Susana Navarro

Since her PhD Susana Navarro has been working on the development and improvement of Gene Therapy for rare diseases focused in anemias and bone marrow failure syndromes such as porphyrias, Fanconi Anemia (FA) and Pyruvate kinase Deficiency (PKD). In 2003 she joined the Division and Innovative Therapies of the Hematopoietic System at CIEMAT/CIBERER/IIS-FJD and she´s been involved in the study of animal models for both FA and PKD and specially in the study of human bone marrow samples from patients with both of these diseases and the development of transduction protocols to restore their clinical manisfestations. Additionally, she has participated in the generation of 2 lentiviral vectors that carry the FANCA and PKLR gene respectively, and in particular, in efficacy and safety studies of the vectors generated (stay in the hematology laboratory of the Hannover Medical School, directed by Dr. Christopher Baum, 2007). Due to this she has participated in 4 Orphan Drug Designations (ODD) both in Europe and the USA for therapeutic FANCA ( EMA/COMP/662962/2010 and #DRU-2016-5193) and PKLR ( EMA/COMP/865665/2014 and DRU-2016-5168) lentiviral gene therapy vectors and the subsequent presentation of the IMPD according to EMA guidelines for the subsequent development of a Phase I/II gene therapy trials on these diseases. All this has allowed her to participate as an associate researcher in the clinical trial for the treatment of patients with AF through cells corrected with this therapeutic lentiviral vector carrying the FA gene and in phase II of this study that is carried out under the Sponsor of the company Rocket Pharmaceuticals as well as she´s also participating in the manufacturing of the medicinal product as Supervisor of the Clinistem GMP facility at the CIEMAT. Likewise, she is also currently participating as a researcher in the first Phase I and global clinical trial for the treatment of this disease "A Phase I Clinical Trial to Evaluate the Safety of the Infusion of Autologous CD34 + Cells Transduced With a Lentiviral Vector Carrying the Codon Optimized Red Cell Pyruvate Kinase (coRPK) Gene in Adult and Pediatric Subjects With PKD ".Aditionally during some year she intended to new sources of hematopoietic cells, working in the generation of iPSCs and in the differentiation of these to hematopoietic progenitors, as well as in the development of new gene therapy strategies working in the field of gene editing for Fanconi cells. Due to this expertise she´s currently coordinating a research national project from CIBERER and co-cordinating a European Project from EJPRD in gene therapy for Blackfan Diamond anemia with a permanent position as “Investigador Científico de OPIS at CIEMAT”.