Cystic fibrosis is an autosomal recessive, multisystem disorder that has been historically

associated with poor life expectancy. Due to the defective cystic fibrosis transmembrane conductance

regulator protein, patients with cystic fibrosis develop viscous secretions that are difficult to clear, resulting

in numerous abnormalities such as chronic airway obstruction, maldigestion and malabsorption. While our

understanding of the pathophysiology and disease management have improved, pulmonary disease remains

the leading cause of morbidity and mortality in patients with cystic fibrosis. However, since the introduction

of precision medicine, novel therapeutic agents have been developed to target the underlying defective

protein, resulting in improved disease management and life expectancy. The goal of precision medicine is

to provide timely diagnosis, phenotyping, and personalized treatments, based on an individualized analysis

of a patient’s genome. This article reviews current and potential precision medicine treatments for patients

with cystic fibrosis, including cystic fibrosis transmembrane conductance regulator modulators and other

modulators designed for patients who would not benefit from currently available therapies. We will also

discuss other investigational treatment modalities, such as ribosomal read-though agents and RNA therapy,

which may continue the advancement of cystic fibrosis treatment. Current research into methods aimed to

better predict patients’ responses to personalized treatment, such as theratyping, will also be discussed. Given

the benefits of applying precision medicine in cystic fibrosis, future research in this therapeutic approach will

also likely benefit other life-threatening monogenetic disorder