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Cystic Fibrosis Drug Development Pipeline 2025: Gene Therapy and CFTR Modulators

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What is the projected growth rate (CAGR) of the Cystic Fibrosis Pipeline Insights 2025, and what is the market value expected to change by 2033?

The Cystic Fibrosis Market is projected to reach a significant value by 2033, growing at a promising CAGR during the forecast period.

What is Cystic Fibrosis Pipeline Insights 2025?

Cystic fibrosis (CF) is a rare, inherited genetic disorder that primarily affects the lungs and digestive system, caused by mutations in the CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) gene.

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Top Key Companies:

Spirovant Sciences, Inc., Vertex Pharmaceuticals Incorporated, SpliSense Ltd., Enterprise Therapeutics Ltd, Boehringer Ingelheim, International GmbH, 4D Molecular Therapeutics, Clarametyx Biosciences, Inc., Arcturus Therapeutics, Inc., Krystal Biotech, Inc., Cystetic Medicines, Inc., Kither Biotech Srl, ReCode Therapeutics.

Pipeline Analysis:

In January 2025, Arcturus Therapeutics announced the initiation of dosing for participants with CF and OTC deficiency in its Phase II multiple ascending dose study of ARCT-032, with patients receiving daily treatments over 28 days.

In September 2024, Vertex Pharmaceuticals shared updates on its Phase III trial for the investigational once-daily triple combination therapy vanzacaftor/tezacaftor/dutivacaftor (“vanza triple”), which will be presented at the North American Cystic Fibrosis Conference (NACFC).

Meanwhile, Clarametyx Biosciences revealed in October 2024 that it has completed the Phase Ib portion of its trial evaluating CMX-101, a novel immune-enabling antibody therapy for CF-associated pulmonary infections, and is now advancing to Phase IIa. These developments underscore the growing momentum in CF therapeutic innovation.

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Technology Roadmap Analysis
Sustainability Impact Analysis
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Market Volatility & Emerging Risks Analysis
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