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Gene therapy effective in hemophilia B patients with neutralizing antibodies

Key clinical point: Patients with hemophilia B have maintained increased levels of factor IX activity 1 year after receiving etranacogene dezaparvovec (AMT-061).

Major finding: At 52 weeks, factor IX activity levels were 50.2% in the first patient, 40.8% in the second, and 31.3% in the third.

Study details: A phase 2b trial of three patients with moderate to severe hemophilia B and anti-AAV5 neutralizing antibodies at baseline

Disclosures: The trial is sponsored by uniQure. Dr. Pipe disclosed relationships with uniQure and other companies.

Citation:

Pipe SW et al. EAHAD 2020, Abstract OR10.