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haemophilia

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

FDA expecting boom in cell and gene therapies

FDA expecting boom in cell and gene therapies

The FDA says it also planning a series of clinical guidance documents to help guide the development of cell and gene therapies, including some targeted at specific diseases such as haemophilia

Latest news

  • Chugai gains FDA breakthrough status for rare CNS treatment Chugai gains FDA breakthrough status for rare CNS treatment

    Chugai is part of the Roche group, and the announcement marks the seventh Breakthrough Designation Therapy (BTD) received for four drugs discovered by the Japanese firm, which include haemophilia treatment Hemlibra.

  • Bayer axes 1,250 pharma jobs, with R&D taking the brunt Bayer axes 1,250 pharma jobs, with R&D taking the brunt

    Another 350 pharma jobs will go as a result of a decision not to make use of a recently-completed manufacturing facility for haemophilia treatment factor VIII in Wuppertal. ... In its statement, Bayer said the introduction of a number of new products has

  • UniQure skyrockets on haemophilia B gene therapy data UniQure skyrockets on haemophilia B gene therapy data

    Competition with Pfizer and Spark rival heats up. It’s a measure of the anticipation about gene therapy that data positive data in three haemophilia patients caused UniQure shares to close ... The phase 2b results for UniQure’s AMT-061 candidate for

  • AZ cuts $1.5bn-plus deal to pass US drug rights to Sobi AZ cuts $1.5bn-plus deal to pass US drug rights to Sobi

    The deal represents a major expansion for Sobi, which is currently focused on rare diseases and haemophilia in Europe (the latter through a licensing deal with Bioverativ), with limited penetration into

  • BioMarin eyes 2019 FDA filing for haemophilia A gene therapy BioMarin eyes 2019 FDA filing for haemophilia A gene therapy

    Rare disease specialist BioMarin is hoping to file for approval its haemophilia A gene therapy valoctocogene roxaparvovec (valrox) in the second half of next year, if its ongoing phase 1/2 ... The California biotech said during an R&D update yesterday

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Latest appointments

  • Sobi appoints Torbjorn Hallberg as general counsel Sobi appoints Torbjorn Hallberg as general counsel

    Also sitting on the new team are Sobi's head of specialty care Norbert Oppitz, h ead of medical and scientific affairs Armin Reininger, h ead of haemophilia Philip Wood and

  • Anne Prener to lead Freeline Therapeutics Anne Prener to lead Freeline Therapeutics

    Freeline comprises some of the world’s leading experts in gene therapy, haemophilia and other debilitating disorders and I look forward to working closely with them to demonstrate the potential of ... She has also worked at Novo Nordisk as senir vice

  • Clark Health Communications expands team Clark Health Communications expands team

    Campbell will support and assist the team across its women’s health, gastroenterology and haemophilia accounts.

  • Shire’s Dr Armin Reininger moves to Sobi Shire’s Dr Armin Reininger moves to Sobi

    global medical affairs for haemophilia. ... Armin will apply his deep experience as an industry leader and academic in haemophilia to our team, as well as his global experience in medical scientific affairs to our stakeholders.”.

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