News

Antibody test for Roctavian cleared under new, stringent EU rules

AAV5 DetectCDx, a companion diagnostic test used to determine patient eligibility for the hemophilia A gene therapy Roctavian (valoctocogene roxaparvovec-rvox), has been cleared for use under more stringent European Union rules coming into effect in a few years. Specifically, the test gained the Conformité Européenne (CE) mark under…

Genevant, Novo Nordisk partner on gene-editing treatment for hem A

Genevant Sciences is teaming up with Novo Nordisk to develop a gene-editing treatment for hemophilia A that combines Genevant’s proprietary lipid nanoparticle (LNP) platform with cutting-edge mRNA-based megaTAL technology. The partners will seek to advance a treatment involving gene editing — used to correct, add, or delete…

Prix Galien USA award goes to CSL and uniQure for Hemgenix

Hemgenix (etranacogene dezaparvovec), the first and only gene therapy approved for adults with hemophilia B, has won this year’s Prix Galien USA award in the category of Best Product for Rare/Orphan Diseases. The award — the nation’s top prize for leading-edge advances in life sciences — was received…

Hope Conference set to educate Nov. 2-4 in person and online

This year’s Hope Conference, a national educational event presented by Hope Charities for the bleeding disorders community, is set for Nov. 2-4 in Florida, with an online option as well. The annual conference, to be presented at the Baytowne Conference Center, in Miramar Beach, is designed to bring together…