BioPharm
Gene therapy transforms lives for people with haemophilia A

“A normal life.” That, in the words of a clinical trial patient, is what a world-first gene therapy for severe haemophilia A can offer. ROCTAVIAN received marketing approval in Europe in 2022 and US FDA approval in 2023 and is now available to patients commercially. ROCTAVIAN (valoctocogene roxaparvovec) is a ‘one and done’ treatment approach: patients who previously needed regular protein injections to manage the bleeding disorder are experiencing life-changing benefits.
Learn more about the impact of this gene therapy. Hear from Professor Amit Nathwani, Richard Fagan, and a patient who has benefitted from the treatment.
Discover more, listen to Professor Amit Nathwani's interview in Episode 13 of the UCLB Big Talks on Big Impacts podcast series.