Source: IE
Context: The UK drug regulator recently approved Casgevy, a gene therapy for sickle cell disease and thalassaemia, marking a breakthrough as the world’s first licensed therapy using Crispr-Cas9 gene editing technology.
What is Casgevy?
It is a groundbreaking gene-editing therapy for sickle cell anaemia, utilizing the CRISPR-Cas9 tool. Casgevy edits the faulty gene causing these blood disorders, potentially providing a lifelong cure. The therapy, targets the BCL11A gene to increase foetal haemoglobin production, reducing severe pain crises and the need for transfusions.
However, challenges include the high potential cost (estimated up to $2 million per patient) and the necessity for improved local manufacturing facilities to make the therapy more accessible globally
About Sickle Cell Disease:
| Description | ||
| What is Sickle Cell Anaemia? | It is an inherited blood disease common among people of African, Arabian, and Indian origin. It affects haemoglobin, delivering oxygen to cells. | 
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| A typical haemoglobin molecule (haemoglobin S) distorts red blood cells, causing a sickle shape, and blocking blood flow and oxygen. | ||
| Symptoms include severe pain (sickle cell crises) and organ damage over time. | ||
| Treatment | Medication, blood transfusions, and occasionally bone marrow transplants. | |
| Implications | High Prevalence: 30,000 to 40,000 children are born with sickle cell disease annually in India, mostly in tribal areas | |
| Government Initiatives | India aims to eliminate the SCA by 2047 through a national program. | |
| National Sickle Cell Anaemia Elimination Programme | ||
| ICMR and NRHM implement outreach programs for disease management and control | ||
| Universal screening, awareness, and counselling funded under the National Health Mission | ||
