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Wilson disease

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

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

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

It also took a 15% stake in  Vivet Therapeutics and its lead programme for Wilson disease, a devastating rare, chronic and potentially life-threatening liver disorder.

Latest news

  • Pfizer buys dwarfism drug company Therachon for $810m Pfizer buys dwarfism drug company Therachon for $810m

    Pfizer has been steadily adding to its rare disease assets via a series of takeovers and alliances to complement its own R&D in this area. ... TA-46 will add to other pipeline candidates including Sangamo-partnered haemophilia A gene therapy SB-525,

  • Pfizer hedges bets on gene therapy deal Pfizer hedges bets on gene therapy deal

    Vivet Therapeutics’ lead candidate is VTX-801, a gene therapy for Wilson disease, a devastating rare, chronic, and potentially life-threatening liver disorder. ... Pfizer will work closely with Vivet to help it develop its expertise in liver-directed

  • Alexion adds to pipeline with $1.2bn Syntimmune buy Alexion adds to pipeline with $1.2bn Syntimmune buy

    Alexion has agreed a $1.2bn takeover of US biotech Syntimmune that will add another clinical-stage rare disease drug to its R&D pipeline. ... $855m, adding orally-active copper binder WTX-101 in phase 3 testing for genetic disorder Wilson’s disease.

  • Alexion adds to pipeline rebuild with $855m Wilson buy Alexion adds to pipeline rebuild with $855m Wilson buy

    Also acquires the Swedish group’s lead product for Wilson’s disease. Alexion Pharma’s drive to reduce its reliance on cash cow Soliris has gone up a gear with a ... Sweden-based Wilson’s lead product is WTX101, currently in phase III development

  • mRNA - message received mRNA - message received

    Shire. Eukarys. monogenic liver diseases; Wilson disease. Moderna Therapeutics. cancer; cardiovascular disease; infectious disease; metabolism. ... Moreover, broadly-applicable and generic RNA manufacturing techniques are compatible with the rapid

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