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
- Genetic Literacy Project’s FAQ on gene editing
