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2019 AWARDS WINNERS

Outstanding achievement award

Nathalie Cartier

After her medical residency, Nathalie Cartier held a postdoctoral position at Institut Cochin in the laboratory of Axel Kahn, in which she developed transgenic animal model for cancer research. In 1993, she joined the laboratory of Pierre Bougnères to work on the development of the first therapeutic clinical trial of gene therapy in human. This study enabled the cure of four children with adrenoleukodystrophy, a so far deadly and uncured disease. She pursued this therapeutic approach by developing the gene therapy of the Metachromatic leukodystrophy. A clinical trial is currently ongoing at the Bicêtre Hospital in Paris.

Today, she investigates a gene therapy approach targeting the cerebral cholesterol pathway to cure Alzheimer and Huntington disease at the CEA Centre d’imagerie Moléculaire (MirCen).

Nathalie Cartier has been president of the French and of the European Society of Gene and Cell Therapy (SFTCG and ESGCT) where she has been involved in the development of new approach of gene therapy. She has received the Legion of Honor, the awards of the Medical Academy, the Prix Jean Valade of the French foundation and the Prix Thermo Biotherapy.


Supported by:


Young Investigator Award

Paula Rio

has been working in the field of Fanconi anemia (FA) gene therapy for 20 years, in the design of new vectors for the treatment of the disease (González-Murillo et al, 2010), in the characterization of different preclinical models of the disease (Rio et al., 2002; Rio et al., 2008) and in the translation of gene therapy to the clinic (Jacome et al. 2006; Jacome et al., 2009; Tolar et al., 2012). Her studies in the field of gene therapy in Fanconi anemia started with her Thesis entitled” Gene therapy for Fanconi anemia subtype A in a mouse model” directed by Prof. J.A. Bueren and Dr. J.C. Segovia with a distinction “cum laude”. She got a post-doctoral position in Prof. Hanenberg’ lab (Children’s Hospital, Düsseldorf), where she was involved in the characterization of a new FA gene (Levran et al., 2005). In 2005 she moved to CIEMAT where she started working in the development of a new protocol to correct hematopoietic stem cells (HSCs) from FA patients by gene therapy (Jacome et al., 2006; Jacome et al.,2009) and the design of a new lentiviral vector that has received the orphan drug designation both by the EMA and FDA (Ref: EMA/COMP/662962/2010 and #DRU-2016-5193, respectively). In 2017 she participated in a study demonstrating for the first time that a short transduction protocol allows the engraftment of corrected cells from FA patients in immunodeficient mice (Rio*, Navarro* et al., 2017). Now she is deeply involved as Associated investigator in two different clinical trials focused on the correction of HSCs from Fanconi anemia A patients (NCT03157804 and NCT04248439) (Río et al. 2019). She is also in charge of Production department of the GMP facility Clinistem for the development of gene therapy trials for rare diseases.

Since 2010 she has been also working in gene editing, first by targeting HSCs from FA patients by Homologous recombination (Rio*, Baños* et al., 2014; Diez et al., 2017) and nowadays by Non-homologous end joining using the CRISPR/Cas9 system (Román-Rodriguez et al., 2019).

She has published 42 articles (PubMed; H index: 18), received 6 International Awards including the Young Investigator Award by the European Society of Gene and Cell Therapy (ESGCT) in 2019.

She has directed 5 Doctoral Thesis, and nowadays is supervising two PhD students.

Paula has been for four years a Board Member of the Spanish Society of Cell and Gene Therapy. She also evaluates papers from different specialized journals and participates actively in the Fanconi anemia Gene Therapy working group, organized by the Fanconi Anemia Research Fund.

Nowadays she is co-principal investigator in two different projects, one focused on Gene editing in FA, granted by Rocket Pharma and a project supported by the Spanish Ministry of Science, Innovation and Universities entitled: Gene therapy and other innovative therapies for the treatment of inherited bone marrow failure syndromes (GENECURE).

Travel award winners





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