Morgane Wartel is a Scientist in the Vector Technology Department at uniQure (Amsterdam, The Netherlands). She leads the Cargo team, consisting of Scientists, Technicians and Students. Her team focuses on the adeno-associated virus (AAV) expression cassette — or Cargo, used in the AAV-based gene therapy pipeline developed at uniQure. The Cargo team, closely working with the Disease Biology teams, is currently developing technologies to improve cell specificity, cell targeting as well as transgene expression levels. Her team is also responsible for the iPSC platform, where suitable in vitro models are developed to better assess on- and off-target effects of AAV lead candidates, and their potency and safety.
Prior to joining uniQure, Morgane was a Postdoctoral Researcher at the European Molecular Biology Laboratory (EMBL), Heidelberg, Germany, where she developed a platform to systematically monitor the activation of bacterial signaling pathways. Morgane holds a PhD in Biology from Aix-Marseille University, Marseille, France.
Morgane is the leader of Work Package 4 (SPREAD). Within GUTVIBRATIONS, Morgane will be supervising the Postdoctoral Researcher Renata Vieira de Sá.
uniQure is a global leader in gene therapy and is developing single treatments with potentially curative results. uniQure is using adeno-associated virus (AAV)-based gene therapy to rapidly advance a pipeline of proprietary and partnered gene therapies to treat patients with hemophilia, Huntington’s disease, and other severe genetic diseases.
uniQure is currently conducting a pivotal phase 3 trial in their lead indication, hemophilia B, where the targeted number of patients has received the one-time dose of the AAV-based gene therapy and has been closely monitored for already a year. uniQure has also initiated a phase 1/2 trial in Huntington’s disease, with patient enrollment completed for the first-dose cohort.