1 out 30 and you don’t know it  finaced for 100.000 Euros.
The project addresses the problem of the cystic fibrosis (CF) carriers following two lines of research and service: one of information and training, the other of health technology assessment with a multidisciplinary approach. The first line plans to create a website that represents a tool for an informed choice and aims to inform the population about the existence of the carrier test, the different ways in which it can be performed and the quality criteria. The website will allow users to guide the choice of whether or not to undergo the test, at what time, and in which version (targeted on CF or extended to other pathologies) and educate on the correct interpretation of the test result.
The second line of research will focus on health technology assessment (HTA) with the aim of performing an in-depth assessment of cystic fibrosis carrier screening in the Italian context. This part of the project aims to evaluate the efficacy, safety and any impact in organizational, economic, ethical, social and legal terms of the screening of the healthy carrier aimed at the general population.
The aim of the project is to reach, inform and train the largest possible number of people potentially interested in carrying out the carrier test, including health professionals who propose the test and are interested to deepen their knowledge through the use of a simple and interactive tool which also allows for self-assessment.
These tools will encourage an informed and conscious choice on whether to access the carrier test.
Project Manager: Carlo Castellani, Centro Fibrosi Cistica, Istituto Giannina Gaslini
Partner: Cinzia Colombo, Paola Mosconi (Lab. di Ricerca per il Coinvolgimento dei Cittadini in Sanità – Istituto Mario Negri); Chiara Gerardi, Rita Banzi (Centro Politiche Regolatorie in Sanità – Istituto Mario Negri); Emanuela Foglia, Lucrezia Ferrario, Daniele Bellavia, Elisabetta Garagiola, Fabrizio Schettini (LIUC Università Carlo Cattaneo); Giulia Candiani, Dania Puggioni (Zadig srl)

Molecole 3.0 for cystic fibrosis financed for 100.000 Euros.
This project aims to develop a new class of modulators also active on rare CFTR mutations. The research group plans to synthesize and test the compounds following an iterative process of chemical synthesis and in-vitro functional evaluations to select and to optimize a small number of compounds. The project is based on the complementarity of the laboratories of Prof. Barraja and Dr. Galietta. The research groups share their expertise in synthetic organic chemistry and pharmacology and they use consolidated procedures of chemical synthesis.
Thanks to previous funded projects by FFC Ricerca, FFC#4/2018 and FFC#3/2020, researchers have identified a molecule, belonging to a new class of correctors. After the optimizations of this first obtained molecule, a new one, called PP028, was also generated. This molecule, was already tested on primary cells obtained from CF patients, showing a strong activity, especially when combined with known correctors.
The “Molecole 3.0” project aims to further improve the properties of this new class of correctors, through the coordinated work of pharmaceutical chemists who will generate new derivatives and by biologists who will evaluate the effectiveness of the compounds with in-vitro assays.
The synthesis process and the functional evaluations will allow to improve the potency and efficacy of the selected compounds. The functional tests will be conducted with in vitro tests, also using the Primary Culture Service (Servizio Colture Primarie) by FFC Ricerca. The optimization of the molecules will also take into account the properties of solubility, metabolic stability and the absence of toxicity, which are essential for any drug development. The ultimate goal of the project is to select a compound that can be considered for preclinical and clinical development.
Project manager: Paola Barraja (STEBICEF – Laboratorio di sintesi degli eterocicli, Università di Palermo) – Luis Galietta (Università degli Studi di Napoli Federico II e Telethon Institute of Genetics and Medicine – TIGEM, Napoli)

TASK FORCE FOR CYSTIC FIBROSIS (TFCF) finaced for 130.000 Euros.
Project Manager; Tiziano Bandiera (Dip. Drug Discovery and Development, Istituto Italiano Tecnologia, IIT, Genova);
Partner: Nicoletta Pedemonte (Lab. Genetica Molecolare, Istituto G. Gaslini, Genova);
Co-responsible Partner: Luis Galietta (Università degli Studi di Napoli Federico II e Telethon Institute of Genetics and Medicine – TIGEM, Napoli)