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Source: The post is based on the article “Lyfgenia and Casgevy: U.S FDA approves gene therapies for sickle cell disease” published in “The Hindu” on 9th December 2023
Why in the News?
The U.S Food and Drug Administration (FDA) has approved a pair of gene therapies named Lyfgenia and Casgevy for the treatment of sickle cell disease.
What is Lyfgenia?
| Specifications | Details |
| What is it | Lyfgenia is a cell-based gene therapy developed for the treatment of sickle cell disease. |
| Developed by | Bluebird Bio |
| Eligibility | The therapy is approved for people aged 12 years and older. |
| Treatment process | 1) The treatment involves removing stem cells out of bone marrow from a patient’s blood. 2) The stem cells are then genetically modified to produce HbAT87Q. It is a gene-therapy-derived haemoglobin that functions similarly to haemoglobin A or the normal adult haemoglobin produced by those who do not have sickle cell disease. 3) The modified stem cells are then delivered to the patient in a one-time, single-dose infusion. |
What is Casgevy?
What is Sickle Cell Disease?
1) It is a genetic red blood cell disorder that affects the shape of the hemoglobin, a protein that carries oxygen through the body.
2) The RBCs become crescent- or “sickle”-shaped which makes the flow of hemoglobin through the blood vessels difficult, blocking the blood flow to the rest of the body.
UPSC Syllabus: Science and Technology
