The use of CFTR (cystic fibrosis transmembrane conductance regulator) correctors for the treatment of rare and devastating genetic muscular diseases, namely sarcoglycanopathies and Brody myopathy, is the object of this invention.
Sarcoglycanopathies and Brody myopathy are rare inherited striated muscle diseases that lead to severe disability and are potentially lethal. Currently no available treatment exists. Both diseases are caused by the loss of function of a potentially active protein that is unable to retain its correct structure. To re-establish its function, this invention focuses on favouring the folding of the faulty protein by using molecules known as CFTR correctors, already selected for the treatment of cystic fibrosis. Although on its own each disease is rare, the two put together affect a significant number of the population, notwithstanding the incidence of Brody myopathy is likely largely underestimated.

TRL (Technology Readiness Level)

Lead Optimization/Preclinical

Inventors

Pompeo
Volpe

Romeo
Betto

Francesco
Mascarello

Elisa
Bianchini

Roberta
Sacchetto

Dorianna
Sandonà

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PoC@Unipd project

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 ranked 4th in the classification, and was authorized to develop 9 PoC projects.

The projects were selected by the University through an internal call and the program took the first steps in February 2021. 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 9 projects, through its highly qualified, innovation-oriented staff, in the different PoC project sectors.

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