Gene therapy is permitted but must comply with clinical trial regulations, the EU Commission Regulation on advanced therapy medicinal products, and the European GMO Directive, written in 2001, that outlines regulations for organisms altered through genetic modification. Requirements include environmental risk assessments and assessments on the potential adverse effects on human health. In addition, the regulations require that agencies are able to trace all gene therapy substances.
Gene therapy is an experimental technique that uses genes to allow doctors to treat or prevent a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery. Examples include: (1) Replacing a mutated gene that causes disease with a healthy copy of the gene; (2) Inactivating, or “knocking out,” a mutated gene that is functioning improperly; (3) Introducing a new gene into the body to help fight a disease. One type of gene therapy is stem cell therapy, in which a patient’s own stem cells are used to repair or rebuild tissue.
Gene therapy is regulated by the EU Commission through the EU Commission Regulation on advanced therapy medicinal products, the European Medicines Agency (EMA) and the Federation of European Academies of Medicine (FEAM). Gene therapies must also comply with the Directive on medicinal products for human use. To be approved, research and clinical trials on gene therapy require an Investigational Medicinal Product Dossier, which includes a risk assessment, safety and efficacy data. Marketing authorization is centralized (one license is valid in entire EU) and reviewed by the Committee for Advanced Therapies (CAT) within 90 days.
Products/Research
- Liver disease: In 2019, Promethera Biosciences tested a new stem cell treatment for severe liver diseases in the first patient. The clinical trial will be conducted across eight European countries.
- Cancer vaccine: In 2019, researchers in Germany tested an RNA-based vaccine for patients with melanoma.
- Wiskott-Aldrich syndrome: In 2019, researchers from France and England successfully treated a rare genetic disease that causes bleeding, severe and recurrent infections, severe eczema and in some patients autoimmune reactions and the development of cancer.
- Blood disorder: Gene therapy to treat a blood disorder called beta thalassemia that reduces a patient’s ability to produce hemoglobin, the protein in red blood cells that contains iron, leading to life-threatening anemia. Approved in 2019.
- Fatal muscle disease: Clinical trials ongoing for gene therapy for X-linked myotubular myopathy, a muscle disease in which patients typically survive only into early childhood.
- Rare form of blindness: Gene therapy for patients with a rare form of inherited blindness called biallelic RPE65 mutation-associated retinal dystrophy. Approved in 2018.
- Lymphoma: Gene therapy to treat a cancer called large B cell lymphoma. Approved in 2018.
- Crohn’s disease symptoms: A cell therapy used to treat specific severe symptoms of Crohn’s disease. Approved in 2018.
- Leukemia: Gene therapy for patients with B cell lymphoblastic leukemia. Approved in 2018.
- Vein disease: Gene therapy to treat severe cases of veno-occlusive disease, a disorder in which the small veins of the liver become obstructed, in patients who have received a bone marrow transplant. Approved in 2017.
- “Bubble boy” disease: Approved in 2016 to treat ADA Severe Combined Immune Deficiency (ADA-SCID), a disease in children that causes them to be extremely susceptible to infections.
- Eye damage: First stem cell therapy approved in Europe in 2015 to treat physical or chemical burns to the eye.
- Melanoma: A genetically engineered virus used to treat inoperable melanoma. Conditionally approved in Europe in 2015.
- Inability to digest fats: First gene therapy approved in Europe in 2012 to treat lipoprotein lipase deficiency, a rare disease that leaves individuals unable to digest fats and can cause life-threatening pancreatitis.
Regulatory Timeline
2018: European Court of Justice (ECJ) rules that organisms developed through gene editing are genetically modified organisms (GMOs) and are subject to the same regulations as transgenic organisms, rejecting a regulatory exemption or the issuance of a revised directive.
2012: First gene therapy in Europe is approved.
2007: EU Commission Regulation on advanced therapy medicinal products is finalized, which outlines the procedure for gene therapy approval.
2001: Directive on medicinal products for human use is finalized.
2000: EU Charter of Fundamental Rights
1997: Convention on Human Rights and Biomedicine (Oviedo Convention) of the Council of Europe.
NGO Reaction
None
Additional Resources
- Genes and Science’s FAQ on gene editing
- Library of Congress summary of EU gene regulations includes detailed analysis of the country’s evolving biosafety laws and liabilities
- European Society of Gene and Cell Therapy Organization Heatmap
