BIRSA-101

NEWS – India has achieved a major scientific milestone with the development of an indigenous gene-editing tool by CSIR–IGIB. The tool, now transferred to the Serum Institute of India (SII), is set to revolutionise treatment for sickle cell disease through an affordable, home-grown therapy named Birsa-101.

KEY HIGHLIGHTS

• Indigenous CRISPR-based tool developed entirely by Indian scientists; all patents held domestically.
• Birsa-101 therapy targets the root genetic mutation of sickle cell disease.
• Manufacturing unit established at IGIB; Phase I trials expected next year in collaboration with AIIMS.
• SII to lead Phase II & III trials once initial safety is confirmed.
• Cost-effective alternative to global therapy Casgevy (approx. USD 2.2 million).

WHY THIS MATTERS

High Disease Burden

• India records 30,000–40,000 new sickle cell cases annually.
• Most prevalent among tribal populations.
• Government mission targets elimination of the disease by 2047 through screening and awareness.

ABOUT BIRSA-101

Mechanism

• Uses precise gene editing to correct the mutation causing sickle cell disease.
• Delivered as a single-dose infusion.
• Promotes normal red blood cell production, preventing sickling.

How It Differs from Casgevy

• Birsa-101 corrects the genetic error directly.
• Casgevy increases levels of foetal haemoglobin to bypass the defect.
• Same approach of Birsa-101 can be extended to thalassaemia therapy.

DEVELOPMENT OF THE INDIGENOUS GENE EDITING TOOL

Scientific Advancements

• Based on highly specific Cas9 proteins sourced from bacterial immune systems.
• Engineered to minimise off-target effects, ensuring safety and precision.
• The platform has been shared with SII on a non-exclusive basis, enabling wider use.

Future Prospects

• Initial rollout for patients with severe disease or organ damage.
• Potential later approval for paediatric use.
• Possibility of multiple therapies for genetic disorders using the same tool.