The research group proceeds the study of the mimetic peptide PI3Kγ derived from the enzyme phosphatidylinositol 3-kinase gamma, which has been shown to influence the functionality of CFTR (projects FFC#8/2018, FFC#25/2014 and FFC#23/2015). Thanks to the results of previous projects, the PI3Kγ peptide has shown a synergistic action with the combinations of modulators of the CFTR channel for patients carrying the already approved F508del-CFTR allele (Kaftrio). With this project, the research team will evaluate whether PI3Kγ MP is able to restore CFTR recovery even in the presence of rare mutations (class III-IV, such as G551S, G1244E, S549N, R117C, S945L) as a single agent or in combination with approved modulators, such as Kalydeco (ivacaftor) and Kaftrio (Ivacaftor/Tezacaftor/Elexacaftor). The research team also plans to study the peptide’s mechanism of action in restoring the mutated CFTR functionality. The evaluations of PI3Kγ activity will be performed in cell lines and in epithelial cells isolated from people with CF.
PI3Kg is a compound at an advanced pre-clinical development stage, the research team reports that certified safety and toxicology studies on animals are underway and that the safety assessments of the drug on humans will soon begin. The results of this project could be transferred to the patient through an accelerated path as they could benefit from already completed regulatory procedures.