Question 41
What is the name of India’s first indigenously developed CRISPR gene therapy designed to treat Sickle Cell Disease?
- AOption (a)
GeneHeal Pro
- BOption (b)
BIRSA 101
- COption (c)
CRISPR-Cure
- DOption (d)
SCD Cure X
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Correct answer
BIRSA 101
Why this answer is correct
The correct answer is BIRSA 101.
Key Points
- BIRSA 101 is India’s first indigenously developed gene therapy based on CRISPR technology, specifically designed to treat Sickle Cell Disease (SCD).
- This ground-breaking therapy was developed by the CSIR-Institute of Genomics and Integrative Biology (CSIR-IGIB) located in New Delhi, in collaboration with clinical partners.
- In 2024, the Central Drugs Standard Control Organisation (CDSCO) granted approval for Phase I/II clinical trials of BIRSA 101, marking a significant milestone in Indian biotechnology.
- The therapy utilizes the CRISPR-Cas9 (Clustered Regularly Interspaced Short Palindromic Repeats) gene-editing tool to correct the genetic mutation in the HBB gene that causes the production of abnormal hemoglobin.
- The development of BIRSA 101 is a major step toward Atmanirbhar Bharat (self-reliant India) in the field of advanced medical treatments, aiming to provide a one-time cure for a disease that currently requires lifelong management.
- Hence, the correct answer is BIRSA 101.
Additional Information
- Sickle Cell Disease (SCD):
- It is a genetic blood disorder characterized by red blood cells that assume an abnormal, rigid, sickle-like shape.
- This condition leads to anemia, severe pain crises, organ damage, and a reduced life expectancy due to the blockage of blood flow in small vessels.
- In India, the disease is particularly prevalent among tribal populations in states like Madhya Pradesh, Chhattisgarh, Odisha, and Gujarat.
- National Sickle Cell Anaemia Elimination Mission:
- Launched by the Government of India in July 2023, this mission aims to eliminate Sickle Cell Disease as a public health problem by the year 2047.
- The mission focuses on screening 7 crore people in the age group of 0-40 years in 278 districts across the country.
- CRISPR-Cas9 Technology:
- This technology acts as "molecular scissors" that can cut DNA at specific locations, allowing scientists to remove, add, or alter sections of the DNA sequence.
- The Nobel Prize in Chemistry was awarded in 2020 to Emmanuelle Charpentier and Jennifer Doudna for the development of this method for genome editing.
Important Points
- Naming and Significance:
- The therapy BIRSA 101 is named in honor of the legendary tribal freedom fighter Birsa Munda, reflecting the focus of the treatment on the tribal communities who are disproportionately affected by SCD.
- The indigenous nature of the project is expected to significantly reduce the cost of treatment compared to international gene therapies like Casgevy, which can cost millions of dollars per patient.
- Mechanism of Action:
- The therapy involves ex-vivo gene editing, where a patient’s blood stem cells are harvested, modified using CRISPR-Cas9 in a laboratory to increase the production of fetal hemoglobin (HbF), and then infused back into the patient.
- Increased levels of fetal hemoglobin prevent the sickling of red blood cells, effectively neutralizing the symptoms of the disease.