Cystic Fibrosis Pipeline Outlook 2025: Insights into Drug Development and Leading 50+ Companies
Stay ahead with the latest insights! Download DelveInsight's comprehensive Cystic Fibrosis Pipeline Report to explore emerging therapies, key players, and future treatment landscapes @ Cystic Fibrosis Pipeline Outlook Report
Key Takeaways from the Cystic Fibrosis Pipeline Report
In July 2025, Enterprise Therapeutics Ltd announced a study is the first to give ETD001 to people with CF. The study will be run in two parts. Part A will assess if ETD001 is safe to give to people with CF, and Part B will assess if ETD001 improves lung function. The study drug is taken twice a day, in Part A it is taken for 7 days and in Part B for 28 days. In Part B there will be a separate period where dummy medicine is given for 28 days so the treatments can be compared.
In July 2025, Vertex Pharmaceuticals Incorporated conducted a study is to evaluate the pharmacokinetics, safety, tolerability and efficacy of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants with at least 1 triple combination responsive (TCR) mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
DelveInsight's Cystic Fibrosis pipeline report depicts a robust space with 75+ active players working to develop 80+ pipeline therapies for Cystic Fibrosis treatment.
The leading Cystic Fibrosis Companies such as Verona Pharma, Armata Pharmaceuticals, 4D Molecular Therapeutics, Spirovant Sciences, Vertex Pharmaceuticals, Krystal Biotech, Porosome Therapeutics, SpliSense, Enterprise Therapeutics Ltd and others.
Promising Cystic fibrosis Pipeline Therapies such as Tigerase®, Pulmozyme®, danazol, Brensocatib, SPL84, ETD001, ELX/TEZ/IVA, BI 3720931, JBT-101 (lenabasum) and others.
Discover how the Cystic Fibrosis treatment paradigm is evolving. Access DelveInsight's in-depth Cystic Fibrosis Pipeline Analysis for a closer look at promising breakthroughs @ Cystic Fibrosis Clinical Trials and Studies
Cystic Fibrosis Emerging Drugs Profile
VX-121: Vertex Pharmaceuticals
VX-121 and tezacaftor are designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the CFTR protein. VX-561 (deutivacaftor) is a potentiator designed to keep CFTR proteins at the cell surface open longer to improve the flow of salt and water across the cell membrane, which helps hydrate and clear mucus from the airways. The triple combination of VX-121/tezacaftor/VX-561 is being developed as an investigational once-daily treatment for people with CF with certain mutations in the CFTR gene. It is currently being investigated in Phase III stage of development.
Brensocatib: AstraZeneca
Brensocatib is a small molecule, oral, reversible inhibitor of dipeptidyl peptidase I (DPP1) being developed by Insmed for the treatment of patients with bronchiectasis. DPP1 is an enzyme responsible for activating neutrophil serine proteases (NSPs), such as neutrophil elastase, in neutrophils when they are formed in the bone marrow. Neutrophils are the most common type of white blood cell and play an essential role in pathogen destruction and inflammatory mediation. In chronic inflammatory lung diseases, neutrophils accumulate in the airways and result in excessive active NSPs that cause lung destruction and inflammation. Brensocatib may decrease the damaging effects of inflammatory diseases such as bronchiectasis by inhibiting DPP1 and its activation of NSPs. The drug is currently being evaluated in Phase II clinical trial to evaluate the pharmacokinetics of brensocatib in participants with cystic fibrosis following once daily oral administration of study drug.
ELX-02: Eloxx Pharmaceuticals
ELX-02 is an investigational compound being developed as a therapy for genetic diseases caused by nonsense mutations such as cystic fibrosis. Structurally, ELX-02 is an aminoglycoside analogue that induces read-through of nonsense mutations through interaction with the ribosome, resulting in the production of full-length functional proteins. The therapy was designated an orphan drug in the US, an orphan medicinal product in Europe, and given fast track designation in the US. ELX-02 is being tested in CF patients carrying least one G542X mutation in two parallel open-label Phase II clinical trials.
S-1226: SolAeroMed Inc.
S1226 is SolAeroMed's lead therapy. S1226 is formulated to rapidly reopen constricted, mucus plugged airways, and should increase the effectiveness of respiratory drug delivery. The S1226 formulation consists of aerosolized carbon dioxide (CO2) and nebulized perflubron; which is delivered into the lung. The delivery of this formulation results in an immediate relaxant effect on the patient's constricted airways, supported by a lowering of surface tension in inflamed areas (resulting in enhanced bronchial dilation) and possible clearing of mucus plugs of blocked airways. SolAeroMed is currently conducrting a phase II clinical trial in cystic fibrosis.
The Cystic Fibrosis Pipeline Report Provides Insights into
The report provides detailed insights about companies that are developing therapies for the treatment of Cystic Fibrosis with aggregate therapies developed by each company for the same.
It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Cystic Fibrosis Treatment.
Cystic Fibrosis Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
Cystic Fibrosis Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Cystic Fibrosis market
Explore groundbreaking therapies and clinical trials in the Cystic Fibrosis Pipeline. Access DelveInsight's detailed report now! @ New Cystic Fibrosis Drugs
Cystic Fibrosis Companies
Verona Pharma, Armata Pharmaceuticals, 4D Molecular Therapeutics, Spirovant Sciences, Vertex Pharmaceuticals, Krystal Biotech, Porosome Therapeutics, SpliSense, Enterprise Therapeutics Ltd and others.
Cystic fibrosis pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
Intravenous
Subcutaneous
Oral
Intramuscular
Cystic Fibrosis Products have been categorized under various Molecule types such as
Monoclonal antibody
Small molecule
Peptide
Download DelveInsight's latest report to gain strategic insights into upcoming therapies and key developments @ Cystic Fibrosis Market Drivers and Barriers, and Future Perspectives
Scope of the Cystic Fibrosis Pipeline Report
Coverage- Global
Cystic Fibrosis Companies- Verona Pharma, Armata Pharmaceuticals, 4D Molecular Therapeutics, Spirovant Sciences, Vertex Pharmaceuticals, Krystal Biotech, Porosome Therapeutics, SpliSense, Enterprise Therapeutics Ltd and others.
Cystic fibrosis Pipeline Therapies- Tigerase®, Pulmozyme®, danazol, Brensocatib, SPL84, ETD001, ELX/TEZ/IVA, BI 3720931, JBT-101 (lenabasum) and others.
Cystic Fibrosis Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
Cystic Fibrosis Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
Which companies are leading the race in Cystic Fibrosis drug development? Find out in DelveInsight's exclusive Cystic Fibrosis Pipeline Report—access it now! @ Cystic Fibrosis Emerging Drugs and Major Companies
Table of Content
Introduction
Executive Summary
Cystic Fibrosis: Overview
Pipeline Therapeutics
Therapeutic Assessment
Cystic Fibrosis – DelveInsight's Analytical Perspective
Late Stage Products (Phase III)
Drug name: Company Name
Drug profiles in the detailed report…..
Mid-Stage Products (Phase II)
AP-PA02: Armata Pharmaceuticals
Drug profiles in the detailed report…..
Early Stage Products (Phase I)
VX-522: Vertex Pharmaceuticals
Drug profiles in the detailed report…..
Preclinical and Discovery Stage Products
SPL23: SpliSense
Drug profiles in the detailed report…..
Inactive Products
Cystic Fibrosis -Key Companies
Cystic Fibrosis -Key Products
Cystic Fibrosis - Unmet Needs
Cystic Fibrosis - Market Drivers and Barriers
Cystic Fibrosis - Future Perspectives and Conclusion
Cystic Fibrosis -Analyst Views
Cystic Fibrosis- Key Companies
Appendix
About Us
DelveInsight is a leading healthcare-focused market research and consulting firm that provides clients with high-quality market intelligence and analysis to support informed business decisions. With a team of experienced industry experts and a deep understanding of the life sciences and healthcare sectors, we offer customized research solutions and insights to clients across the globe. Connect with us to get high-quality, accurate, and real-time intelligence to stay ahead of the growth curve.
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