The FDA has approved Krystal Biotech’s gene therapy Vyjuvek for dystrophic epidermolysis bullosa (DEB), a rare genetic disease characterised by fragile skin that can split
In the latest instalment of the pharmaphorum podcast, editor-in-chief Jonah Comstock connects with Kylie O’Keefe to talk about gene therapy in rare diseases.
An FDA advisory committee has given tentative backing to Sarepta’s gene therapy for Duchenne muscular dystrophy (DMD), even though the agency’s reviewers said they were no
UK start-up Complement Therapeutics has raised an impressive €72 million ($79 million) in first-round financing that will be used to take a gene therapy for sight-robbing
The influential Institute for Clinical and Economic Review (ICER) in the US has issued draft guidance suggesting that new gene therapies for sickle cell disease (SCD) from
A pharma company’s core mission is to improve patient outcomes. This hinges on effectively influencing HCP clinical behaviour and driving disease education.