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European Union: Therapeutic / Stem Cell

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Highly Regulated

Limited somatic cell gene therapies permitted and hundreds of clinical trials ongoing.

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

 

Click on a country (eg. Brazil, US) or region (eg. European Union) below to find which human / health products and processes are approved or in development and their regulatory status.

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Human / Health Gene Editing Index
Compare Regulatory Restrictions Country-to-Country

Gene editing regulations worldwide are evolving. The Gene Editing Index ratings below represent the current status of gene editing regulations and will be updated as new regulations are passed.

Colors and ratings guide
 

Regulation StatusRating
Determined: No Unique Regulations*10
Lightly Regulated8
Proposed: No Unique Regulations†6
Ongoing Research, Regulations In Development5
Highly Regulated4
Mostly Prohibited2
Limited Research, No Clear Regulations1
Prohibited0
Lightly Regulated: Gene and stem cell therapies regulated with minimal restrictions and requirements.
*Determined: No Unique Regulations: Gene and stem cell therapies regulated as phamaceuticals with no additional restrictions.

†Proposed: No Unique Regulations: Decrees under consideration for gene and stem cell therapies that would not require unique regulations beyond current restrictions on pharmaceuticals.

Therapeutic:
Gene editing of adult human cells, including gene therapy and stem cell therapy, that is used to treat and cure disease. Recent breakthroughs include CAR T-cell therapy, which uses patients’ own immune cells to treat their cancer.
Germline:
Gene editing of the human embryo or germline that results in genetic changes that are passed down to the next generation. This type of gene editing is the most controversial because changes are inherited and because it could theoretically be used to create “designer babies”. A Chinese scientist announced in 2018 that he had successfully edited twins that were brought to term. International backlash from the announcement has resulted in China and other countries working to clarify regulations on germline gene editing.

Rating by Country / Region
Click each column header and arrow to sort the countries / regions

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Country / RegionTherapeuticGermlineHuman Rating
Japan888
Brazil402
Canada402
Russia1057.5
Argentina513
Israel825
Australia402
China846
US402
Chile412.5
New Zealand402
Ukraine1057.5
Central America111
Paraguay111
Uruguay111
India402
UK444
Mexico804
EU402
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Gene editing is a set of techniques that can be used to precisely modify the DNA of almost any organism. It is being used for applications in human health, gene drives and agriculture. There are numerous gene-editing tools besides CRISPR-Cas 9, which gets most of the attention because it is a comparatively easy tool to use.

Gene editing does not usually involve transgenics – moving ‘foreign’ genes between species. It also refers to a specific technique in contrast to the general term GMO, which is scientifically ambiguous, as genetic modification is a process not a product. Most gene editing involves creating new products by deleting very small segments of DNA (sometimes in agriculture called Site-Directed Nuclease 1 or SDN-1 techniques), which can silence a gene or change a gene’s activity. Countries are evaluating whether or not to regulate this type of gene editing, since it is so similar to natural mutations. The GLP’s Gene Editing Index ratings reflect the regulatory status of SDN-1 techniques, which are the most liberally regulated and will generate most products in the near term.

To develop different products, gene editing can change larger segments of DNA or add DNA from other species (a form of transgenics sometimes in agriculture called SDN-2 or SDN-3 techniques). While many countries are not regulating or lightly regulating SDN-1 techniques, most are moving toward tightly regulating or even restricting SDN-2 and SDN-3.

For more background on the various gene editing SDN techniques, read background articles here and here.

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