NanoMedSyn is working on Lysosomal Storage Disorders. This biotechnology company is developing new targeted therapeutics to treat Lysosomal Storage Disorders. Thanks to the company’s proprietary synthetic derivatives (named AMFA), NanoMedSyn provides a compound with an high affinity for the mannose 6-phosphate receptor. This receptor is key for lysosomal targeting and thus for the treatment of Lysosomal Storage Disorders.
In September 2016, NanoMedSyn was granted by E.M.A. the orphan drug designation for its first compound, recombinant acid alpha-glucosidase conjugated with AMFA, aimed for the treatment of Pompe disease.
Lysosomal Storage Disorders, of which more than 50 are known, are caused by the absence or function impairment of key lysosomal enzymes leading to the intra-lysosomal accumulation of undegrated metabolites. Each Lysosomal Storage Disorder has a distinct clinical picture, depending on the type of accumulating substrate and the tissue in which it accumulates.
The AMFA compound is designed for the targeting of a specific membrane lectin, the mannose 6-phosphate receptor, the major addressing pathway to lysosomes. These 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. NanoMedSyn applies the AMFA technology to Enzyme Replacement Therapy (E.R.T.) of orphan Lysosomal Storage Disorders and to cancers overexpressing the mannose 6-phosphate receptor.
Regarding ERT, NanoMedSyn‘s first ambition is not to market drugs directly but to become a designer and a provider of a new generation of enzymes to treat Lysosomal Storage Disorders.
The preclinical proof of concept studies indicate the higher efficacy of the enzyme-AMFA constructs as compared to classical recombinant enzyme productions and lead the company to develop a first candidate drug up to the clinical phase I/II stage, and to look at the creation of new AMFA-Enzymes for untreated diseases among Lysosomal Storage Disorders.
Registered in 2012, NanoMedSyn is a private venture-capital funded company based in Montpellier.