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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.

BioMarin ticks over in Q2 ahead of gene therapy filing

BioMarin ticks over in Q2 ahead of gene therapy filing

all-important filing of what will be a first-in-class gene therapy for haemophilia A later this year. ... The game-changer for the company, however, is its gene therapy for haemophilia A,  valoctocogene roxaparvovec, known as valrox.

Latest news

  • Dupixent drives Sanofi again, but other new drugs are lagging Dupixent drives Sanofi again, but other new drugs are lagging

    A lot of the recovery is due to Regeneron-partnered Dupixent, which contributed 496m in the quarter – a rise of 181%. ... There was bad news for Genzyme’s blood disorders unit, however. Long-acting haemophilia A drug Eloctate – acquired as part of

  • ACPs to lead gene therapies in haemophilia market growth ACPs to lead gene therapies in haemophilia market growth

    GlobalData predicts the wave of novel treatments for haemophilia A and haemophilia B market will see the market grow from $6.93bn in 2018 to $9.29bn in 2028. ... That means even though potential gene therapies for both haemophilia A and B could offer

  • New results back Sangamo, UniQure haemophilia gene therapies New results back Sangamo, UniQure haemophilia gene therapies

    Encouraging results for firms in four-way race. Sangamo and UniQure have continued to build the case for their one-shot gene therapies for haemophilia A and B, with new data ... Sangamo presented updated results from a phase 1/2 Alta study of its SB-525

  • EMA clears Novo Nordisk’s long-acting haemophilia A drug EMA clears Novo Nordisk’s long-acting haemophilia A drug

    Entering newly competitive market. Novo Nordisk has gained EU approval for its new haemophilia A treatment Esperoct, a long-acting factor VIII replacement therapy that is part of the company’s ... Mads Krogsgaard Thomsen. “We are confident that

  • BioMarin eyes haemophilia therapy filing, but data scares investors BioMarin eyes haemophilia therapy filing, but data scares investors

    Doubts remain on longer term efficacy. BioMarin has reported three-year data on its gene therapy for haemophilia A that sets up filings in Europe and the US, but also raises ... BioMarin’s therapy, called valoctocogene roxaparvovec or ‘valrox’,

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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

  • Am I wrong to be apprehensive about gene therapy?

    So, a degree of caution, then. Dr Hart gave a practical overview of how the Haemophilia Centre at the Royal London Hospital, which has been a pivotal trial centre, has had ... His response to the congress audience was that while phenotypically they do

  • 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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