DelveInsight’s, “Cystic fibrosis Pipeline Insight, 2025” report provides comprehensive insights about 50+ companies and 55+ pipeline drugs in Cystic fibrosis pipeline landscape. It covers the Cystic Fibrosis pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Cystic Fibrosis pipeline therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

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 May 2025, Boehringer Ingelheim announced a study is for people with cystic fibrosis who took part in a previous study with a medicine called BI 3720931. The main purpose of this study is to monitor the long-term health of participants who were treated with BI 3720931 in the previous study. Participants in this study do not receive additional treatment with BI 3720931.
• In May 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 50+ active players working to develop 55+ 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

• AP-PA02: Armata Pharmaceuticals

AP-PA02 is a therapeutic phage cocktail that targets the pathogen P. aeruginosa, to treat serious respiratory infections, with an emphasis on patients with cystic fibrosis (CF) and non-cystic fibrosis bronchiectasis (NCFB). Phages that comprise AP-PA02 were selected with desired attributes for a product candidate targeting P. aeruginosa lung infections. AP-PA02 is comprised of a cocktail of natural P. aeruginosa phages originating from distinct families and subfamilies, targeting multiple receptor classes, functioning with compatibility and cooperativity, and further characterized by being highly potent and having a broad host range. Currently, the drug is in the Phase II stage of its development for the treatment of Cystic Fibrosis.

• VX-522: Vertex Pharmaceuticals

VX-522 is an investigational messenger ribonucleic acid (mRNA) therapy aims to address the underlying cause of cystic fibrosis (CF). It is being evaluated by Vertex to treat lung disease for people living with CF who cannot benefit from cystic fibrosis transmembrane conductance regulator (CFTR) modulator treatments because they do not make any CFTR protein that responds to a CFTR modulator therapy. VX-522 is delivered to the lung through inhalation of a CFTR mRNA encapsulated by a lipid nanoparticle. Once delivered to the target lung cells, the mRNA is designed to produce functional copies of the CFTR protein. VX-522 is the result of an exclusive research collaboration established with Moderna in 2016. Currently the drug is in Phase I/II stage of its development for the treatment of Cystic fibrosis.

• KB407: Krystal Biotech

KB407 is an investigational, redosable gene therapy designed to correct the underlying cause of CF by delivering two copies of the CFTR gene directly to the airway epithelial cells when delivered via a nebulizer. By inducing expression of full length, normal CFTR protein in the lung, treatment with KB407 has potential to restore ion and water flow into and out of lung cells to correct the lung manifestations of the disease in patients regardless of their underlying genetic mutation. Currently the drug is in Phase I stage of its development for the treatment of Cystic fibrosis.

• SPL23: SpliSense

The CF W1282X mutation generates a Premature Termination Codon (PTC), leading to RNA degradation induced by the Nonsense Mediated RNA Decay (NMD) mechanism. W1282X does not respond to CFTR modulator drugs. Heterozygous patients carrying the F508del mutation and a minimal function mutation are responsive to Vertex triple combination therapy (Trikafta®). Significantly, around 50% of CF patients carrying W1282X also carry a non-F508del mutation on the second allele. For those patients, no effective treatment is available. SPL23 ASO given by inhalation, penetrates the cells and induces skipping over exon 23 in the CFTR transcript. Skipping exon 23 bypasses the NMD leading to mature and active CFTR. The activity of these CFTR channels can be further augmented by CFTR modulators. Currently the drug is in Preclinical stage of its development for the treatment of 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

1. Introduction
2. Executive Summary
3. Cystic Fibrosis: Overview
4. Pipeline Therapeutics
5. Therapeutic Assessment
6. Cystic Fibrosis – DelveInsight’s Analytical Perspective
7. Late Stage Products (Phase III)
8. Drug name: Company Name
9. Drug profiles in the detailed report…..
10. Mid-Stage Products (Phase II)
11. AP-PA02: Armata Pharmaceuticals
12. Drug profiles in the detailed report…..
13. Early Stage Products (Phase I)
14. VX-522: Vertex Pharmaceuticals
15. Drug profiles in the detailed report…..
16. Preclinical and Discovery Stage Products
17. SPL23: SpliSense
18. Drug profiles in the detailed report…..
19. Inactive Products
20. Cystic Fibrosis -Key Companies
21. Cystic Fibrosis -Key Products
22. Cystic Fibrosis – Unmet Needs
23. Cystic Fibrosis – Market Drivers and Barriers
24. Cystic Fibrosis – Future Perspectives and Conclusion
25. Cystic Fibrosis -Analyst Views
26. Cystic Fibrosis- Key Companies
27. Appendix

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