Fitusiran (ALN-AT3SC)

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Mechanism of action

siRNA therapeutic, targets antithrombin

Preliminary data

Inherited coagulopathy

  1. ATLAS-A/B: Srivastava A, Rangarajan S, Kavakli K, Klamroth R, Kenet G, Khoo L, You CW, Xu W, Malan N, Frenzel L, Bagot CN, Stasyshyn O, Chang CY, Poloskey S, Qiu Z, Andersson S, Mei B, Pipe SW. Fitusiran prophylaxis in people with severe haemophilia A or haemophilia B without inhibitors (ATLAS-A/B): a multicentre, open-label, randomised, phase 3 trial. Lancet Haematol. 2023 May;10(5):e322-e332. Epub 2023 Mar 29. link to original article PubMed NCT03417245