India launches indigenous gene therapy to eradicate sickle cell disease

Aligning with the aim of eradicating sickle cell disease which is largely prevalent among the tribal population, India on Wednesday launched the first indigenous CRISPR-based gene therapy.

The breakthrough was developed at CSIR–Institute of Genomics & Integrative Biology (IGIB). Sickle cell anaemia is an inherited blood disorder where red blood cells misshapen, leading to pain crises, anaemia and organ damage. 

CRISPR (clustered regularly interspaced short palindromic repeats) is a gene-editing technology that allows scientists to modify DNA in sickle cell disease patients. Doctors collect blood from the patient and isolate the patients carrying the sickle cell mutation and stem cells are edited. The edited stem cells are reinfused into the patient. Once inside the body, they produce healthy blood thereby eliminating the disease at source. 

A formal technology transfer and collaboration agreement was also exchanged between CSIR-IGIB and the Serum Institute of India Pvt. Ltd., enabling translation of IGIB’s engineered enFnCas9 CRISPR platform into scalable, affordable therapies for Sickle Cell Disease and other critical genetic disorders. 

While Phase 1 clinical trials were conducted on only three patients with funding from the Department of Science, Phase 2 and 3 trials will be done by Serum institute. 

“Sickle cell anaemia among tribals is a big challenge. The technology which was in developed countries was very costly and hence a need for the development of indigenous CRISPR technology was felt. DST came on the table. The Phase 1 clinical trials were on three patients funded by DST catalysed by the Ministry of Tribal Affairs. We cannot take the technology to market with only Phase 1 clinical trials done only on three patients," Souvik Maiti, Director of CSIR-IGIB informed. 

During the launch, Union Minister of State for Science & Technology Dr Jitendra Singh said that India has formally begun its journey towards becoming a sickle cell disease-free nation. 

The therapy, named "BIRSA 101" is dedicated to Birsa Munda, a tribal freedom fighter, whose 150th birth anniversary was observed last week.

Singh emphasised that the major breakthrough in gene therapeutics has demonstrated India’s capability to produce path-breaking therapies at a fraction of global costs, potentially replacing treatments priced at Rs 20–25 crore overseas. He underlined that this innovation carries deep national significance, especially for tribal communities in central and eastern India, where the disease burden is highest.

Explaining the gene-editing approach in simple terms, the minister said that the technology works like a ‘precise genetic surgery’, capable not only of curing sickle cell disease, but also transforming treatment pathways for several hereditary disorders.