Objectives and technology

NanoMedSyn develops a new Enzyme Replacement Therapy. Its development plan is based on a strong intellectual property of patented synthetic derivatives called AMFA, 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.

The main application is the production of new modified recombinant enzymes for the treatment by Enzyme Replacement Therapy of orphan lysosomal storage disorders. Thanks to the results obtained in the proof of concept and the preclinical phases conducted on two different Lysosomal Storage Disorders, NanoMedSyn has decided to develop the lead compound enzyme-AMFA in the Pompe disease up to the clinical phases 1/2.

Pompe disease is also known as glycogen storage disease type II, in which patients suffer from a deficiency of the lysosomal enzyme acid alpha-glucosidase. The lysosomal accumulation of the substrate of the deficient enzyme leads to a progressive and deleterious weakness of muscles. There are different degrees of severity of this disease and also two main ages of onset: infantile and adult. According to preclinical results the grafting of AMFA onto the enzyme has the potential to generate a drug able to be effective on both forms of the Pompe disease.

Today, an Enzyme Replacement Therapy (ERT) is only available for 7 diseases out of the 53 known lysosomal storage diseases. As the AMFA’s technology can be applied to existing as well as new recombinant enzymes, NanoMedSyn provides a technological platform able to generate a range of new drugs for treated and non-treated Lysosomal Storage Disorders.

The objectives in 6 points
Based on AMFA

NanoMedSyn’s development plan is based on a strong intellectual property of patented synthetic derivatives called AMFA.

The main application

is the production of new modified recombinant enzymes for the treatment of orphan lysosomal storage disorders.

Development

NanoMedSyn has decided to develop the lead compound enzyme-AMFA in the Pompe disease up to the clinical phases 1/2.

Pompe disease

Also known as glycogen storage disease type II, in which patients suffer from a deficiency of the lysosomal enzyme acid alpha-glucosidase.

The lysosomal accumulation

leads to a progressive deterioration of various organs.

NanoMedSyn provides

a technological platform able to generate a range of new drugs for treated and non-treated Lysosomal Storage Disorders.