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haemophilia B

This page shows the latest haemophilia B news and features for those working in and with pharma, biotech and healthcare.

FDA removes clinical hold on uniQure’s haemophilia gene therapy

FDA removes clinical hold on uniQure’s haemophilia gene therapy

The US Food and Drug Administration (FDA) has removed a clinical hold on Dutch biotech uniQure’s haemophilia B gene therapy, following concerns over a case of cancer in a patient ... In December 2020, the FDA placed a hold on uniQure’s haemophilia B

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  • 20 for 2020 – Five pharma leaders to watch in 2020 20 for 2020 – Five pharma leaders to watch in 2020

    pipeline. The company submitted its lumasiran candidate for primary hyperoxaluria to the FDA and has another ATTR amyloidosis candidate – called vutrisiran – in late-stage along with Sanofi-partnered fitusiran for haemophilia

  • Deal Watch January 2018

    Paying $105 per share in cash (64% premium over Bioverativ’s closing price on 19 January), Sanofi gains the marketed products, Eloctate (haemophilia A therapy) and Alprolix (haemophilia B therapy), as ... Portfolio/pipeline in haemophilia and other

  • Deal Watch August 2016 Deal Watch August 2016

    premiums. One commentator suggested BioMarin could be a Plan B for Sanofi. ... This will help with the collaboration with Spark Therapeutics that is developing a treatment for haemophilia B that incorporates a bio-engineered rAAV vector and the academic

  • A rare opportunity A rare opportunity

    Sobi, handing Sobi commercialisation control over the haemophilia A treatment just four months after it received EU approval. ... Last month Alprolix was approved by the European Commission, becoming the first licenced long-acting treatment for

  • Deal Watch February 2016 Deal Watch February 2016

    Indeed Baxalta already has ongoing gene therapy programmes for the treatment of haemophilia A and B.

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