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UniQure

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NICE backs GSK’s €594k gene therapy for rare disease

The treatment offers 'bubble baby' patients an alternative to a stem cell transplant

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Spark sets Luxturna price, igniting US affordability debate

The IRD treatment is set to cost patients $425,000 per eye

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Spark’s Luxturna becomes US’s first approved gene therapy

FDA says its move 'reinforces the potential of this breakthrough'

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Gene therapy

When will it deliver?

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Shire’s gene therapy candidate receives orphan drug status in the US

Investigational factor VIII treatment SHP654 is being developed for haemophilia

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FDA grants orphan drug status to UniQure’s Huntington’s Disease treatment

Becomes first investigational gene therapy to receive ODD for the disease

Will gene therapies finally be commercially viable to pharma?

Our latest Free Thinking white paper explores the science behind the idea of gene therapy and takes a look at some of the core challenges medical researchers have faced when...

Research Partnership

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Spark closes on first US gene therapy approval

FDA starts review of Luxturna for patients with inherited retinal disease

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Novo Nordisk’s long-acting haemophilia B drug cleared by FDA

US approval sets up marketing battle with Bioverativ and CSL Behring

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UniQure pulls first EU gene therapy

Decision made due to lack of demand

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uniQure appoints chief executive officer

Matthew Kapusta takes up the lead role in a permanent capacity

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Opening up innovation

Paul-Peter Tak on GlaxoSmithKline’s R&D strategy and the pivotal role of immunology

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