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Padua variant factor IX gene shines in AMT-061

Key clinical point: AMT-061, a novel gene therapy, provides clinically meaningful factor XI activity in hemophilia B.

Major finding: Mean factor IX activity level was 38% of normal 12 weeks after gene therapy.

Study details: An ongoing, phase 2b study involving three patients with moderate to severe or severe hemophilia B.

Disclosures: The investigators reported financial relationships with Bayer, UniQure, Pfizer, Novo Nordisk, Shire, and others.

Citation:

Von Drygalski A et al. EAHAD 2019, Abstract OR10.