Skip to main content

Activities

N2ERT aims to bridge the gap between the production of therapeutic systems and their biological validation, with an integrated platform for prototype production, evaluation and validation of advanced nanomedicines for therapeutic protein replacement. It will employ recombinant human therapeutic enzymes (aMAN alpha-mannosidase and AM acid sphingomyelinase) used for the treatment of rare neurometabolic lysosomal storage diseases.

N2ERT has the following technological objectives:

  1. Development of 2 NMed to protect the biological activity of the enzyme (nERT-aMAN and nERT-ASM);
  2. Development of at least 1 NMed designed for delivery across the blood-brain barrier (BBBnERT);
  3. Ability to deliver an effective dose (up to 10% of the injected dose) of enzymes into the CNS;
  4. Consolidation of rapid, accurate, efficient and robust ‘high-throughput’ in vitro safety tests for rare lysosomal storage diseases;
  5. Identification and validation of efficacy readout assays by rescuing the enzymatic activities of nERT-aMAN and nERT-ASM in vitro and in vivo.

Human fibroplasts labeled with LysoTracker to analyze lysosome morphology

Human fibroplasts labeled with LysoTracker to analyze lysosome morphology

Human fibroplasts visualized using three-dimensional image analysis software

Human fibroplasts visualized using three-dimensional image analysis software

LAMP1-labeled human fibroplasts for three-dimensional analysis of lysosome morphology

LAMP1-labeled human fibroplasts for three-dimensional analysis of lysosome morphology

Analysis of an image acquired by confocal microscopy of primary cultures of LAMP1-labeled neurons for lysosome diameter analysis

The N2ERT project is co-funded by the Emilia–Romagna ERDF Regional Programme (ERDF RP) 2021-2027, ACTION 1.1.2 

Close Menu