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Yposkesi’s viral vectors used in gene therapy-based clinical trial


10th September 2019
Yposkesi, a leading Contract Development and Manufacturing Organization (CDMO) for preferred access and reserved capacity for cGMP grade viral vector production, today announces it produced the viral vectors used in the gene therapy-based clinical trial in patients with Fanconi anemia. In this study, the authors demonstrate for the first time the production of blood cells derived from the patients’ genetically corrected stem cells. The work on Fanconi anemia patients is published in Nature Medicine.
 
“Yposkesi is very pleased to have contributed to this major study of patients with Fanconi anemia," said Alain Lamproye, CEO of Yposkesi. “The results obtained in this study offer new opportunities in the treatment of Fanconi anemia patients, while also demonstrating the quality and efficiency of our bioproduction processes."
 
Fanconi anemia is a rare genetic disease characterized by bone marrow failure and cancer predisposition that appears in most patients with poor blood cell production at very young ages. Yposkesi was selected to contribute to the clinical trial because of its extensive expertise and track record in manufacturing of lentiviral vectors, which it developed under Genethon, a pioneer in gene therapy.
 
Yposkesi supplied the viral vectors to the EuroFancoLen program, which carried out the research. Coordinated under Dr. Juan Bueren of the Center for Energy, Environment and Technology (CIEMAT), it involved the Center for Biomedical Network Research on Rare Diseases (CIBERER) and the Institute of Health Research of the Jiménez Díaz Foundation (IIS-FJD). It was guided under the clinical direction of Dr. Julián Sevilla of the Foundation of the Hospital del Niño Jesús, in Madrid (Spain), the clinical sponsor of the trial. The first authors of the study are Dr. Paula Río and Dr. Susana Navarro, affiliates of the CIEMAT / CIBERER / IIS-FJD Consortium.