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Fri, Jan

Gene Therapy breakthrough brings New Hope to Haemophilia Patients

Allied Healthcare (GAHC)
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A breakthrough in gene therapy for haemophilia has shown promising results in an Indian clinical trial, offering hope to thousands of patients affected by the condition. The trial, which included 22 participants, demonstrated significant improvements, with bleeding episodes reduced by 50-60% and some patients achieving complete elimination of bleeding. This marks a major advancement in the treatment of haemophilia, potentially transforming how the condition is managed.

The therapy functions by introducing a functional gene into the patient’s system, enabling sustained production of the clotting factor necessary for blood coagulation. This approach offers a long-term solution compared to traditional treatments, which typically involve frequent injections of clotting factor concentrates. Patients who received the gene therapy in the trial showed a sustained expression of clotting factors, reducing the severity and frequency of symptoms.

While the medical outcomes are groundbreaking, the high cost of the therapy—estimated between ₹1.5 crore to ₹2 crore per patient—poses a significant challenge. The steep expense has raised concerns about accessibility, particularly in India, where many patients lack the financial resources for such advanced treatments. The high costs stem from the complexity of developing and producing gene therapies, making it inaccessible to a majority of patients in need.

This breakthrough has also highlighted the urgency for policy interventions to ensure equitable access to such life-changing treatments. Calls are growing for government initiatives to subsidize costs, encourage public-private partnerships, and invest in local production to make the therapy more affordable. Healthcare experts emphasize the need for a robust regulatory framework to ensure the safe and effective deployment of gene therapies in India while addressing the affordability gap.

For India’s haemophilia community, this advancement represents a significant step forward, signaling the possibility of a life with fewer medical complications and improved quality of life. The potential for long-term benefits could ease the physical and emotional burdens faced by patients and their families. However, making this breakthrough widely accessible will require coordinated efforts from policymakers, healthcare providers, and researchers.

If challenges related to cost and accessibility are addressed, this therapy could usher in a transformative era in haemophilia treatment, offering hope and relief to thousands of patients across the country.