FFC#11/2017

Development of a peptide derived from the enzyme PI3Kγ as a new and effective potentiator of the mutant F508del-CFTR

AREA 1 Therapies to correct the underlying defect

FFC#11/2017

Development of a peptide derived from the enzyme PI3Kγ as a new and effective potentiator of the mutant F508del-CFTR
€ 0 still needed
0%
€ 60.000 goal

pRINCIPAL INVESTIGATOR

Alessandra Ghigo (Dipartimento di Biotecnologie Molecolari e Scienze per la Salute, Università degli Studi di Torino)

Researchers

7

Category

AREA 1 Therapies to correct the underlying defect

Duration

1 year

Goal

€ 60.000

Funds raised

€ 60.000

Objectives

Researchers demonstrated that the peptide derived from the PI3Kγ enzyme (patent n°WO/2016/103176) acts both as a potentiator and a corrector of CFTR carrying F508del mutation, appearing more efficient than currently used drugs, including the standard potentiator, VX-770, and the combined-action drug (potentiator/corrector) Orkambi® (Project FFC#23/2015 and FFC #4/2016).This project aims at completing the optimization of the PI3Kγ-derived peptide, synthesizing a molecule with preserved biological activity and better pharmacokinetic profile for CF therapy, and clarify the underlying mechanisms of action. The potency and toxicity of newly synthesized PI3Kγ peptide derivatives will be studied in vitro and in vivo (BALB/c mice), the therapeutic efficacy in human F508del intestinal organoids and bronchial epithelial cells. The ultimate goal is to validate a novel therapeutic strategy for CF basic defect treatment, in view of the potential development of PI3Kγ peptide as human medicinal product (Orphan Drug Designation N°EU/3/17/1859).

WHO ADOPTED THE PROJECT

Lifc Toscana Onlus

€ 60.000

“Un fiore per Valeria” Assemini – Cagliari

€ 8.000

Gruppo di Sostegno FFC di Vigevano

€ 15.000

OTHER PROJECTS

Discover the other projects

GMRF#1/2026

Exploring the role of PKD1 in promoting CFTR stability and function at the cell surface

FFC#1/2026

Mapping proteins regulating CFTR mRNA stability to identify new therapeutic targets for nonsense mutations

FFC#2/2026

Evaluating the potential of phosphodiesterase inhibitors to enhance the efficacy of CFTR modulators and support personalized therapeutic approaches