Pre-clinical development<< Back to Drug research and development
This section outlines the different classes of agents, based on their mechanism of action, that are currently being developed for the treatment of cystic fibrosis. There are two main categories: CFTR-modulating therapies (mutation-specific therapies); and non-CFTR-based therapies.
This section outlines how CFTR therapies are validated in pre-clinical models before they enter clinical trials.
This section summarizes the development of therapeutic approaches to target different classes of CFTR mutations.
Non-CFTR-modulating therapies aim to restore CFTR function by modulating cellular components to restore ion transport or by introducing a new non-mutated copy of the CFTR gene to the cell.