Objectives

N2ERT aims to eliminate some bottlenecks in the development of enzyme replacement proteins by optimising the flow of technological activities (NMed production) and safety and efficacy testing.
It addresses the need to deliver ERT into the CNS, for the neurological complications of alpha-mannosidosis deficiency disease (aMAN) and acid sphingomyelinase. For both diseases there are approved enzyme therapies (Lamzede and Xenpozyme), but limited to peripheral complications of the diseases, as the enzyme is unable to cross the BBB.

After the design and production of the NMed (CIDSTEM, NanoTech lab), they will be tested for safety and efficacy on cellular platforms based on in vitro models of the target diseases coupled to high content screening systems (IRET Foundation, CIRI-SdV) and, finally, in vivo (IRET Foundation), also following the GLP regulatory framework in both cases.