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India: Therapeutic / Stem Cell

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Highly Regulated

Gene therapy is regulated and research is ongoing.

Gene therapy is allowed but requires approval from the Central Drugs Standard Control Organisation (CDSCO).

Guidelines issued in 2019 establish a regulatory framework for gene therapy in an effort to standardize gene therapy product development and clinical trials. The guidelines require long-term follow up of at least five years for all clinical trials, and up to 10 years of follow up is recommended after commercialization. In 2019, the government proposed the creation of an independent body of biomedical and gene therapy experts, The Gene Therapy and Advisory and Evaluation Committee (GTEAC), to supervise proposed therapies.

The National Ethical Guidelines for Biomedical and Health Research Involving Human Participants applies to all clinical trials involving human participants, including gene therapy trials. The guidelines set out principles to ensure the safety and dignity of human participants, including the right to privacy and the principle of voluntariness.

India has banned the commercial use of stem cell therapy due to “rampant malpractice”. Stem cells are allowed, with approval from the CDSCO, for use in clinical trials only, except for when treating certain forms of blood cancers, malignant lymphomas and tumors. The non-malignant diseases exempt from the ban include sickle cell disease and thalassemia major. 

Products/Research

  • Hemophilia: The first application for a trial of gene therapy for hemophilia, an inherited blood-clotting disorder, submitted in 2019.
  • Sickle cell anemia: Institute of Genomics and Integrated Biology (IGIB) used CRISPR to develop a cure for sickle cell anemia, a genetic blood disease that is particularly prevalent and devastating to populations in India.
  • Mechanism for rejuvenating old stem cells: The National Centre for Cell Science (NCCS) developed a mechanism that makes stem cells from older donors more viable for bone marrow transplantation, expanding the donor cohort and thus the breadth of treatment.
  • Alzheimer’s disease research: National Centre of Biological Sciences (NCBS) used CRISPR on stem cells to study a gene linked to Alzheimer’s disease.
  • Inherited blindness: Institute for Stem Cell Biology and Regenerative Medicine used stem cells to explore a possible treatment for retinitis pigmentosa, a common cause of blindness in India.
  • Beta-thalassemia research: IGIB used CRISPR to study a possible treatment for beta-thalassemia, an inherited blood disorder, as well as hemophilia A and hemophilia B.

Regulatory Timeline

2019: ICMR issues guidelines “to ensure that gene therapies can be introduced in India and clinical trials for gene therapies can be performed in an ethical, scientific and safe manner.” It recommends the creation of an independent body of biomedical and gene therapy experts, The Gene Therapy and Advisory and Evaluation Committee, to supervise proposed therapies.

2019: New Drugs and Clinical Trials Rules consider gene therapy products as new drugs and require approval from the Central Drugs Standard Control Organisation.

2017: National Ethical Guidelines for Biomedical and Health Research Involving Human Participants sets out principles for clinical trials involving human participants.

2003: The government of India forms the Stem Cell Task Force to encourage stem cell research.

2000: The Ethical Guidelines on Biomedical Research Involving Human Subjects produced by the Indian Council of Medical Research restricts studies to somatic cell gene therapy. Such studies are permitted only for the purpose of preventing or treating serious disease. Germline therapy is prohibited.

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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