ForumIAS LATEST
- 04 June | MGP Strategy Series | GS Paper 4 (Ethics) with AIR 7 A.R. Rajah Mohaideen Click Here to register for the session →
- 04 June | GS Advance Program begins from 4th June 2026 | First 2 classes open to all Click Here to register for the event →
- 05 June | MGP Strategy Series | GS Paper 3 Strategy Session with AIR 406 Mannat Luthra Click Here to register for the session
- 06 June | Open Orientation on Essay Guidance Program (EGP 2026) Click Here to register →
- 07 June | Open Orientation for Current Affairs for Mains 2026 Click Here to register →
- 07 June | Sociology Optional Strategy Session with AIR 10 Ujjwal Priyank Click Here to register →
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
