Centre Amends Drugs Rules for Advanced Cell and Gene Therapies

Centre Amends Drugs Rules for Advanced Cell and Gene Therapies

Why in News?

The Central Government has amended the Drugs Rules, 1945 to bring Advanced Cell and Gene Therapies (ACGT) under the Centrally License Approving Authority (CLAA) framework.

Key Amendments to the Drugs Rules, 1945

• Notification: The Central Government notified amendments to the Drugs Rules, 1945 under the Drugs and Cosmetics Act, 1940 through G.S.R. 530(E) (29 June 2026).
• Expansion: The amendment expands the Centrally License Approving Authority (CLAA) framework, ensuring uniform national licensing instead of fragmented regulation.
  • CLAA is the designated division of the Central Drugs Standard Control Organization (CDSCO) that approves clinical trials, new drugs, and specific high-risk medical devices. 
  • Headquartered at FDA Bhavan in New Delhi, it regulates the import of all medical devices and the manufacture of Class C and D devices.
  • It conducts facility inspections (both domestic and overseas) and coordinates with State Licensing Authorities (SLAs) for nationwide enforcement.
  • It is headed by the Drugs Controller General of India (DCGI), it governs approvals that require a uniform national standard. 
• Coverage: Cell- or stem cell-derived products are now formally covered under CLAA, bringing regenerative medicine within a robust regulatory structure for manufacturing and licensing.
  • The Rules now explicitly regulate gene therapeutic products, including innovative gene replacement and gene-editing therapies, through centralized approval and oversight. 
  • Xenografts—animal-derived tissues or organs used in humans—have been included, ensuring scientific evaluation, quality control and patient safety before clinical use. 
  • The newly covered therapies join previously regulated categories such as vaccines, large-volume parenterals and recombinant DNA-derived drugs under the CLAA system.
• Licensing Rules: Relevant provisions under Rules 75A, 76 and 76A now extend licensing requirements beyond recombinant DNA drugs to advanced cell and gene therapies.
  • Official licence forms such as Forms 27D, 27DA, 28D and 28DA have been revised to include cell therapies, gene therapies and xenografts.
• Joint Authority: The amendment establishes joint regulation by Central and State Licensing Authorities, improving regulatory consistency while retaining state-level supervision.
  • This mandates higher scientific and regulatory scrutiny before licensing.
• Objectives: The principal objective is to create a uniform, transparent and science-based regulatory framework that strengthens public health, encourages biotechnology innovation.
  • It aligns India with evolving global regulatory practices. 
  • The amendment recognizes that living-cell and gene-based products involve complex biological risks, requiring stricter quality assurance.

What are Advanced Cell and Gene Therapies (ACGT)?

• About: Advanced Cell and Gene Therapies (ACGT) are biological medicinal products that use living cells, tissues or genetic material to treat, prevent or potentially cure diseases of damaged cells or defective genes. 
• Principle: Unlike conventional drugs that mainly relieve symptoms, ACGT targets the root biological cause of disease by restoring normal cellular or genetic function.
• Components: ACGT broadly comprises cell therapies, gene therapies, and related advanced regenerative products.
  • India’s latest regulatory framework also covers cell or stem cell-derived products, gene therapeutic products and xenografts. 
  • Cell therapy involves administering living human cells, including stem cells or immune cells, to regenerate damaged tissues or destroy diseased cells. 
  • Gene therapy introduces, replaces or edits defective genes using engineered genetic material to restore normal function and treat inherited or acquired diseases.
    • It is designed to treat inherited genetic disorders such as sickle cell disease, thalassaemia, spinal muscular atrophy (SMA), haemophilia, inherited retinal blindness, and some immune deficiencies.
  • Stem cells possess self-renewal and differentiation abilities, enabling regeneration of damaged organs and tissues, making them valuable for chronic and degenerative disorders.
    • It is designed to treat blood cancers (leukaemia, lymphoma), thalassaemia, aplastic anaemia, spinal cord injury, Parkinson’s disease (research), osteoarthritis, burns, damaged tissues and organs.
  • CAR-T (Chimeric Antigen Receptor T-cell) therapy genetically modifies a patient’s T lymphocytes to recognize and destroy cancer cells, particularly leukaemia and lymphoma.
    • It is designed to treat blood cancers, especially acute lymphoblastic leukaemia (ALL), diffuse large B-cell lymphoma (DLBCL), and multiple myeloma.
  • Modern ACGT incorporates gene-editing platforms capable of correcting disease-causing mutations.
    • CRISPR-based therapies can correct disease-causing mutations in sickle cell disease, β-thalassaemia, inherited blindness, and other monogenic disorders.
  • Xenografts address organ and tissue shortages by providing animal-derived heart valves, skin grafts, and potentially organs for transplantation (research and limited clinical use). It is widely used in cardiology and orthopedics. 
• Manufacturing: Production requires Good Manufacturing Practices (GMP), sterile laboratories, advanced biotechnology infrastructure, cryogenic storage and stringent quality testing. 

India’s Progress in Advanced Cell and Gene Therapies

• Indigenous Therapy: India achieved a historic milestone with NexCAR19 (Actalycabtagene autoleucel), the country’s first indigenously developed CAR-T cell therapy, approved by CDSCO for relapsed or refractory B-cell leukaemia and lymphoma.
  • NexCAR19 costs nearly one-tenth of comparable imported CAR-T therapies, significantly improving affordability.
  • The therapy was jointly developed by IIT Bombay, Tata Memorial Centre, and ImmunoACT.
  • The Department of Biotechnology (DBT) and Biotechnology Industry Research Assistance Council (BIRAC) provided sustained financial and technical support.
  • Indian multicentre Phase I/II trials involving about 60 patients reported an overall response rate of around 70%, with favourable safety.
  • By 2026, NexCAR19 became available across 30+ tertiary hospitals, expanding nationwide access.
• Indigenous CRISPR-based Platform: India officially launched its first indigenous CRISPR-based gene therapy named BIRSA 101.
  • This gene-editing initiative specifically targets tribal populations and marginalized communities, making breakthrough genetic corrections highly accessible.
• Policy: The BioE3 (Biotechnology for Economy, Environment and Employment) Policy promotes gene editing, precision medicine, and advanced cell therapies.
  • The policy is spearheaded by DBT and BIRAC. It targets a $300 billion bioeconomy by 2030. 
  • It targets six priority domains—climate-resilient agriculture, smart proteins, precision biotherapeutics (vaccines/gene therapy), bio-based chemicals, carbon capture, and marine/space biotechnology.
  • It promotes artificial intelligence and data integration to accelerate biological discovery and biomanufacturing processes.
  • It accelerates commercialization of bio-based products through three core “Bio-Enabler” infrastructures:
    • Biomanufacturing Hubs facilities provide shared infrastructure for academia and industry to scale up promising bio-based products from pilot to pre-commercial levels.
    • Bio-AI Hubs centers driving data-driven R&D, powering AI-informed predictive analytics to accelerate the discovery of sustainable biotechnologies.
    • Biofoundries specialized infrastructure dedicated to high-throughput biological engineering and prototyping.
• Collaboration: Biotech pioneers like ImmunoACT and Immuneel Therapeutics (which launched the CAR-T therapy Qartemi for lymphomas) are scaling operations.
  • India is expanding global partnerships, including the Indo-Singapore collaboration for developing dual-targeting CAR-T therapies against multiple myeloma. 
  • Clinical development for actalycabtagene autoleucel (NexCAR19) received critical support and study validation from the National Cancer Institute (NCI) in United States.
  • Bangalore-based NKure Therapeutics is developing affordable off-the-shelf Natural Killer (NK) cell immunotherapies. 

FAQs:

1. What are cell and gene therapies?
   They are advanced treatments using living cells or genetic material to repair, replace, or modify defective cells and genes.
2. Why were the Drugs Rules amended for cell and gene therapies?
   To ensure uniform regulation, centralized licensing, quality standards, and patient safety for advanced therapeutic products.
3. Which authority regulates cell and gene therapies in India?
   The Centrally License Approving Authority (CLAA) under the Central Drugs Standard Control Organisation regulates them.
4. How will the amended rules benefit patients?
   They improve safety, quality, regulatory oversight, and faster access to advanced therapies nationwide.
5. What diseases can cell and gene therapies treat?
   They treat cancers, thalassaemia, sickle cell disease, haemophilia, spinal muscular atrophy, and certain inherited disorders.
6. How do gene therapies differ from conventional medicines?
   Gene therapies correct genetic defects, while conventional medicines mainly manage symptoms or disease progression.
7. What is the role of clinical trials in cell and gene therapies?
   Clinical trials evaluate safety, efficacy, dosage, and long-term outcomes before regulatory approval.
8. How will the new rules promote innovation in healthcare?
   They provide regulatory clarity, encourage research, attract investment, and accelerate development of advanced therapies.

Disclaimer: Information in this article is based on official announcements and public records. Regulations and implementation details may evolve over time.