A Public Resource Compiled by the

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

  • 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 countries (eg. Brazil, US) or regions (eg. European Union) to find what agricultural products or therapies are approved or in development and their regulatory status.

Hotspots Background

European Union

EUROPEAN UNION

 

Agricultural Gene Editing
- Crops / Food
- Animals

 

Human Gene Editing
- Therapeutic / Stem Cell
- Germline / Embryonic

 

Gene Drives

 

Brazil

BRAZIL

 

Agricultural Gene Editing
- Crops / Food
- Animals

 

Human Gene Editing
- Therapeutic / Stem Cell
- Germline / Embryonic

 

Gene Drives

 

New Zealand

NEW ZEALAND

 

Agricultural Gene Editing
- Crops / Food
- Animals

 

Human Gene Editing
- Therapeutic / Stem Cell
- Germline / Embryonic

 

Gene Drives

 

United States

UNITED STATES

 

Agricultural Gene Editing
- Crops / Food
- Animals

 

Human Gene Editing
- Therapeutic / Stem Cell
- Germline / Embryonic

 
 

Gene Drives

 

Australia

AUSTRALIA

 

Agricultural Gene Editing
- Crops / Food
- Animals

 

Human Gene Editing
- Therapeutic / Stem Cell
- Germline / Embryonic

 

Gene Drives

 

Canada

CANADA

 

Agricultural Gene Editing
- Crops / Food
- Animals

 

Human Gene Editing
- Therapeutic / Stem Cell
- Germline / Embryonic

 
 

Gene Drives

 

China

CHINA

 

Agricultural Gene Editing
- Crops / Food
- Animals

 

Human Gene Editing
- Therapeutic / Stem Cell
- Germline / Embryonic

 
 

Gene Drives

 

United Kingdom

UNITED KINGDOM

 

Agricultural Gene Editing
- Crops / Food
- Animals

 

Human Gene Editing
- Therapeutic / Stem Cell
- Germline / Embryonic

 
 

Gene Drives

 

Israel

ISRAEL

 

Agricultural Gene Editing
- Crops / Food
- Animals

 

Human Gene Editing
- Therapeutic / Stem Cell
- Germline / Embryonic

 
 

Gene Drives

 

Argentina

ARGENTINA

 

Agricultural Gene Editing
- Crops / Food
- Animals

 

Human Gene Editing
- Therapeutic / Stem Cell
- Germline / Embryonic

 
 

Gene Drives

 

Japan

JAPAN

 

Agricultural Gene Editing
- Crops / Food
- Animals

 

Human Gene Editing
- Therapeutic / Stem Cell
- Germline / Embryonic

 
 

Gene Drives

 

Mexico

MEXICO

 

Agricultural Gene Editing
- Crops / Food
- Animals

 

Human Gene Editing
- Therapeutic / Stem Cell
- Germline / Embryonic

 
 

Gene Drives

ARGENTINA

 

Agricultural Gene Editing
Crops / Food
Animals

 

Human Gene Editing
Therapeutic / Stem Cell
Germline / Embryonic

 
 

Gene Drives

 

AUSTRALIA

 

Agricultural Gene Editing
Crops / Food
Animals

 

Human Gene Editing
Therapeutic / Stem Cell
Germline / Embryonic

 

Gene Drives

 

BRAZIL

 

Agricultural Gene Editing
Crops / Food
Animals

 

Human Gene Editing
Therapeutic / Stem Cell
Germline / Embryonic

 

Gene Drives

 

CANADA

 

Agricultural Gene Editing
Crops / Food
Animals

 

Human Gene Editing
Therapeutic / Stem Cell
Germline / Embryonic

 
 

Gene Drives

 

CHINA

 

Agricultural Gene Editing
Crops / Food
Animals

 

Human Gene Editing
Therapeutic / Stem Cell
Germline / Embryonic

 
 

Gene Drives

 

EUROPEAN UNION

 

Agricultural Gene Editing
Crops / Food
Animals

 

Human Gene Editing
Therapeutic / Stem Cell
Germline / Embryonic

 

Gene Drives

 

ISRAEL

 

Agricultural Gene Editing
Crops / Food
Animals

 

Human Gene Editing
Therapeutic / Stem Cell
Germline / Embryonic

 
 

Gene Drives

 

JAPAN

 

Agricultural Gene Editing
Crops / Food
Animals

 

Human Gene Editing
Therapeutic / Stem Cell
Germline / Embryonic

 
 

Gene Drives

 

MEXICO


Agricultural Gene Editing
Crops / Food
Animals


Human Gene Editing
Therapeutic / Stem Cell
Germline / Embryonic



Gene Drives

NEW ZEALAND

 

Agricultural Gene Editing
Crops / Food
Animals

 

Human Gene Editing
Therapeutic / Stem Cell
Germline / Embryonic

 

Gene Drives

 

UNITED KINGDOM

 

Agricultural Gene Editing
Crops / Food
Animals

 

Human Gene Editing
Therapeutic / Stem Cell
Germline / Embryonic

 
 

Gene Drives

 

UNITED STATES

 

Agricultural Gene Editing
Crops / Food
Animals

 

Human Gene Editing
Therapeutic / Stem Cell
Germline / Embryonic

 
 

Gene Drives