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narrative_review·pulmonology, pediatrics, internal medicine, clinical trial, biomedical research·PMC10532558

Advances in the Cystic Fibrosis Drug Development Pipeline

Life · 6 authors, 3 centres

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AGENT 01 · STRUCTURED SUMMARY

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TL;DR

The central implication is that the abundance of novel therapies represents a dramatic and optimistic shift in the prognosis for people with CF.

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144 CHARS

The central implication is that the abundance of novel therapies represents a dramatic and optimistic shift in the prognosis for people with CF.

STUDY TYPE

narrative_review

SPECIALTY

pulmonology

SUMMARISED BY

AI pipeline

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100% · A

ORIGINAL PAPER

PMC10532558

Advances in the Cystic Fibrosis Drug Development Pipeline

Life

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ABSTRACT

Abstract Cystic fibrosis is a genetic disease that results in progressive multi-organ manifestations with predominance in the respiratory and gastrointestinal systems. The significant morbidity and mortality seen in the CF population has been the driving force urging the CF research community to further advance treatments to slow disease progression and, in turn, prolong life expectancy. Enormous strides in medical advancements have translated to improvement in quality of life, symptom burden, and survival; however, there is still no cure. This review discusses the most current mainstay treatments and anticipated therapeutics in the CF drug development pipeline within the mechanisms of mucociliary clearance, anti-inflammatory and anti-infective therapies, restoration of the cystic fibrosis transmembrane conductance regulator (CFTR) protein (also known as highly effective modulator therapy (HEMT)), and genetic therapies. Ribonucleic acid (RNA) therapy, gene transfer, and gene editing are being explored in the hopes of developing a treatment and potential cure for people with CF, particularly for those not responsive to HEMT. Keywords: cystic fibrosis (CF), cystic fibrosis transmembrane conductance regulator (CFTR), highly effective modulator therapy (HEMT), gene-based therapy, antibiotics, bacteriophage, anti-inflammatory, mucolytic therapy

CITATION EXPORT

Christine Esposito, Martin Kamper, Jessica Trentacoste et al.. Advances in the Cystic Fibrosis Drug Development Pipeline. Life. (2023). https://doi.org/10.3390/life13091835

AUTHORS

6 · 3 CENTRES

Christine Esposito, Martin Kamper, Jessica Trentacoste, Susan Galvin, Halie Pfister, Janice Wang

1Division of Pulmonary, Critical Care and Sleep Medicine, Donald and Barbara Zucker School of Medicine at Hofstra/Northwell, New Hyde Park, New York, NY 11042, USA; [email protected] (M.K.); [email protected] (J.W.)
2Division of Pediatric Pulmonology, The Steven and Alexandra Cohen Children’s Medical Center, Donald and Barbara Zucker School of Medicine at Hofstra/Northwell, Lake Success, New York, NY 11042, USA; [email protected]
3Manhasset Office of Clinical Research, The Feinstein Institutes for Medical Research, Lake Success, New York, NY 11042, USA; [email protected]