Latest news

• AZ’s new rare disease unit wins in Wilson disease

  Alexion purchase looks increasingly ‘copper-bottomed’ as AstraZeneca prepares to submit data for ALXN1840 in Wilson disease. ... Wilson disease is a rare, progressive genetic condition in which the body’s pathway for removing excess copper is

• 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.

• 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

  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 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.