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haemophilia A

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

Pfizer hedges bets on gene therapy deal

Pfizer hedges bets on gene therapy deal

Pfizer had looked like the most obvious buyer for Spark, as the two companies are co-developing a gene therapy treatment for haemophilia B. ... Vivet Therapeutics’ lead candidate is VTX-801, a gene therapy for Wilson disease, a devastating rare, chronic

Latest news

  • Roche gets EU nod for much-expanded Hemlibra label Roche gets EU nod for much-expanded Hemlibra label

    The EMA has approved Roche’s Hemlibra for use in the majority of patients with haemophilia A, extending its use beyond the minority who have developed resistance to factor VIII drugs. ... The EU medicines regulator has approved Hemlibra (emicizumab) to

  • Roche buys gene therapy specialist Spark for $4.3bn Roche buys gene therapy specialist Spark for $4.3bn

    clinical trials headed by SPK-8011, a therapy for haemophilia A which is expected to start phase 3 testing before year-end. ... Moreover, the deal also gives it multiple gene therapy candidates for haemophilia, a category the company broke into a couple

  • BioMarin prepares for haemophilia A gene therapy data readout, filing BioMarin prepares for haemophilia A gene therapy data readout, filing

    Key long-term data expected in July. BioMarin has recorded strong growth for its full 2018 results, as it prepares to file a first-in-class gene therapy for haemophilia A ... for severe haemophilia A that demonstrated the elimination of need for

  • Pfizer closes on EU approval of lung cancer drug Vizimpro Pfizer closes on EU approval of lung cancer drug Vizimpro

    Roche claimed a positive opinion for Hemlibra (emicizumab) for routine prophylaxis of bleeding episodes in adults and children with severe haemophilia A without factor VIII inhibitors, greatly extending the number if ... considered cisplatin ineligible,

  • UniQure cleared to start Huntington gene therapy trial UniQure cleared to start Huntington gene therapy trial

    Since then, UniQure has been focusing on bringing a gene therapy for haemophilia B (AMT-061) into mid-stage testing, and the Huntington programme will be its second to enter clinical ... trials ahead of other candidates for haemophilia A and Fabry disease

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Latest Intelligence

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

    lives, with the effect lasting a median of 33 months after just one treatment. ... Gilead and Novartis aside, the only companies with a stake in gene therapy with a higher market capitalisation are BioMarin, in pole position to develop a potentially

  • ‘How is your day?’ ‘How is your day?’

    World champion cyclist Alex Dowsett lives with severe haemophilia. He also speaks for ‘How Is Your Day?’. ... plasma. Haemophilia is a condition that can be treated by both laboratory-produced recombinant plasma therapies or medicines made from

  • Deal Watch January 2018

    s closing price on 19 January), Sanofi gains the marketed products, Eloctate (haemophilia A therapy) and Alprolix (haemophilia B therapy), as well as a pipeline of candidates focused on rare blood ... Portfolio/pipeline in haemophilia and other rare

  • The good, the bad and the ugly The good, the bad and the ugly

    However, investors fear that challenges in the $11bn haemophilia market could lead to a slump in the haemophilia business it inherited through the Baxalta purchase. ... These two players will have a comfortable advantage over their nearest rival - Roche (

  • Attracting doctors to low-demand specialties Attracting doctors to low-demand specialties

    Haemophilia. One of the specialties in which Porterhouse has worked for many years is haemophilia, a disease that finds itself in a somewhat anomalous position. ... such as leukaemias, lymphomas and myelomas, while haemophilia warrants just one or two

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Latest appointments

  • 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

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Latest from PMHub

  • Living with haemophilia – the power of knowledge

    Haemophilia is a genetic disorder that prevents blood from clotting [1]. It is a predominantly male disorder, with haemophilia A occurring in 1 in 5, 000 live male births [1]. ... Untreated, haemophilia can be extremely debilitating, making everyday

  • Attracting doctors to low-demand specialties: A role for industry?

    Haemophilia  .  One of the specialties in which Porterhouse has worked for many years is haemophilia, a disease that finds itself in a somewhat anomalous position. ... such as leukaemias, lymphomas and myelomas, while haemophilia warrants just one or

  • Take a walk on the wild side!

    Say Comms Haemophilia Walk-a-Thon . What is this about a walk on the wild side? ... Sunday 17th April is World Haemophilia Day a very important day for the blood disorder community around the world.

  • Say Communications

    The Say Comms team are raising money for The Haemophilia Society with a walk-a-thon sponsor them here http://bit.ly/1MtvDO8. ... England is out of the 2015 rugby world cup, but is your health a winner?

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