India will start clinical trial for treating sickle cell disease among central India tribal

On the occasion of Janjatiya Gaurav Divas, the Council for Scientific and Industrial Research Institute of Genomic and Integrative Biology (CSIR-IGIB) has transferred indigenous technology for developing a CRISPR-based gene-editing therapy for Sickle Cell Disease (SCD) to the Serum Institute of India Private Limited (SII). India has the second-highest number of children born with SCD globally, with an estimated 15,000 to 25,000 new cases each year, particularly among tribal communities in Central India. SII will now conduct clinical trials to develop a more cost-effective treatment for this genetic disorder. At present, CRISPR-based gene-editing therapy for SCD costs around USD 3 million (approximately Rs 26 crore), mainly due to high licensing fees. The newly developed indigenous technology, however, is expected to bring the cost down to around Rs 50 lakh. Meanwhile, IGIB has already begun the first phase of clinical trials in collaboration with multiple agencies, including the Drug Controller General of India (DCGI), the Department of Biotechnology (DBT), the Department of Science and Technology (DST), the Ministry of Tribal Affairs, and CSIR. We have created a pool of around 150 potential participants from cities such as Jabalpur in Madhya Pradesh, Raipur in Chhattisgarh, and Ranchi in Jharkhand to begin the clinical trial, said Dr Souvik Maiti, Senior Principal Scientist at CSIR-IGIB. We will finalise three participants from this pool for our first trial after obtaining the necessary approvals, added Maiti, the lead investigator for this flagship project. Extraction of the affected gene from bone marrow will be carried out at AIIMS Delhi, while the gene editing will take place at IGIBs dedicated centre. The extraction process is expected to take 120 days. Following the transfer of technology, SII will collaborate with IGIB to conduct the second and third phases of clinical trials, involving a larger pool of participants. IGIB noted that a similar treatment was successfully administered to three African individuals in the United States. Encouraged by this progress, Indian scientists decided to replicate the process domestically using indigenous technology. It has taken nearly six years for India to develop its own CRISPR platform. Experts believe that similar indigenisation of CRISPR technology can also be achieved in the agriculture sector. Currently, ICAR has been negotiating licensing fees with foreign agencies rather than developing its own indigenous CRISPR systems.