Evidence map›Paper›PMID 40202089›Full record

ReviewExpert opinion on drug discovery2025

Recent developments in cystic fibrosis drug discovery: where are we today?

Miquéias Lopes-Pacheco, Ashlyn G Winters, JaNise J Jackson, John A Olson, Minsoo Kim, Kaitlyn V Ledwitch, Austin Tedman, Ashish R Jhangiani, Jonathan P Schlebach, Jens Meiler and 2 more

Abstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Expert opinion on drug discovery, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

4 citing papers in PubMed.

  1. Article
  2. Article
  3. Article
  4. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

12 authors.

Miquéias Lopes-PachecoDepartment of Pediatrics, Emory University School of Medicine, Atlanta, GA, USA.ORCID 0000-0002-7444-9359
Ashlyn G WintersDepartment of Pediatrics, Emory University School of Medicine, Atlanta, GA, USA.
JaNise J JacksonDepartment of Pediatrics, Emory University School of Medicine, Atlanta, GA, USA.
John A OlsonDepartment of Chemistry, Vanderbilt University, Nashville, TN, USA.ORCID 0009-0000-9654-6611
Minsoo KimDepartment of Chemistry, Vanderbilt University, Nashville, TN, USA.
Kaitlyn V LedwitchDepartment of Chemistry, Vanderbilt University, Nashville, TN, USA.
Austin TedmanThe James Tarpo Junior & Margaret Tarpo Department of Chemistry, Purdue University, West Lafayette, IN, USA.
Ashish R JhangianiThe James Tarpo Junior & Margaret Tarpo Department of Chemistry, Purdue University, West Lafayette, IN, USA.
Jonathan P SchlebachThe James Tarpo Junior & Margaret Tarpo Department of Chemistry, Purdue University, West Lafayette, IN, USA.
Jens MeilerDepartment of Chemistry, Vanderbilt University, Nashville, TN, USA.
Lars PlateDepartment of Chemistry, Vanderbilt University, Nashville, TN, USA.
Kathryn E OliverDepartment of Pediatrics, Emory University School of Medicine, Atlanta, GA, USA.ORCID 0000-0002-7781-7006

Funding

Rational optimization of combinatorial therapies for the treatment of rare cystic fibrosis variantsR01HL167046 · NHLBI · PURDUE UNIVERSITY · PI Lars Plate, Jonathan Patrick Schlebach · 2023 to 2026
$2.7M
Graduate Training Program in Quantitative and Chemical Biology at Indiana University BloomingtonT32GM131994 · NIGMS · TRUSTEES OF INDIANA UNIVERSITY · PI JARED C LEWIS · 2019 to 2026
$2.4M
Tuning translation efficiency to overcome refractory defects in CFTRR01HL173500 · NHLBI · EMORY UNIVERSITY · PI Kathryn E Oliver · 2025 to 2026
$1.2M
NHLBI NIH HHS R01 HL167046NHLBI NIH HHS R01 HL173500NIGMS NIH HHS T32 GM131994
6 · The paper itself

Abstract

introductionThe advent of variant-specific disease-modifying drugs into clinical practice has provided remarkable benefits for people with cystic fibrosis (PwCF), a multi-organ life-limiting inherited disease. However, further efforts are needed to maximize therapeutic benefits as well as to increase the number of PwCF taking CFTR modulators. AREA COVERED: The authors discuss some of the key limitations of the currently available CFTR modulator therapies (e.g. adverse reactions) and strategies in development to increase the number of available therapeutics for CF. These include novel methods to accelerate theratyping and identification of novel small molecules and cellular targets representing alternative or complementary therapies for CF. EXPERT OPINION: While the CF therapy development pipeline continues to grow, there is a critical need to optimize strategies that will accelerate testing and approval of effective therapies for (ultra)rare CFTR variants as traditional assays and trials are not suitable to address such issues. Another major barrier that needs to be solved is the restricted access to currently available modulator therapies, which remains a significant burden for PwCF who are from racial and ethnic minorities and/or living in underprivileged regions.

Indexed as

Cystic FibrosisDrug DevelopmentDrug DiscoveryAnimalsCystic Fibrosis Transmembrane Conductance RegulatorHumansMolecular Targeted TherapyCFTR protein, humanCystic Fibrosis Transmembrane Conductance RegulatorCFTR modulatordrug developmentfunctional genomicshigh throughput methodsprecision medicineproteomicstheratypingtranscriptomics

Identifiers

PMID40202089
PMCPMC12088121

What OpenQuestion holds

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Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.