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Source: The post is based on the article “Casgevy: How gene therapy will revolutionize treatment of sickle cell disease” published in “Indian Express” on 25th November 2023
Why in the News?
The UK drug regulator has approved a gene therapy called Casgevy for the cure of sickle cell disease and thalassaemia.
What is Casgevy?
| Specifications | Details |
| What is it? | It is a CRISPR Cas9 based gene-edited therapy developed for the treatment of Sickle cell disease and Thalassaemia. |
| Working | 1) Casgevy is a one-time treatment. 2) The treatment involves removing stem cells out of bone marrow from a patient’s blood using a process called apheresis that filters out the blood for different components. 3) CRISPR gene editing technology is then used in a laboratory to edit the faulty gene. 4) The edited cells are then infused back into the patient, allowing the body to produce functioning haemoglobin. |
| Target Gene | – A gene called BCL11A which is crucial for switching from foetal to adult haemoglobin is targeted by the therapy. – Note: Foetal haemoglobin which is naturally present in everyone at birth, does not carry the same abnormalities as adult haemoglobin. – The therapy uses the body’s own mechanisms to start producing more of this foetal haemoglobin alleviating the symptoms of the two conditions. |
| Challenges | 1) High Cost is one of the biggest limitations of gene therapies. 2) Absence of local manufacturing facilities which means that the harvested blood stem cells have to be sent across countries. |
| Significance | This is the first licensed therapy in the world based on the gene editing technology Crispr-Cas9 that earned its innovators a Nobel Prize in 2020. |
What is CRISPR Cas9 Technology?
What is Sickle Cell Disease?
| Specifications | Details |
| What is it | It is an inherited red blood cell disorder that affect hemoglobin, a protein that carries oxygen through the body. |
| Characteristics of the disease | 1) Normally, red blood cells are disc-shaped and flexible enough to move easily through the blood vessels. 2) In sickle cell disease, red blood cells become crescent- or “sickle”-shaped due to a genetic mutation. 3) Such a shape makes the flow of hemoglobin through the blood vessels difficult and can block blood flow to the rest of the body. 4) The blocked blood flow through the body can lead to serious problems, including stroke, eye problems, infections, and episodes of pain called pain crises. |
| Treatment | Bone Marrow Transplant |
| Initiatives | National Sickle Cell Anaemia Elimination Mission (NSCEM) |
What is Thalassaemia?
1) Thalassaemia is a chronic and genetic blood disorder due to which a patient’s body cannot make enough hemoglobin leading to anemia.
2) This disease is passed from parents to children through genes and people suffering from it require blood transfusions to survive.
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