FFC Ricerca 2022-2023 Strategic Project: Molecules 3.0 for CF. Phase 2

New generation of pharmacological modulators to rescue mutant CFTR protein

FFC Ricerca 2022-2023 Strategic Project: Molecules 3.0 for CF. Phase 2

New generation of pharmacological modulators to rescue mutant CFTR protein
€ - still needed
0%
€ 270.000 goal

Researchers

Category

AREA 1 Therapies to correct the underlying defect

Duration

1 year

Goal

€ 270.000

Funds raised

€ 270.000

Objectives

The project, renewed for a further year of activity, aims to develop a new class of modulators also active on rare mutations of CFTR. In the first phase of the project, researchers examined the characteristics of a specific molecule called PP028. Experiments conducted showed that PP028 could act as a class 3 corrector, such as elexacaftor (VX-445). Furthermore, through the strategy called scaffold hopping (SH) and computational simulations, new compounds called SH have been obtained starting from the already known structures of the PP compounds.

In this phase, the compounds PP and SH have been tested in vitro and the obtained results suggest that PP and SH are promising for the development of combined treatments. 

who adopted the project

Rotary Club di Verona e Provincia

€ 28.000

Delegazione FFC Ricerca del Lago di Garda

€ 100.000

Associazione – La camminata del respiro “È ora di vivere”

€ 130.000

Latteria Montello

€ 12.000

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