ASH 2025 | Expanding access to gene therapy for patients with sickle cell disease

Sickle cell is something which unfortunately lagged behind again. We had lovely three drugs or two approved drugs so to say the third drug was not approved in the UK. Two drugs which were approved but then withdrawn unfortunately and at the moment of course we have the gene therapy. The gene therapy is very expensive and it is again happening in very concentrated centers, which I totally agree and fine with that. But I think what we might need to do is that we need to make it more accessible and we need to fast track these patients. I have got so many patients in our center who are suffering in a big way from sickle cell anemia and gene therapy should be made accessible to these patients much quicker and you might have relaxed the qualifying criteria for these patients because as you can imagine that these patients’ quality of life is quite poor and of course their organ damage will happen more and more as they actually wait longer for the gene therapy so that is my anticipation and expectation again in this area we have, we are making fantastic progress in other rare diseases like hemophilia and thalassemia or sickle cell anemia, we are the second largest center in the country and gene therapy needs to come thick and fast to these patients so that they can be benefited with the new advances in science. This is the most happening time for hematology in the last 10 years and we have seen massive progress and that is good for science, that is good for patients.

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