Patient-derived organoids as a model for treatment diagnosis in Cystic Fibrosis – combinatory treatment with read-through agent, correctors and potentiator on rare mutations.

Cystic fibrosis (CF) patient prognosis has recently improved due to the development of new, more effective treatments and better combination strategies for most patients carrying common mutations. However, there are more than 1500 known mutations in the CFTR gene - which cause CF - with some genotypes not yet considered for clinical treatment.

A biobank of organoids from a total of 498 CF patients has been established at HUB, which is representative of multiple different CF genotypes.

By making use of HUB’s CF biobank, we assessed the therapeutic potential of combinations of approved CFTR correctors, potentiators, and experimental read-through agent for CFTR function recovery in organoids with different types of CFTR mutations.

Download this poster to discover:

• The identification of responsive mutational variants not yet considered eligible for current therapy
• CFTR function recovery in organoids bearing multiple nonsense mutations by combination of CFTR correctors and potentiators with read-through agents
• How HUB Organoids® are facilitating personalized medicine approaches in CF