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Gene Therapy for Haemophilia
July 19 , 2025
16 hrs 0 min
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- The Indian scientists develop novel gene therapy treatment for haemophilia.
- Haemophilia A, a rare hereditary condition, is resulting from a faulty gene.
- It triggers severe, spontaneous, and potentially fatal bleeding episodes.
- Though only tested on five patients in Tamil Nadu so far, none of them have reported bleeding episodes over an average follow-up period of 14 months.
- It is not unusual for those afflicted by haemophilia to have the weekly bleeding episodes, requiring frequent treatment.
- A gene introduced into the body teaches it to create enough of the clotting factor that can prevent such haemorrhage.
- Haemophilia can be classified as minor or severe depending on the percentage of clotting factor present in those afflicted.
- Haemophilia A, the more common version of the condition, is caused by the absence of a blood-clotting factor called Factor VIII.
- Even though haemophilia is a rare disorder, India has the world’s second largest patient pool, with an estimated 40,000 to 100,000 patients.
- There is only one gene therapy — Roctavian — which was approved by the U.S. Food and Drug Administration for commercial use in 2023.
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