SYLLABUS

GS-3: Awareness in the fields of IT, Space, Computers, robotics, nano-technology, bio-technology and issues relating to intellectual property rights 

Context: 

Union Minister of State (Independent Charge) for Science & Technology has launched India's first indigenous CRISPR based gene therapy for Sickle Cell Disease.

More on the News

• India has launched its first indigenous CRISPR-based gene therapy for Sickle Cell Disease (SCD), named BIRSA 101, marking a milestone in the country’s biotechnology sector.
• The launch coincides with the 150th birth anniversary of tribal leader Birsa Munda, underscoring the therapy’s symbolic and social relevance to India’s tribal populations most affected by SCD.
• This initiative aligns with the National Sickle Cell Anaemia Elimination Mission (2023–2030), aiming for early screening, holistic management, and eventual eradication of the disease.

About the BIRSA 101 

• BIRSA 101 uses CRISPR-Cas9 gene-editing technology to correct the defective beta-globin gene responsible for Sickle Cell Disease.
• The procedure involves collecting bone marrow stem cells from patients, editing the defective gene in a controlled lab environment, and reinfusing the corrected cells into the patient.
• Birsa-101 precisely corrects the disease-causing genetic mutation and is delivered as a one-time infusion, enabling the body to produce normal red blood cells instead of sickle-shaped ones.

Significance of the Technology  

• Indigenous Innovation: India demonstrates strong scientific self-reliance in advanced biomedical research and precision medicine.
• Affordable Therapy: The therapy provides treatment at a fraction of global costs, helping democratize access to life-saving gene therapies.
• Biotech Strengthening: It strengthens the country’s biotech innovation ecosystem, supporting policies of the Department of Biotechnology and the Make-in-India framework.
• Healthcare Transformation: The gene-editing approach works like a “precise genetic surgery”, capable not only of curing Sickle Cell Disease but also transforming treatment pathways for several hereditary disorders.
• Equitable Public Health: It reinforces India’s commitment to equity in public health, aligning scientific progress with social justice by prioritizing underserved communities.

Challenges Associated with the Technology 

• Skilled Expertise: Gene-editing therapies demand highly skilled expertise, specialized lab infrastructure, and stringent biosafety oversight.
• Ethical Concerns: Ethical considerations over germline editing, long-term genomic stability, and equitable access remain global concerns.
• Regulatory Evolution: Regulatory frameworks in India are still evolving; consistent oversight from agencies like the Genetic Engineering Appraisal Committee (GEAC) and the Indian Council of Medical Research (ICMR) is needed.
• Implementation Challenge: Ensuring affordability and scalability of CRISPR therapies beyond major research hospitals poses implementation challenges.
• Reactive Approach: Sustained public awareness and preclinical screening are vital to ensure early intervention and prevention rather than reactive treatment.

CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats)

• CRISPR is a revolutionary gene-editing tool derived from bacterial immune defense systems that allows precise, targeted changes to DNA sequences.
• The Cas9 enzyme acts as molecular scissors, cutting the DNA at a desired location so genetic material can be added, removed, or altered.
• In the context of Sickle Cell Diseases, it corrects the mutated haemoglobin (HbS) gene responsible for deformed red blood cells that cause oxygen transport issues and organ damage.
• The technology is celebrated for being faster, cheaper, and more accurate compared to older gene-editing methods.
• CRISPR/Cas9 gene-editing tool was awarded 2020 Nobel Prize in Chemistry. 

Sickle Cell Disease (SCD) 

• Sickle Cell Disease is a genetic blood disorder where Red blood cells assume an abnormal crescent shape, leading to blockages, anaemia, pain episodes, and organ complications.
• It primarily affects India’s tribal populations, with over 20 million carriers and a significant number of affected children born each year.
• The disease exacerbates health inequities, as access to consistent treatment in tribal and rural regions remains limited.

Sources:
Indian Express
Medline Plus
Pib. Gov