Type of information: Research agreement
- enzyme replacement therapy using AMFA (recombinant human acid alpha-glucosidase conjugated with mannose-6-phosphate analogues)
Company: Nanomedsyn (France) Shire (UK - USA)
Therapeutic area: Rare diseases - Genetic diseases
Type agreement: R&D - research
- enzyme replacement therapy. Nanomedsyn has developed a new enzyme replacement therapy coupled with patented synthetic derivatives called AMFA. These derivatives have been designed for the targeting of a specific membrane lectin, the mannose 6-phosphate (M6P) receptor, the major addressing pathway to lysosomes. The AMFA compounds have the potential to target various proteins or drugs to tissues and cells expressing these receptors in order to facilitate their cellular entrance and finally lysosomal uptake.
- 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 enzyme replacement therapy.
- NanoMedSyn holds the exclusive worldwide rights on the technology. In September 2016, the European Medicines Agency granted orphan drug designation for NanoMedSyn’s first compound, recombinant acid alpha-glucosidase conjugated with AMFA for the potential treatment of Pompe disease, a lysosomal storage disorder.
Disease: undisclosed lysosomal disease
- • On March 26, 2018, Shire, a global biotechnology leader in rare diseases, and NanoMedSyn, a biotechnology company dedicated to innovation in enzyme replacement therapy (ERT), have entered into a preclinical research collaboration to evaluate a potential ERT using NanoMedSyn’s proprietary synthetic derivatives named AMFA. 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. Further terms of the agreement were not disclosed.