Shire teams up with NanoMedSyn for enzyme replacement therapy research

The FTSE 100 company said that under the terms of the agreement, the two parties would perform preclinical evaluations of AMFA conjugated to recombinant enzyme.

Shire said it would provide funding to NanoMedsyn under the agreement, with further terms of the agreement not disclosed.

It explained that lysosomal storage disorders were inherited metabolic disorders, characterised by an abnormal build-up of various toxic materials in the body's cells as a result of enzyme deficiencies.

There were more than 50 of the disorders altogether, and they could affect different parts of the body, including the central nervous system.

Shire said NanoMedSyn’s AMFA compound was designed for the targeting of a specific membrane lectin - the mannose 6-phosphate receptor - a major intracellular lysosomal trafficking pathway.

Preclinical data demonstrated that AMFA had a “high affinity” for binding to the M6P receptor.

Additionally, in preclinical models, the AMFA compound lead to increased lysosomal exposure and enhanced activity of enzyme replacement therapy compared to a current available therapy.

“NanoMedSyn has demonstrated innovation in advancing the next generation of enzyme replacement therapy, and Shire is pleased to enter this research agreement with NanoMedSyn,” said Shire’s head of research and development, and chief scientific officer, Andreas Busch.

“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.”

NanoMedSyn’s chief executive officer and chairman Henry-Vincent Charbonné said the agreement provided the opportunity to further evaluate molecules based on his company’s proprietary AMFA technology, which could potentially benefit patients with lysosomal storage disorders that were currently treated with traditional enzyme replacement therapies.

“As a global biotech leader in the development and commercialisation of biologic therapeutics, Shire is an ideal research partner, particularly given their extensive expertise in the area of lysosomal storage disorders.”