The consortium aims to develop and produce an Anticalin, a member of a novel high-affinity scaffold derived from the lipocalin protein family. The Anticalin is specific for hepcidin which is a central regulator of iron homeostasis, and will be used to antagonize hepcidin for the treatment of “anemia of chronic disease” (ACD). Anticalins are genetically modified lipocalins that can target almost any desired molecule. Unlike Immunoglobulins, they can be produced at low cost in microbial expression systems, are expected to be non immunogenic and offer therapeutic advantages where antibody effector functions are not desired. ACD, the most frequent anemia in hospitalized patients, develops in subjects suffering from infections, inflammatory and auto-immune disease, cancer and chronic kidney disease. It is often successfully treated by administering Erythropoiesis-Stimulating Agents. However, a significant number of patients are hypo- or non-responsive to ESA. Anti-hepcidin therapies, alone or together with ESAs, may improve anemia and the patients’ erythropoietic response and enable the use of no or even much lower ESA doses, avoiding the potential detrimental effects of high doses of ESA. The Consortium has already generated proof-of-concept data in an animal model with early candidates. The project aims at identifying, validating, and developing a specific, high affinity drug candidate based on the lipocalin scaffold as promising alternatives to immunoglobulins and a therapeutic approach based on the neutralization of hepcidin. Animal models will be developed and utilized to characterize pharmacokinetic and pharmacodynamic relationships, optimize dosing, to determine safety, biomarker responses and potential synergy with ESA’s. Furthermore, production processes will be optimized leading to a scalable GMP process which provides material for preclinical and clinical studies to establish the safety, tolerability, and PK/PD of an Anticalin hepcidin blocker (Phase Ia/b).
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Meccanismo di finanziamentoCP-FP - Small or medium-scale focused research project