Casgevy – Gene Editing Tool
Introduction:
- Casgevy, which employs the gene-editing technique CRISPR-Cas 9, was approved for use last week in the UK, marking a significant milestone for the millions of people worldwide, including those in India, who suffer from sickle cell anaemia.
About gene editing:
- With the use of a process called gene editing, scientists may precisely alter an organism’s DNA.
- It is possible to achieve this by altering, deleting, or inserting particular DNA sequences.
- Gene editing has several potential uses, such as the generation of novel animals and crops, new biofuels, and the development of novel disease therapies.
What is anaemia with sickle cells?
- A class of hereditary blood illnesses known as sickle cell anaemia alters the structure of red blood cells.
- Red blood cells often flow through blood channels with ease since they are round and flexible.
- Certain red blood cells with sickle cell anaemia have a sickle or crescent moon shape.
- Additionally, these sickle cells harden and cling, which can hinder or delay blood flow.
Treatment options for sickle cell anaemia
- Sickle cell anaemia cannot be cured, however, some therapies can help control the condition’s symptoms and avoid consequences.
- The disease’s current treatment, hydroxyurea chemotherapy, lowers the frequency of patient problems.
More on the ruling by the UK regulator:
- Based on Casgevy’s remarkable capacity to completely eradicate excruciating inflammatory attacks in over 90% of trial participants, the UK authority made its conclusion.
- The go-ahead might open the door for approvals by other regulatory agencies and the US FDA, which is scheduled to make a judgement on December 8.
Difficulties in ensuring that everyone can receive therapy:
- It’s a complex process to make space in the body for altered cells.
- Although the company that makes Casgevy, Vertex, has not provided a price for the therapy, estimates from the US and the UK place the average cost of treatment for a single patient at $2 million.
How is the new treatment carried out?
- The oxygen-carrying protein in red blood cells, haemoglobin, is damaged in sickle cell disease. Normal hemoglobin-containing red blood cells have a disc shape and are malleable.
- They pass easily through the blood vessels. The cells lacking haemoglobin are nearly identical in comparison.
- The form of these cells becomes crescent when they run out of oxygen. They pass away far faster than the average person.
- Because of the way they are shaped, sickle cells adhere to blood vessels, obstructing the flow of blood that carries oxygen and posing a serious threat to life.
- Patients develop chronic anaemia due to a decrease in the number of healthy red blood cells in their bodies. The illness has a genetic component.
- The mutation causing the sickness is not eliminated by casgevy.
- However, its purpose is to make up for the haemoglobin by bringing back the foetal haemoglobin, which is turned off soon after birth.
Sickle cell anaemia and India:
- Sickle cell disease is the second most common disease in India, primarily affecting the nation’s tribal population.
- To address the difficulties caused by the disease, the National Sickle Cell Anaemia Elimination Programme was proposed in the Union Budget 2023.
- The initiative, which was conceived of as the first gene therapy findings were beginning to come in, intends to eradicate the illness by 2047.
- Casgevy appears to be too expensive for the nation at the moment. Research institutes ought to investigate the potential of gene editing in order to create reasonably priced treatments.
- In conclusion, organisations dedicated to gene research were crucial to the administration of Covid. However, by all accounts, cell editing therapy research is still in its infancy in India. Casgevy’s achievement ought to encourage funding for this area.
