Shire plc (LON: SHP) has entered into a preclinical research agreement with NanoMedSyn to evaluate a potential enzyme replacement therapy (ERT) using NanoMedSyn's proprietary synthetic derivatives named AMFA, the global biotechnology announced on Monday.
The agreement will enable Shire to further expand its commitment to evaluating potential advancements in lysosomal storage disorder treatments.
Lysosomal storage disorders are inherited metabolic disorders that are characterised by an abnormal build-up of numerous toxic materials in the body's cells due to enzyme deficiencies. There are over 50 lysosomal storage disorders, affecting different parts of the body, including the central nervous system.
The AMFA compound is designed to target a specific membrane lectin, the mannose 6-phosphate (M6P) receptor, a major intracellular lysosomal trafficking pathway. Preclinical data has demonstrated that AMFA has a high affinity for binding to the M6P receptor, while preclinical models have shown that the AMFA compound increases lysosomal exposure and enhances activity of enzyme replacement therapy when compared to currently available ERT.
The terms of the agreement state that the parties will perform preclinical evaluations of AMFA conjugated to recombinant enzyme. Shire will provide NanoMedSyn with funding. Further terms of the agreement were not disclosed.
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