News uniQure poleaxed as FDA blocks Huntington's therapy uniQure has revealed that the FDA has said it is not prepared to review its Huntington's gene therapy in what seems to be a change in policy.
News Rocket aborts FDA filing for Fanconi gene therapy Rocket Pharma has withdrawn a filing in the US for its experimental gene therapy for Fanconi anaemia, but may consider partnering the programme.
News Joy as uniQure Huntington's therapy clears pivotal trial uniQure's gene therapy AMT-130 has cleared a trial in Huntington's, setting up filings and raising the prospect of a first treatment for the disease.
News FDA restricts use of bluebird's Skysona gene therapy The FDA has added a warning label to bluebird bio's Skysona, after launching a probe into cases of blood cancer linked to the gene therapy.
News FDA gives the go-ahead to restart Elevidys shipments Sarepta has restarted distribution of its Duchenne muscular dystrophy gene therapy Elevidys, but only for patients who can walk.
News Sangamo cues up Fabry gene therapy filing on STAAR data Sangamo plans to file for approval of its gene therapy for Fabry disease next year, but a launch could depend on finding a commercial partner.
News MFN model for Medicaid revealed by CMS The Trump administration has released details of a most-favoured nation (MFN) drug pricing programme for medicines provided by Medicaid.
Sales & Marketing Sponsored How to meet your physicians where they are online A pharma company’s core mission is to improve patient outcomes. This hinges on effectively influencing HCP clinical behaviour and driving disease education.