biostudies-literatureSamelson-Jones BJNHLBI NIH HHS5179-5189https://www.ebi.ac.uk/biostudies/studies/S-EPMC11530397biostudies-literatureUnknown8(19)<h4>Abstract</h4>After successful efforts in adeno-associated virus (AAV) gene addition for hemophilia B gene therapy, the development of valoctocogene roxaparvovec (Roctavian; Biomarin) over the past decade represents a potential new hemophilia A (HA) treatment paradigm. Roctavian is the first licensed HA gene therapy that was conditionally approved in Europe in August 2022 and approved in the United States in June 2023. Beyond Roctavian, there are ongoing pivotal trials of additional AAV vectors for HA, others that are progressing through preclinical development or early-phase clinical trial, as well as non-AAV approaches in clinical development. This review focuses on the clinical development of Roctavian for which the collective clinical trials represent the largest body of work thus fBlood advancesRoctavian gene therapy for hemophilia A.PMC11530397K08 HL146991P01 HL139420Small JCGeorge LASamelson-Jones BJfalseRoctavian gene therapy for hemophilia A.<h4>Abstract</h4>After successful efforts in adeno-associated virus (AAV) gene addition for hemophilia B gene therapy, the development of valoctocogene roxaparvovec (Roctavian; Biomarin) over the past decade represents a potential new hemophilia A (HA) treatment paradigm. Roctavian is the first licensed HA gene therapy that was conditionally approved in Europe in August 2022 and approved in the United States in June 2023. Beyond Roctavian, there are ongoing pivotal trials of additional AAV vectors for HA, others that are progressing through preclinical development or early-phase clinical trial, as well as non-AAV approaches in clinical development. This review focuses on the clinical development of Roctavian for which the collective clinical trials represent the largest body of work thus f2024-01-01T00:00:00Z2024 Oct2026-06-02T18:37:48.27Z2025-04-04T02:45:38.059ZS-EPMC115303973899111810.1182/bloodadvances.2023011847