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.
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
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 Considerations
- ICH Considerations: general principles to address virus and vector shedding
- ICH Considerations: oncolytic viruses
Position statements