Late-stage development and upstream process characterization of UX701, an AAV gene therapy for Wilson Disease

Authors

• Jan Panteli, Ultragenyx Pharmaceutical Inc., USAFollow
• Jun Li, Ultragenyx Pharmaceutical Inc.
• Patrick Hossler, Ultragenyx Pharmaceutical Inc.
• James Warren, Ultragenyx Pharmaceutical Inc.
• Benjamin Hanson, Ultragenyx Pharmaceutical Inc.
• Tiego Bezerra, Ultragenyx Pharmaceutical Inc.

Conference Dates

April 27 - May 2, 2025

Abstract

Please click Additional Files below to see the full abstract

Recommended Citation

Jan Panteli, Jun Li, Patrick Hossler, James Warren, Benjamin Hanson, and Tiego Bezerra, "Late-stage development and upstream process characterization of UX701, an AAV gene therapy for Wilson Disease" in "Cell Culture Engineering XIX", A Khetan, BMS; Marcella Yu, Gilead; Mike Betenbaugh, Johns Hopkins University Eds, ECI Symposium Series, (2025). https://dc.engconfintl.org/cell_culture_engineering_xix/191