Kedrion Biopharma has developed Project PODs (Plasma Orphan Drugs) in collaboration with the Institute of Clinical Physiology of the CNR (National Research Council) in Pisa and with the support of the Region of Tuscany. On the 14th and 15th of July Project PODs took the center stage at SMAU Florence – Toscana Technologica, an event dedicated to highlighting excellence in the fields of technology and innovation in Tuscany.
For the past ten years, Kedrion has invested in research on plasma-derived orphan drugs to treat rare diseases because of the vocation and skills that are at the heart of the Company's activities. Project PODs was born for the purification, characterization and development of four plasma-derived orphan drugs, plasminogen, factor V, factor H and factor XI. All are to be used in the treatment of particularly rare diseases caused by a deficiency of plasma proteins.
"Deciding to invest in the field of orphan drugs was a strategic and ethical choice for Kedrion,” stated Paolo Marcucci, Chairman and CEO of Kedrion. "The invitation we received to take part in the event is further proof of the value of the R&D we carry out in our Company. In Kedrion, we have always been strongly committed to finding new products and to discovering new clinical applications of existing products."
The heads of the POR CreO FESR regional observatory deemed PODs a project of excellence for its level of innovation.
The project kicked off in June 2011 and wrapped up in August 2014. Studies on human plasminogen concentrate have already passed the stage of clinical trials, while the other products are in an earlier stage of research. In the case of factor V, the development of the method and the characterization of the finished product is concluded. In addition, the first study of acute toxicity has been carried out. In the case of factor H, method development and characterization of the finished product have been completed; efficacy studies on animal models and pharmacokinetic studies were carried out. Finally, as pertains to factor XI, the development of the purification method has been determined, as has its initial characterization.
As part of the Project PODs, Kedrion set up a small pilot plant in Siena, renamed IKOD (Plant Kedrion Orphan Drugs) aimed at the experimental production of these drugs.
"The project PODs is an example of the Italian excellence that it offered to the world. We talk about rare diseases, but there are many patients involved and we are committed to extending access to the plasma-derived medicinal products they vitally need as much as possible,” said Claudia Nardini, Kedrion R & D Senior Specialist.
"Partnerships such as this one enhance the quality and excellence of our research,” stated Piero Salvadori, scientific coordinator of the CNR Research Team. “IFC-CNR has been Kedrion’s long-standing partner as we both place the patients at the center of our research mission. In the PODs Project, we used enabling technologies in the biomedical and pharmaceutical fields. We have also applied the most advanced protein separation, proteomics and molecular micro-imaging techniques with the aim of accelerating to the maximum the complex path of pharmaceutical development for the studied products."
"The partnership with research institutions such as the CNR, with whom we have carried out a fruitful collaboration for many years, is of strategic importance in the company’s product and process innovation path", concluded Dr Nardini.