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UniQure

This page shows the latest UniQure 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

In a statement, uniQure said that the patient diagnosed with HCC had ‘multiple risk factors’ associated with this type of cancer. ... UniQure added that all patients in its haemophilia clinical programme have abdominal ultrasounds performed one year

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  • Bluebird: on a mission to ‘recode’ the DNA of healthcare Bluebird: on a mission to ‘recode’ the DNA of healthcare

    Gene therapy failures. The industry and Europe’s healthcare systems are wary of that magic one million threshold, thanks to the failure of the first ever gene therapy, UniQure’s Glybera. ... The success or failure of Bluebird will be closely watched

  • Bluebird Bio: on the cusp of a gene therapy revolution Bluebird Bio: on the cusp of a gene therapy revolution

    success. Europe has already seen two gene therapies approved but fail commercially – UniQure’s Glybera and GSK’s Strimvelis – and so Bluebird has to learn lessons on pricing and reimbursement and ... The EMA approved the world’s first ever

  • Rare diseases: meeting the challenge Rare diseases: meeting the challenge

    Unfortunate message. Some of these thorny problems were encapsulated in uniQure’s recent decision not to seek an extension to its five-year European marketing authorisation - which expires in October - for

  • Pharma deals in April 2015 Pharma deals in April 2015

    BMS will also take a 4.9% equity stake in uniQure which will cost around $32m, with another 5% ownership scheduled before the end of the year (at 10% premium). ... UniQure also stands to receive research, development and regulatory milestones, including

  • Medius Deal Watch table for April 2015 Medius Deal Watch table for April 2015

    32, 700. uniQure NV/ Bristol-Myers Squibb. R&D collaboration plus equity investment.

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  • Solaris Health looks at the gene therapy revolution Solaris Health looks at the gene therapy revolution

    There has already been one notable disappointment. In 2017 uniQure pulled its gene therapy, Glybera (alipogene tiparvovec) for the rare inherited disorder lipoprotein lipase deficiency (LPLD) from Europe after 5 years,

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