Regulation of Human Cellular and Gene Therapeutic Products Incorporating Genome Editing Technologies in India

Abstract

New and advanced cellular and gene therapeutic (CGT) products using genome editing technologies have emerged in recent years as a curative treatment option for rare and genetic diseases. CGT products that edit the genome of human somatic cells are making it possible to treat the root cause of many rare and genetic diseases previously regarded as untreatable, and clinical trials of such products are under way worldwide. At present the only approved CGT product using genome editing is Casgevy, which is approved in the United States, the European Union and several other jurisdictions for the treatment of sickle cell disease and beta thalassaemia. In India a closely comparable therapy, BIRSA 101, has been developed indigenously for sickle cell disease and is expected to secure approval in the coming years. The Government of India has notified the New Drugs and Clinical Trials Rules, 2019 under the Drugs and Cosmetics Act, 1940 to accelerate the approval of CGT products. Under the 2019 Rules the Central Drugs Standard Control Organisation grants commercial approval of stem cell derived products and gene therapeutic products as new drugs. Alongside their curative potential, CGT products that edit the genome of human somatic cells carry additional risks, because they make permanent changes to the human genome and may produce off-target effects and other adverse events. The legal framework and the guidelines issued by the regulatory bodies must secure the safe and ethical application of these products, while timely approval is equally necessary if the unmet need for these lifesaving therapies is to be met. This article critically examines the legal framework and offers suggestions for addressing the challenges now facing India.