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ASCAT 2023 | Overcoming the financial challenge of gene therapy for SCD

Subarna Chakravorty, MBBS, MRCPCH, FRCPath, PhD, King’s College Hospital NHS Foundation Trust, London, UK, addresses the biggest hurdle to implementation of gene therapy for sickle cell disease (SCD): its high cost. This is a particular barrier to providing this treatment in low- to middle-income countries, although there are projects taking place which aim to investigate methods to mitigate this high cost. This interview was recorded at the 18th Annual Scientific Conference on Sickle Cell and Thalassemia (ASCAT) 2023, held in London, UK.

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Transcript (edited for clarity)

So that is, I think, a significant thing. That’s perhaps the most important thing. Right now it is very, very costly. So I imagine that with time and more products coming through with competition and the ability to produce gene therapy products in a very agile and lean way, hopefully the cost will come down with time. If there are other ancillary costs reduced, like you know, if we can somehow bypass the need for severe myeloablation, you know, or not require autologous transplant in a conventional way, that may also reduce cost...

So that is, I think, a significant thing. That’s perhaps the most important thing. Right now it is very, very costly. So I imagine that with time and more products coming through with competition and the ability to produce gene therapy products in a very agile and lean way, hopefully the cost will come down with time. If there are other ancillary costs reduced, like you know, if we can somehow bypass the need for severe myeloablation, you know, or not require autologous transplant in a conventional way, that may also reduce cost. And there are others, you know, some of the low- and middle-income countries are also developing, you know, gene therapy projects through their own sort of state funded research projects where they are looking at how best you can make it a very lean and cheaper way of producing things. So there will be a lot to learn from research that’s coming through from the low- and middle-income countries.

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