Multidisciplinary: gene therapy
HumanAdvanced therapiesRegulatory and procedural guidanceInnovationResearch and developmentScientific guidelines
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.
Guidelines
- Guideline on quality, non-clinical and clinical requirements for investigational advanced therapy medicinal products in clinical trials - Scientific guideline
- Guideline on safety and efficacy follow-up and risk management of advanced therapy medicinal products - Scientific guideline
- Quality, preclinical and clinical aspects of gene therapy medicinal products
- Quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells - Scientific guideline
- 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 - Scientific guideline
- Follow-up of patients administered with gene therapy medicinal products
- Scientific requirements for the environmental risk assessment of gene-therapy medicinal products
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 - Scientific guideline
- Management of clinical risks deriving from insertional mutagenesis
Questions and answers
- Questions and answers on comparability considerations for advanced therapy medicinal products (ATMP) - Scientific guideline
- 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