Valoctocogene Roxaparvovec: First Approval

Lack of germline transmission in male mice following a single intravenous administration of AAV5-hFVIII-SQ gene therapy

(2022) Carlos Fonck et al.   GENE THERAPY

Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A

(2022) Margareth C. Ozelo et al.   NEW ENGLAND JOURNAL OF MEDICINE

Haemophilia: factoring in new therapies

(2021) Hannah Fassel et al.   BRITISH JOURNAL OF HAEMATOLOGY

Persistence of haemostatic response following gene therapy with valoctocogene roxaparvovec in severe haemophilia A

(2021) K John Pasi et al.   HAEMOPHILIA

Molecular analysis of AAV5-hFVIII-SQ vector-genome-processing kinetics in transduced mouse and nonhuman primate livers

(2021) Choong-Ryoul Sihn et al.   Molecular Therapy-Methods & Clinical Development

Early Phase Clinical Immunogenicity of Valoctocogene Roxaparvovec, an AAV5-Mediated Gene Therapy for Hemophilia A

(2020) Brian R. Long et al.   MOLECULAR THERAPY

The Impact of Pre-existing Immunity on the Non-clinical Pharmacodynamics of AAV5-Based Gene Therapy

(2019) Brian R. Long et al.   Molecular Therapy-Methods & Clinical Development

Prednisolone Does Not Regulate Factor VIII Expression in Mice Receiving AAV5-hFVIII-SQ: Valoctocogene Roxaparvovec

(2019) Lening Zhang et al.   Molecular Therapy-Methods & Clinical Development

Gene Therapy with BMN 270 Results in Therapeutic Levels of FVIII in Mice and Primates and Normalization of Bleeding in Hemophilic Mice

(2018) Stuart Bunting et al.   MOLECULAR THERAPY

AAV5–Factor VIII Gene Transfer in Severe Hemophilia A

(2017) Savita Rangarajan et al.   NEW ENGLAND JOURNAL OF MEDICINE