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Hunter syndrome
This page shows the latest Hunter syndrome news and features for those working in and with pharma, biotech and healthcare.
Takeda highlights 12 NMEs with $10bn-plus sales potential at R&D day
It’s scheduled for regulatory approval in 2020. TAK-609 for Hunter syndrome:TAK-609 (formerly SHP609) is a new intrathecal formulation of Takeda’s existing intravenous Hunter syndrome therapy Elaprase ... including Dravet syndrome, and is tipped
Latest news
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Sangamo surges on data for haemophilia A gene therapy
nuclease (ZFN) gene-editing platform for rare disease Hunter syndrome.
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Trial dents confidence in Sangamo’s gene-editing tech
Hunter syndrome was able to induce expression of the IDS enzyme that is deficient in the disease. ... was able to induce expression of the IDUA enzyme missing in the disease, which is also known as Hurler syndrome.
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Sangamo’s first gene-editing trial doesn’t convince investors
Market spooked by inconclusive early results. Sangamo Therapeutics has claimed a first with a trial of its genome-editing technology for rare disease Hunter syndrome, but the data didn’t convince ... gene-editing technology that is designed to modify
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Shire separates out neuroscience as it considers its future
Hunter syndrome therapy.
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Shire suffers pipeline failure as takeover speculation builds
Several big pharma companies are reportedly considering bids. Shire Pharmaceuticals has suffered a setback with the failure of its therapy for Hunter syndrome to meet its objectives in a pivotal trial, ... The rare disease specialist revealed yesterday
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Latest Intelligence
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2019: CRISPR and therapeutic gene editing comes of age
The phase 1/2 CHAMPIONS study did show that a single injection of SB-913 for the rare genetic disease mucopolysaccharidosis type II (MPS II) or Hunter syndrome was able to
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Deal Watch table for July 2014
250. ArmaGen/ Shire . Licence, collaboration . AGT-182, enzyme replacement therapy for Hunter syndrome (preclinical).
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Latest from PMHub
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Onyx Health Joins Forces with Rare Disease Specialists Actigen
The current focus of Actigen's work is on the development of a drug for a potential breakthrough treatment for the life-limiting rare disease known as MPS II or Hunter ... Syndrome. MPS II is a serious, rare disease that has a major impact on the
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TVF Communications shortlisted for two awards at PMEA 2016!
For both target diseases, Hunter Syndrome(MPS II) and Hidradenitis Suppurativa (HS), a timely diagnosis is imperative, as the early symptoms are often mistaken for far more common and far less ... refer to a geneticist if the syndrome is suspected.
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