In a first, govt begins indigenous gene therapy to combat sickle cell disease

In a step towards eliminating sickle cell disease, which disproportionately affects India’s tribal population, the government on Wednesday launched the country’s first indigenous CRISPR-based gene therapy.

Developed at the Institute of Genomics & Integrative Biology (IGIB) in New Delhi, the breakthrough offers the potential for a permanent cure for sickle cell anaemia — an inherited disorder in which red blood cells become misshapen, causing severe pain episodes, anaemia and long-term organ damage.

Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) is a gene-editing technology that enables precise modification of DNA in patients. In this therapy, doctors collect a patient’s blood, isolate the stem cells carrying the sickle mutation, edit them, and reinfuse the corrected cells. Once inside the body, the edited cells generate healthy blood, addressing the disease at its source.

A formal technology transfer and collaboration agreement was signed between CSIR-IGIB and the Serum Institute of India Pvt. Ltd. to scale IGIB’s engineered enFnCas9 CRISPR platform into affordable therapies for sickle cell disease and other genetic disorders.

“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… We cannot take the technology to market with only Phase 1 clinical trials done only on three patients,” said Souvik Maiti, Director, CSIR-IGIB.

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

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

Explaining the technology in lay terms, the minister described it as a “precise genetic surgery”, capable of not only curing sickle cell disease but also transforming treatment pathways for several hereditary disorders.