FFC/TFCF – Phase 1

Task Force for Cystic Fibrosis

FFC/TFCF – Phase 1

The main objective of the Task Force for Cystic Fibrosis project is to develop new correctors and enhancers for the F508del mutation and, potentially, for other CF mutations. The project, which is expected to last three and a half years, is divided into three phases: 1) Hit identification; 2) Hit-to-lead; 3) Lead optimisation.
€ 0 still needed
0%
€ 200.000 goal

pRINCIPAL INVESTIGATOR

Luis Galietta (Lab. Genetica Molecolare, Ist. G. Gaslini, Genova)

Partner

Tiziano Bandiera (Dip.to Drug Discovery, Istituto Italiano Tecnologia, IIT, Genova)

Researchers

20

Category

AREA 4 Lung inflammation

Duration

1 year

Goal

€ 200.000

Funds raised

€ 200.000

Objectives

In the first phase, the hit identification phase (identification of the initial active compounds), which will last one year (from March 2014 to February 2015), chemical compounds with maximum structural diversity will be screened. A collection of more than 11,000 compounds available at the IIT (Italian Institute of Technology) will be used, carefully selected based on various criteria (in particular, structural novelty and the absence of potentially toxic chemical groups). The screening will use a method already in use for years in the Gaslini laboratory and based on the use of engineered cells. These cells have been genetically modified in the laboratory to simultaneously express both the mutated CFTR protein and a fluorescent sensor called YFP (yellow fluorescent protein), a protein that emits a yellow light when illuminated.

Project continues in Phase 2.

 

who adopted the project

Energy T.I. Group S.p.A. Milano

€ 100.000

LIFC Ass. Siciliana in ricordo di Davide Radicello

€ 20.000

Danone Italia

€ 50.000

OTHER PROJECTS

Discover the other projects

FFC/TFCF – Preclinical phase

The Task Force for Cystic Fibrosis project has identified new compounds that are highly active in vitro in correcting the CFTR protein. Among them, the most potent and promising compound, ARN23765, has been selected for preclinical development, an essential step towards its potential use in clinical trials in humans.

FFC Ricerca 2025 strategic project: De-risking GY

Assessing the safety and clinical potential of GY971, an anti-inflammatory compound for cystic fibrosis

FFC Ricerca 2025-2028 strategic project: MindKids-CF

Development of screening and monitoring tools for anxiety, depression, and behavior Problems in Italian Children with Cystic Fibrosis under 12 Years of Age