Partners

N2ERT is realised by a 3-partner consortium, all laboratories of the High Technology Network, joined by two companies interested in the project and its products.

The laboratories involved include a multi-disciplinary team (chemistry, pharmaceutical technologies, neurobiology, translational medicine) which, in this transversal project, find their operational place and expertise in different aspects, connected and autonomous, but intertwined for the purpose of achieving the study objective, i.e. a transition from TRL4 to TRL6 of a pharmaceutical product. These multidisciplinary transversal skills are a distinctive and characterising element of this project, with expertise in nanomedicine, enzyme delivery, in vitro and in vivo testing in GLP-certified environments, and expertise in the field of therapy of rare genetic diseases and the regulatory procedures necessary for their market entry.

Coordinatore

IRET Foundation is a scientific research organisation in the biomedical field and an accredited laboratory of the High Technology Network. It hosts the Bologna Technopole – Ozzano site, of which it is the managing body, and a section of the CIRI-SDV, University of Bologna.
IRET specialises in preclinical efficacy testing on disease models performed in laboratory animals. It has a long experience in collaborations for preclinical and clinical research with the pharmaceutical and biomedical industry, and has developed numerous in vitro (2D, 3D and 4D) and in vivo screening and biochemical tests of enzyme activity.
IRET’s role in the project will be that of: – coordination of activities and reporting, management of project meetings, coordination of relations with companies – management of colonies of aMAN and ASM mutated mice – set-up of prototype cell platforms for 2 rare diseases for efficacy testing and definition of corresponding readouts – relations with TMR for safety studies in GLP – conduct of in vivo pilot study – responsible for dissemination.

Participants

CIRI-SdV – University of Bologna is an industrial research laboratory of the University of Bologna, accredited as a High Technology Network laboratory. It brings together biomedical and technological knowledge and skills covering the entire research and development chain, from the laboratory bench to the patient’s bedside and home.
CIRI-SdV will provide the project with its capacity to develop and validate prototypes of cellular platforms for high-content analysis for specific readouts, designed and manufactured according to the characteristics and expected results of the specific pharmaceutical product. For these activities, the CIRI-SDV has dedicated laboratories and related instrumentation.
The role of CIRI-SdV in the project will be to – provide support for the interpretation of the in vitro results in cell-based high content analysis – contribute to the design of the in vivo study – contribute to the choice of donor animals for the construction of the cell platforms.

CIDSTEM of the University of Modena and Reggio Emilia is an internationally renowned Interdepartmental Centre for advanced epithelial stem cell therapies. Equipped with a GMP pharmaceutical laboratory and with experts in pharmaceutical technology and delivery, the team engages in research, funded by national bodies and industrial collaborations, to develop delivery systems for the controlled release of active molecules. It focuses in particular on enzyme formulation for rare brain diseases, using surface modification technologies for selective and specific delivery. Skills in nanomedicine include the design and manufacture of nanomedicines, the use of biodegradable materials, formulation technologies and pharmaceutical characterization.
CIDSTEM will be involved in the design, manufacture and characterization of delivery systems for enzymes to the CNS, developing new formulations to improve their efficacy and efficiency.

Companies

Chiesi is a research-oriented international biopharmaceutical group that develops and commercializes innovative therapeutic solutions in the field of respiratory health, rare diseases and specialty care. In 2020 it founded the Global Rare Diseases Unit, and in November 2022 it started to build the Biotech Center of Excellence, specialising in the development and production of therapeutic proteins, including enzymes produced by recombinant DNA techniques.
It has several drugs for rare diseases in its portfolio and has collaborations in the development of candidate ERTs for crossing the blood-brain barrier in rare lysosomal storage diseases.

TransMed Research is certified by the Ministry of Health as a ‘Test Facility’ operating in Good Laboratory Practice. TMR was chosen for its readiness to provide certified studies on prototypes of nanoformulated materials and products produced by N2ERT. This availability has already been demonstrated in previous POR-FESR projects (Step-by-Step and Mat2Rep) and has made it possible to achieve more advanced TRLs than would have been possible in a laboratory environment such as that of accredited laboratories.
TMR is interested in expanding its portfolio of Standard Operating Procedures and certified studies to the field of nanoformulations.

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