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

Long, BR; Veron, P; Kuranda, K; Hardet, R; Mitchell, N; Hayes, GM; Wong, WY; Lau, K; Li, MJ; Hock, MB; Zoog, SJ; Vettermann, C; Mingozzi, F; Schweighardt, B

Long, BR (corresponding author), BioMarin Pharmaceut Inc, 105 Digital Dr, Novato, CA 94949 USA.

MOLECULAR THERAPY, 2021; 29 (2): 597

Abstract

Evaluation of immune responses to adeno-associated virus (AAV)-mediated gene therapies prior to and following dose administration plays a key role in ......

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