Multidisciplinary: gene therapy

The European Medicines Agency's scientific guidelines on gene therapy help medicine developers prepare marketing authorisation applications for human medicines.

For a complete list of scientific guidelines currently open for consultation, see Public consultations.

Reflection papers

• Design modifications of gene therapy medicinal products during development
• Quality, non-clinical and clinical issues relating specifically to recombinant adeno-associated viral vectors
• Management of clinical risks deriving from insertional mutagenesis

Questions and answers

• Questions and answers on comparability considerations for advanced therapy medicinal products (ATMP)
• Questions and answers on gene therapy

ICH

• ICH guideline S12 on nonclinical biodistribution considerations for gene therapy products - Step 2b
• ICH Considerations: general principles to address virus and vector shedding
• ICH Considerations: oncolytic viruses

Position statements

• Creutzfeldt-Jakob disease and advanced therapy medicinal products

Guidelines

• Guideline on quality, non-clinical and clinical requirements for investigational advanced therapy medicinal products in clinical trials
• Guideline on safety and efficacy follow-up and risk management of advanced therapy medicinal products
• Quality, preclinical and clinical aspects of gene therapy medicinal products
• Quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells
• Development and manufacture of lentiviral vectors
• Non-clinical studies required before first clinical use of gene therapy medicinal products
• Non-clinical testing for inadvertent germline transmission of gene transfer vectors
• Risk-based approach according to Annex I, part IV of Directive 2001/83/EC applied to Advanced Therapy Medicinal Products
• Follow-up of patients administered with gene therapy medicinal products
• Scientific requirements for the environmental risk assessment of gene-therapy medicinal products