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Carmen Unzu. Programa Terapia Génica y Regulación de la Expresión Génica. Cima Universidad de Navarra

Dr. Carmen Unzu

Investigator, Gene Therapy for Rare Diseases Program. Interest in development of AAV-mediated gene therapy for inborn rare diseases

Be part of: Cima Universidad de Navarra

INDEX H 11

Professional career

MS in Biochemistry at the University of Navarra (2008). PhD in Biochemistry at the University of Navarra with focus on the development of non-integrative viral vectors for in vivo liver-directed gene therapy (2012).
Postdoctoral fellow at the University of Geneva (UNIGE) and EPFL (Switzerland) 2013-2017.
Senior Scientist at Harvard Medical School/Mass Eye and Ear (Boston, USA) (2017-2019)
Associate Director of Preclinical Research at Apic Bio (Cambridge, USA) (2019-2021)

RESEARCH AREAS

Preclinical development of AAV-mediated gene therapy in academia and industry settings.
One of them, rAAV5.PBGD, an investigational drug for the treatment of Acute Intermittent Porphyria, was tested in a Phase I/II clinical trial (NCT02082860)

Development of a novel method to generate hepatic progenitors from adult hepatocytes for cell therapy.

Co-inventor in 4 patents.

Participation in more than 15 competitive research project funded by both public and private funds such as the Spanish Ministry of Science and Innovation, Carlos III Health Institute, the European Union, the Louis-Jeantet Foundation, Novartis Health Foundation or Pfizer Inc.