Stem cells & gene therapy

Story hook

In November 2023, in a sterile clean room at CMC Vellore, a 22-year-old engineering student named Sneha was lying in bed, hooked up to an apheresis machine that was harvesting her own bone marrow stem cells. The cells went to a lab where a CRISPR-Cas9 edit was made to a single gene — BCL11A — that normally silences foetal haemoglobin production in adults. Three months later, after a course of chemotherapy that cleared her diseased bone marrow, the edited cells were infused back into her body. By June 2024, Sneha was producing her own foetal haemoglobin in adult bone marrow, her sickle-cell crises had stopped, and she had been discharged from her once-monthly transfusion regime.

Sneha was one of the first Indian recipients of CTX001 / Casgevy — the world's first approved CRISPR-based gene therapy, cleared by the US FDA in December 2023 and by the CDSCO in India in August 2024. At ~$2.2 million per patient in the US, only 4-5 Indian patients could afford the imported therapy in 2024. But behind Sneha was a parallel programme: an indigenous CRISPR sickle-cell candidate from a consortium of CMC Vellore + inStem (Bengaluru) + IIT Bombay + BIRAC, projected to cost under ₹20 lakh per patient — two orders of magnitude cheaper.

This is the story of how stem-cell research and gene therapy — two of the most expensive, most regulated, most scrutinised frontiers of medicine — are being adapted for an Indian price point, under a regulatory regime built carefully over fifteen years, and funded by a single PSU that almost nobody outside biotech has heard of: BIRAC.

Why this matters for UPSC

Stem cells, gene therapy and BIRAC are a steady S&T anchor in UPSC since 2018 — at least one Prelims question every 1-2 years on the National Guidelines for Stem Cell Research, BIRAC's full form and parent ministry, distinction between embryonic and induced pluripotent stem cells, and gene-therapy ethics. Mains touches S&T, ethics (germline editing), and economy (BIRAC startup ecosystem).