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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.

Sanofi narrows focus, shedding diabetes and cardiovascular research

Sanofi narrows focus, shedding diabetes and cardiovascular research

This includes a host of investigational therapies for rare disease and cancers, such as Fitusiran (an RNAi therapeutic for haemophilia A and B), BIVV001 (a factor VIII therapy for haemophilia A) ... year. Its insulin Lantus was the top selling product

Latest news

  • Sangamo steps up BioMarin chase with haemophilia gene therapy data Sangamo steps up BioMarin chase with haemophilia gene therapy data

    Updated results presented at ASH 2019. Sangamo has been playing catch-up with BioMarin in the race to develop a gene therapy for haemophilia A, but new data reported at the ... Sangamo is adamant that it is still in contention in the haemophilia A gene

  • Roche moving closer to approval of Spark takeover; report Roche moving closer to approval of Spark takeover; report

    The  concern among shareholders of both companies is that the financial regulators might be concerned about Roche/Spark’s potentially dominant position in haemophilia, so they will be cheered by a ... neparvovec) for a rare inherited retinal disease

  • Sobi set to spend up to $915m to acquire Dova Pharmaceuticals Sobi set to spend up to $915m to acquire Dova Pharmaceuticals

    Guido Oelkers. Sobi’s  haematology franchise  includes Elocta, a recombinant clotting factor VIII therapy for haemophilia A that offers an extended half-life, and Alprolix, a long-lasting recombinant factor IX ... Fc fusion protein for haemophilia B.

  • Pfizer goes big on gene therapy with $500m facility spend Pfizer goes big on gene therapy with $500m facility spend

    That was followed swiftly by a partnership with Spark Therapeutics that brought in a clinical-stage  haemophilia B therapy now in phase 3 testing, and another deal with Sangamo for a  ... haemophilia A candidate that recently reported encouraging phase

  • Roche’s Hemlibra scores broader NHS funding Roche’s Hemlibra scores broader NHS funding

    Expands to around 2, 000 patients in England. Hemlibra (emicizumab) will now be available for around 2, 000 people in England living with haemophilia A, a huge expansion in its market ... There are an estimated 5, 930 people registered with haemophilia

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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 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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