Applications

One Platform Unlocking Multiple Therapeutic Applications

Our platform is versatile and can be applied to multiple therapeutic areas. AMFA is stable in vivo, insensitive to phosphatases (unlike natural M6P), and can be conjugated to antibodies, enzymes, nanoparticles, or biologics—providing a unique solution to unmet clinical needs.

Our team can perform AMFA-grafting, as well as in vitro and in vivo characterization of AMFA-engineered therapeutic candidates.

AMFA Applications illustration

First‑in‑class therapeutic antibodies

Therapeutic AMFA-engineered antibodies acquire a higher cellular uptake and a unique capacity to deplete specific soluble and membrane antigens through lysosomal degradation. They retain all desirable features of standard antibodies (recycling, immunogenicity, ADCC, etc.) and remain compatible with standard manufacturing processes.

This new class of antibodies is relevant for the treatment of autoimmune, inflammatory, and oncology indications.

Enzyme replacement therapy for lysosomal storage diseases

NanoMedSyn has developed acid alpha‑glucosidase conjugated with AMFA, intended for the treatment of Pompe disease (1, 2 & 3).

This AMFA‑engineered enzyme received Orphan Drug Designation from the EMA for enzyme replacement therapy in Pompe patients (4).

The benefits of AMFA-mediated lysosomal delivery suggest broader application to other lysosomal storage disorders and rare diseases (3).

Therapeutic targeting of nanoparticles

AMFA-engineered nanoparticles show increased cellular uptake and enhanced therapeutic efficacy in cancer models that overexpress M6PR, including prostate cancer and rhabdomyosarcoma (5, 6).