Shire, the global biotechnology leader in rare diseases, and NanoMedSyn, a biotechnology company dedicated to innovation in enzyme replacement therapy (ERT), announced that the companies have entered into a preclinical research collaboration to evaluate a potential ERT using NanoMedSyn’s proprietary synthetic derivatives named AMFA.
Lysosomal storage disorders are inherited metabolic disorders that are characterized by an abnormal build-up of various toxic materials in the body’s cells as a result of enzyme deficiencies. There are more than 50 of these disorders altogether, and they may affect different parts of the body, including the central nervous system.
The AMFA compound is designed for the targeting of a specific membrane lectin, the mannose 6-phosphate (M6P) receptor, a major intracellular lysosomal trafficking pathway. Preclinical data demonstrate that AMFA has a high affinity for binding to the M6P receptor. Additionally, in preclinical models the AMFA compound leads to increased lysosomal exposure and enhanced activity of enzyme replacement therapy compared to a current available ERT.
Under the terms of the agreement, the parties will perform preclinical evaluations of AMFA conjugated to recombinant enzyme. Shire will provide funding to NanoMedsyn under the agreement.
NanoMedSyn is a private biotech company based in Montpellier (France) which was created to develop the innovative AMFA technology. NanoMedSyn holds the exclusive worldwide rights on the technology. The technological platform of AMFA compounds have the potential to target various proteins or drugs to tissues and cells expressing the mannose 6-phosphate receptors in order to facilitate their cellular entrance and eventual lysosomal uptake.