Shire and NanoMedSyn Enter into Research Agreement for Potential Lysosomal Storage Disorder Treatment

Rare Daily Staff

Shire said it entered into a preclinical research collaboration with NanoMedSyn to evaluate a potential enzyme replacement therapy using NanoMedSyn’s proprietary synthetic derivatives to treat lysosomal storage disorders.

Lysosomal storage disorders are genetic disorders that are characterized by an abnormal build-up of improperly broken down metabolic waste in the body’s cells as a result of enzyme deficiencies. There are more than 50 of these disorders and they may affect different parts of the body, including the central nervous system. Shire has long been working to address a number of lysosomal storage disorders including Hunter Syndrome, Type 1 Gaucher Disease, and Fabry Disease.

NanoMedSyn’s proprietary technology platform involves the development of what it calls AMFA compounds. 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.

Preclinical data demonstrates that AMFA has a high affinity for binding to the M6P receptor, the companies said. Preclinical models also showed the AMFA compound leads to increased lysosomal exposure and enhanced activity of enzyme replacement therapy compared to a current available enzyme replacement therapies.

Under the terms of the agreement, the companies will perform preclinical evaluations of AMFA conjugated to recombinant enzyme. Shire will provide funding to NanoMedsyn under the agreement. The companies did not disclose additional financial and other details.

“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 Andreas Busch, head of research and development and chief scientific officer at Shire.

March 26, 2018
Photo: Andreas Busch, head of research and development and chief scientific officer at Shire

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