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ASH 2024 | The use of RNA interference-based therapies like fitusiran in hemophilia treatment

Tadashi Matsushita, MD, Nagoya University Hospital, Nagoya, Japan, comments on the use of RNA interference-based therapies, like fitusiran, in hemophilia treatment. He notes that these therapies aim to balance coagulation and anticoagulation in patients and have shown promise in reducing thrombogenesis with minimal safety concerns. This interview took place at the 66th ASH Annual Meeting and Exposition, held in San Diego, CA.

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Transcript

Well, the approach is completely different. This type of approach is just decreasing the level of anticoagulant, like in your case, it’s an antithrombin. And there’s another different approach with decreasing the level of TFPIs. And so, kind of like some balancing, to keep the balancing the coagulation and anticoagulation in the hemophilia patient. And also this approach in decreasing the AT3 antithrombin levels under 20% or so, I thought that it’s going to be very effective and just really a safety concern of the thrombogenesis...

Well, the approach is completely different. This type of approach is just decreasing the level of anticoagulant, like in your case, it’s an antithrombin. And there’s another different approach with decreasing the level of TFPIs. And so, kind of like some balancing, to keep the balancing the coagulation and anticoagulation in the hemophilia patient. And also this approach in decreasing the AT3 antithrombin levels under 20% or so, I thought that it’s going to be very effective and just really a safety concern of the thrombogenesis. I heard that the company introduced a new regimen for drug competition and a new regimen for dosing and looking at the AT3 levels between 15 and 25 percent or so. So I heard that this approach is going to be very effective and also very safe.

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Disclosures

Pfizer: Consultancy; Sysmex: Honoraria; CSL: Honoraria; Sanofi: Honoraria; Novo Nordisk: Consultancy, Honoraria, Research Funding; Chugai: Consultancy, Honoraria, Research Funding; Bayer: Consultancy, Honoraria; KM Biologics: Honoraria; JB Pharma: Honoraria; Takeda: Consultancy, Honoraria.