​New Breakthrough in Haemophilia Treatment

Recently, Indian researchers have successfully developed a gene therapy treatment for severe haemophilia A, showing promising results in a trial, with five patients reporting no bleeding episodes for over a year.

The findings have been published in a peer-reviewed journal in December 2024.

• The gene therapy trial was conducted on five patients in Tamil Nadu, who have not reported any bleeding episodes for an average follow-up period of 14 months.
• The treatment uses a lentivirus vector to introduce the clotting factor gene, which is considered a safer alternative to the adenovirus vector used in Roctavian, a gene therapy approved by ....