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United Kingdom: 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 clinical trials require a license from the Medicines and Healthcare products Regulatory Agency (MHRA) as well as oversight from the Human Tissue Authority (HTA) and the Health Research Authority (HRA).

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.

The MHRA regulates medicines and medical devices by authorizing gene therapies, assessing applications for clinical trial authorization, overseeing and inspecting clinical trials and licensing gene therapies. The HTA, established under the Human Tissue Act 2004, is responsible for the safe and ethical use of human tissue and organs. The HTA grants licenses to hospitals and clinics that remove, test, process, store, and distribute tissues or cells that will be used to treat patients. The HRA is responsible for approving the ethical aspects of clinical trials involving stem cells and other regenerative medicines through the Gene Therapy Advisory Committee (GTAC).

Gene therapies must also comply with the EU Directive on medicinal products for human use. All gene therapies marketed in the EU require authorization through the European Medicines Agency (EMA). Marketing authorization is centralized (one license is valid in entire EU) and reviewed by the Committee for Advanced Therapies (CAT; established by the EMA) within 90 days.

Products/Research

  • HIV: University College London, Imperial College London, Cambridge and Oxford Universities researchers treated a patient with stem cells, which resulted in his HIV as well as Hodgkin’s lymphoma going into remission.
  • Cystic fibrosis: Researchers used gene therapy to improve lung function in patients with cystic fibrosis, a disease in which the lungs and digestive system get clogged with mucus.
  • Donor blood cells: The Great Ormond Street Hospital in the UK has used TALENs for gene editing in donated blood cells to disable the gene which the immune system uses to recognise ‘foreign’ cells. This allowed a patient to receive donated blood cells, without the donor cells attacking the patients’ healthy cells.
  • Muscular dystrophy: Researcher groups from London and Paris, in collaboration with Benitec Biopharma, completed pre-clinical studies in 2017 to treat a neuromuscular disease called oculopharyngeal muscular dystrophy with RNA interference, a type of gene therapy.
  • Hemophilia: A clinical trial began in 2017 for a gene therapy to treat hemophilia, a blood clotting disease.
  • Cancer: Gene therapy clinical trials completed for various types of cancer including head and neck, liver, ovarian, prostate, breast, colorectal, cervical, melanoma and non-Hodgkin’s lymphoma. CAR T-cell therapy clinical trials (using patients’ own immune cells to treat their cancer) completed for various types of cancer including leukemia, head and neck cancer and melanoma.

Regulatory Timeline

2019: Medicines and Healthcare products Regulatory Agency (MHRA) issues Guidance on Substantial Amendments to a Clinical Trial if the UK Leaves the EU with No Deal, which covers amendments to clinical trial regulations.

 2004: Medicines for Human Use (Clinical Trials) Regulations 2004 released, which establishes the regulatory framework for gene therapy clinical trials.

2004: Human Tissue Act passed, establishing the Human Tissue Authority (HTA) and regulating matters relating to human bodies, organs and tissue for research and transplantation.

1992: Committee on the Ethics of Gene Therapy (the Clothier Committee) recommends that gene therapy should be limited to life threatening diseases or disorders and establishes the Gene Therapy Advisory Committee (GTAC), which advises on the ethical acceptability of proposals for gene therapy research.

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
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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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