Shire has linked with US biotech NanoMedSyn to evaluate a potential enzyme replacement therapy (ERT) for an undisclosed lysosomal storage disorder.
Lysosomal storage disorders are inherited metabolic disorders characterised by an abnormal build-up of various toxic materials in the body's cells as a result of enzyme deficiencies.
The parties have signed a preclinical research collaboration that will evaluate a potential ERT using NanoMedSyn’s proprietary synthetic derivatives named AMFA.
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.
According to the firms, preclinical data show that AMFA has a high affinity for binding to this receptor, and in preclinical models the AMFA compound lead to increased lysosomal exposure and enhanced activity of ERT compared to a current available ERT.
“The novel design of AMFA and the promising biological activity demonstrated in preclinical models makes this program an exciting opportunity for Shire to further expand its commitment to evaluating potential advancements in lysosomal storage disorder treatments,” said Shire’s chief executive and head of R&D, Andreas Busch.
Henry-Vincent Charbonné, NanoMedSyn’s chief executive, said the deal provides the opportunity to further evaluate molecules based on its AMFA technology, “which may potentially benefit patients with lysosomal storage disorders that are currently treated with the traditional enzyme replacement therapies”.
Financial terms of the deal weren’t disclosed, apart from that Shire will provide funding to NanoMedsyn.