India has launched its first indigenous CRISPR-based gene therapy, BIRSA 101, for Sickle Cell Disease (SCD).

About “BIRSA 101” Therapy

• Name and Inspiration: The gene therapy is named BIRSA 101 in honour of Bhagwan Birsa Munda, a revered tribal freedom fighter.
• Technology Platform: The therapy uses CRISPR-Cas9 technology, a precision genetic surgery that not only aims to cure Sickle Cell Disease but also has potential applications for other hereditary disorders.
• Affordability: Developed as a low-cost alternative, BIRSA 101 has the potential to replace global treatments priced at ₹20-25 crore with a more affordable solution tailored for Indian conditions.
• Developed by: It has been developed by the CSIR–Institute of Genomics and Integrative Biology (IGIB) located in New Delhi.
  • IGIB has partnered with the Serum Institute of India (SIIPL) to scale up the CRISPR platform into affordable therapies for Sickle Cell Disease
• Working Mechanism:
  • Genetic Correction: The therapy edits the defective part of the genetic code that causes the disease.
  • One-Time Treatment: It is administered as a single infusion, after which the body begins producing normal red blood cells instead of sickle-shaped cells.

About CRISPR-Cas9 technology

• CRISPR-Cas9 is a gene-editing technology that allows precise, targeted alterations (removing, adding, or altering) in the DNA of living organisms.
• The CRISPR-Cas9 system has two main components:
  • Cas9 enzyme: Acts as molecular scissors, cutting both strands of DNA at a targeted site in the genome.
  • Guide RNA (gRNA): A short, pre-designed RNA sequence that directs Cas9 to the specific DNA location by binding to the target sequence.
• Once the DNA is cut, the cell’s natural repair mechanisms are used to delete, insert, or replace genetic material, enabling precise genome editing.

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About Sickle Cell Disease (SCD)

• Definition: Sickle Cell Disease (SCD) is a genetic blood disorder where the body produces abnormal hemoglobin (hemoglobin S), leading to the formation of sickle-shaped red blood cells that disrupt blood flow.
• Causes: SCD is inherited in an autosomal recessive pattern. Both parents must carry the defective gene for a child to inherit the disease.
  • Hemoglobin S causes red blood cells to become rigid and crescent-shaped, which can block blood flow and cause pain and organ damage.
• Symptoms: Pain crises (episodes of severe pain), anemia, fatigue, swelling in hands and feet, frequent infections, delayed growth, and vision problems are common symptoms.
• Government Initiative: The Government of India launched the National Sickle Cell Anaemia Elimination Mission (2023–2030) to strengthen screening, prevention, and treatment mechanisms.
  • Vision: The Vision of the Mission is to eliminate sickle cell disease as a public‑health problem in India before 2047.
  • Goal: A key early goal is to screen approximately 7 crore (70 million) people aged up to 40 years in identified tribal‑dominated and high‑burden states by FY 2025‑26.
  • Focus States: The mission currently covers 17 high‑focus states/UTs, many with significant tribal populations and high prevalence of SCD 
• High Burden Areas in India: Tribal-dominated regions of Odisha, Chhattisgarh, Jharkhand, Maharashtra, Gujarat, and Madhya Pradesh.

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