India Launches Clinical Trials for Sickle Cell Disease Treatment

India has commenced clinical trials for a novel gene-editing therapy aimed at treating sickle cell disease (SCD), a genetic disorder that disproportionately affects tribal populations in the country. This initiative was announced on Janjatiya Gaurav Divas by the Council for Scientific and Industrial Research Institute of Genomic and Integrative Biology (CSIR-IGIB), which has successfully transferred its indigenous technology to the Serum Institute of India Private Limited (SII).

Sickle cell disease is a significant health concern in India, with an estimated 15,000 to 25,000 new cases reported annually, primarily within tribal communities of Central India. The collaboration between CSIR-IGIB and SII aims to develop a more affordable treatment option, as the current CRISPR-based gene-editing therapy can cost around USD 3 million (approximately Rs 26 crore) due to high licensing fees. The indigenous technology is expected to reduce this cost to approximately Rs 50 lakh.

Dr Souvik Maiti, a Senior Principal Scientist at CSIR-IGIB, confirmed that the first phase of clinical trials has already begun in collaboration with various agencies, including the Drug Controller General of India (DCGI), the Department of Biotechnology (DBT), and the Ministry of Tribal Affairs. Dr Maiti stated, "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. We will finalise three participants from this pool for our first trial after obtaining the necessary approvals."

The process will involve extracting the affected gene from the participants' bone marrow at the All India Institute of Medical Sciences (AIIMS) in Delhi, followed by gene editing at IGIB’s dedicated centre. The extraction is anticipated to take around 120 days.

Following the initial trials, SII will work with CSIR-IGIB to carry out the second and third phases of clinical trials, which will include a larger number of participants. This development follows a successful treatment of three individuals in the United States, which has encouraged Indian scientists to replicate the process using local technology. It has taken approximately six years for India to establish its own CRISPR platform.

Experts in the field believe that similar advancements in indigenous CRISPR technology could also benefit the agriculture sector in India. Currently, the Indian Council of Agricultural Research (ICAR) is in discussions regarding licensing fees with foreign entities instead of pursuing its own development of CRISPR systems.

The introduction of this therapy marks a significant step forward in India's healthcare capabilities, particularly in addressing the needs of its tribal communities affected by sickle cell disease.