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CSL Behring

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First NHS patient treated with CSL Behring’s haemophilia B gene therapy Hemgenix

The genetic bleeding disorder affects more than 2,000 people in the UK

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EC approves CSL’s Andembry to prevent hereditary angioedema attacks

Approximately one in every 50,000 people worldwide are affected by the rare genetic disorder

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CSL Behring announces four-year results for haemophilia B gene therapy Hemgenix

The genetic bleeding disorder affects an estimated one per every 19,283 male births in the US

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MHRA approves CSL’s Andembry to treat angioedema attacks in HAE patients

The rare genetic condition occurs in approximately one in every 50,000 people in the UK

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Pfizer’s one-time haemophilia B gene therapy Durveqtix granted EC approval

More than 42,000 people globally are living with the rare bleeding disorder

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NICE recommends CSL Behring’s haemophilia B gene therapy Hemgenix

More than 2,000 people in the UK are currently living with the genetic bleeding disorder

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Pfizer’s one-time haemophilia B therapy Durveqtix receives CHMP recommendation

More than 38,000 people worldwide are currently affected by the inherited bleeding disorder

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NICE publishes draft guidance not recommending CSL’s haemophilia B gene therapy

The company has been seeking approval for use of the therapy in adults with severe or moderately severe haemophilia B without a history of factor IX inhibitors

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EC approves first gene therapy for adults with haemophilia B

Typically, haemophilia B patients must adhere to strict, lifelong IV infusion schedules

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Pfizer announces positive phase 3 results for haemophilia B gene therapy

A one-off, intravenous dose of the therapy candidate reduced bleeding by 71%

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FDA approves first gene therapy for adults with haemophilia B

CSL’s Hemgenix allowed patients to produce mean factor IX activity of 39% at six months

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FDA removes clinical hold on uniQure’s haemophilia gene therapy

Clinical hold placed following a case of hepatocellular carcinoma in a patient in the trial

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