German-based biotechnology company CSL Behring has received conditional marketing authorization (CMA) from the European Commission (EC) for its Hemgenix (etranacogene dezaparvovec) indicated for the treatment of hemophilia B in adults. Hemgenix (also known as CSL222, previously known as AMT-061) is an adeno-associated virus five (AAV5)-based gene therapy intended to be given as a one-time treatment […]
CSL Behring, a US-based biotechnology company, has received approval for its Hemgenix (etranacogene dezaparvovec-drlb) from the US Food and Drug Administration (FDA) as a one-time gene therapy for hemophilia B in adults. Hemophilia B is a bleeding disorder resulting from a single gene defect, which causes inadequate production of factor IX. Produced mainly by the […]
Centessa Pharmaceuticals has secured orphan drug designation from the US Food and Drug Administration (FDA) for SerpinPC in hemophilia B treatment. According to the UK-based clinical-stage pharma company, SerpinPC is an inhibitor of activated protein C (APC). Centessa Pharmaceuticals is looking at Q4 2022 to kick-start registrational studies of SerpinPC. Based on the serpin family […]