Neuroinflammation, characterized by microglial cell-activation, plays a major role in the pathogenesis and progression of multiple neurodegenerative diseases, such as Lysosomal Storage Disorders and Alzheimer’s, severe human conditions currently lacking an effective treatment.
Ex vivo gene therapy based on genetically modified hematopoietic stem cells (HSCs) approach shows great promise, yet the beneficial effect of it relies on CNS-associated microglia-like cells derived from the transplanted HSCs due to their role in neuroinflammation reduction after treatment.
To increase the efficacy of such therapeutic strategy, we have developed a proprietary synthetic promoter for a gene transfer vector that allows for a controlled –low basal levels that are significantly increased upon inflammation– expression of therapeutic transgenes in immune-like cells.
The inducible promoter sequence is validated both in vitro and in vivo.

TRL (Technology Readiness Level)

Lead Optimization/Preclinical

Inventors

Alessandra Biffi

Valentina Poletti

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PoC@Unipd project 2022

With the aim of supporting the competitiveness of businesses by enhancing industrial properties, the Ministry for Economic Development (MISE) has published a call for the establishment of patent enhancement programs, through funding of Proof of Concept (PoC) projects by Italian Universities, Public Research Institutions (EPR), and Scientific Institutions for Research, Hospital and Healthcare (IRCS).

Purpose of the call is establishing TRL (Technology Readiness Level) enhancement paths for technologies patented during academic research, and create the conditions to turn them into strategic opportunities for the business world.

After careful examination of the project proposals received by the Ministry Evaluation Committee, the University of Padua was authorized to develop 8 PoC projects. The projects were selected by the University through an internal call and the program took the first steps in December 2023. From that moment on, the PoC Team started to work to validate the relevant technologies. 

The academic work team has been flanked by an external Expert Committee, comprising top scientific and professional resources (investors and entrepreneurs), capable of providing a respected standpoint on all strategic and planning choices.

UniSMART – Fondazione Università di Padova was tasked with managing the enhancement of the results of 8 projects, through its highly qualified, innovation-oriented staff, in the different PoC project sectors.

PROGETTO PoC@UNIPD2022 – PNRR – Missione 1, Componente 2, Investimento 6 – finanziato dall’Unione Europea – NextGenerationEU

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