A novel, improved biotech molecule has the potential to revolutionize the treatment of hemophilia B: it has been designed to enhance the efficacy of coagulation and decrease the frequency of infusions. The findings, published in Nature Communications, are the result of an international study led by the University of Ferrara, in collaboration with colleagues from the University of Oslo and San Raffaele Hospital in Milan. Professor Alessio Branchini of the Department of Life Sciences and Biotechnology at the University of Ferrara explains, "We have all observed how blood coagulation works when a small wound heals. Regrettably, there are instances of inherited genetic diseases, such as hemophilia B, in which one of the factors that are essential for coagulation, specifically factor IX, is absent. In these patients, replacement therapy is a frequently employed therapeutic approach, which involves the infusion of a "recombinant" factor IX, which is produced in a laboratory. However, the therapy's efficacy is limited over time, necessitating repeated infusions". Now, thanks to genetic and protein engineering, researchers have modified the "recombinant" factor IX molecule to create a super-molecule with improved properties. "To improve it, we worked on several fronts using a biotechnological approach, obtaining two new highly effective molecules. The modifications we made allowed us to make these molecules more ‘personalized’ — aiming for maximum short-term efficacy, as in cases of acute bleeding, or for long-term use typical of prophylaxis, where lasting effectiveness is of utmost importance", emphasizes Branchini.