.

About

Ex-vivo gene therapy medicinal products are ATMPs, which require pharmacy oversight, even when being handled elsewhere within a healthcare organisation. A GTMP is classed as an ex-vivo GMO gene therapy when genetic modification occurs outside the body, during the manufacturing process.

V2 of this guidance covers genome modifying ex vivo 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 ex-vivo (cell based) Gene Therapy Medicinal Product (GTMP).

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

Attachments

Update history

  1. Updated to limit scope to GMO gene therapy. Non-GMO guidance will follow.
  2. Published

Print this page