Gene Therapy for Haemophilia A

Recently, India’s first-in-human gene therapy trial for Haemophilia A  was conducted at BRIC-inStem

About Haemophilia A

• Haemophilia A is a rare genetic bleeding disorder caused by a deficiency of clotting protein Factor VIII, due to mutations in the F8 gene on the X chromosome.
• Vulnerability: It primarily affects males; females can be carriers and may occasionally show symptoms if both X chromosomes carry the mutation.
• Symptoms:  It includes excessive bleeding, easy bruising, and joint pain due to internal bleeding.
• Treatment: Traditionally, treatment involved frequent intravenous infusions of clotting factors, but there is no permanent cure without advanced therapies.

About Gene Therapy for Haemophilia

• Gene therapy offers a potential one-time cure by delivering a functional copy of the F8 gene into the patient’s liver cells using a modified adenovirus vector.
• The therapy instructs the liver to produce Factor VIII internally, thereby correcting the clotting deficiency from the source.
• The first commercial gene therapy, Roctavian, was approved by the U.S. FDA in 2023, setting a precedent for similar global trials, including India’s.
• India’s clinical trials mark a turning point in making gene therapy accessible and affordable for the Indian population.
  

This breakthrough positions India at the forefront of next-generation biomedical innovation, with gene therapy emerging as a pillar of future healthcare and economic growth.

DOWNLOAD PDF