DelveInsight’s, “Cystic Fibrosis Pipeline Insight 2025” report provides comprehensive insights about 75+ companies and 80+ pipeline drugs in Cystic Fibrosis pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

 

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Key Takeaways from the Cystic Fibrosis Pipeline Report

• In March 2025, Vertex Pharmaceuticals Incorporated announced a study is to evaluate the safety, and tolerability and efficacy of VX-522 in participants 18 years of age and older with cystic fibrosis and a cystic fibrosis transmembrane conductance regulator (CFTR) genotype not responsive to CFTR modulator therapy.
• In January 2025:- Enterprise Therapeutics Ltd.:- This 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 January 2025:- Boehringer Ingelheim:- This study is open to adult men with cystic fibrosis and adult women with cystic fibrosis who cannot have children. People with cystic fibrosis can join if they are not eligible to receive cystic fibrosis transmembrane conductance regulator modulator therapy (CFTR-MT). The purpose of this study is to find out how well a medicine called BI 3720931 is tolerated and whether it improves lung function in people with cystic fibrosis. In this study, BI 3720931 is given to humans for the first time.
• DelveInsight’s Cystic Fibrosis pipeline report depicts a robust space with 75+ companies working to develop 80+ pipeline therapies for Cystic Fibrosis treatment.
• The leading Cystic Fibrosis Companies such as Krystal Biotech, Vertex Pharmaceuticals, Translate Bio, Novartis, Algi Pharma, Verona Pharma, Atlantic Healthcare, Calithera Biosciences, Horizon Therapeutics, Santhera Pharmaceuticals, Reveragen Biopharma, Spli Sense, GlaxoSmithKline, EmphyCorp, Abbvie, Galapagos NV, Vertex Pharmaceuticals, PathBio Analytics, AstraZeneca, AxentisPharma AG, and others.
• Promising Cystic Fibrosis Pipeline Therapies such as Ciprofloxacin (Cipro Inhale, BAYQ3939), P-1037, Hypertonic Saline, VX-371 + HS, MP-376 (Levofloxacin solution for Inhalation), and others.

 

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Cystic Fibrosis Emerging Drugs Profile

 

• OligoG: Algi Pharma

OligoG is formulated as a dry powder for inhalation and is AlgiPharma’s lead drug candidate. It is an alginate oligosaccharide derived from seaweed, and is a new class of drug which is modulating mucus and normalizing mucus rheology. It is being developed to help people with cystic fibrosis clear mucus from their lungs. It is anticipated that OligoG may help to slow the progression of the disease. OligoG has been shown to disrupt the infectious biofilm often present in the lungs of individuals with CF. This biofilm disruption is believed to improve antibiotic effectiveness by increasing exposure of bacteria to antibiotics. It is in Phase 2 stage of development for the treatment of Cystic Fibrosis.

 

• Ensifentrine: Verona Pharma

Ensifentrine combines bronchodilator and anti-inflammatory properties in one compound and has the potential to be an effective treatment for COPD and other respiratory diseases, including asthma and cystic fibrosis. It is designed to maximize its effectiveness and reduce adverse events through: high selectivity for PDE3 and PDE4 over other enzymes and receptors to minimize off-target effects; direct delivery to the lungs by inhalation to maximize pulmonary exposure to ensifentrine while minimizing systemic distribution and potential adverse events. Ensifentrine also activates the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR), which is beneficial in reducing mucous viscosity and improving mucociliary clearance. This potentially makes it an attractive therapy for the treatment of cystic fibrosis. It is in Phase 2 stage of development for the treatment of Cystic Fibrosis.

 

• MRT5005: Translate Bio

MRT5005 is the first clinical-stage mRNA product candidate designed to address the underlying cause of CF by delivering mRNA encoding fully functional cystic fibrosis transmembrane conductance regulator (CFTR) protein to the lung epithelial cells through nebulization. MRT5005 is being developed to treat all patients with CF, regardless of the underlying genetic mutation, including those with limited or no CFTR protein. The U.S. Food and Drug Administration (FDA) has granted MRT5005 Orphan Drug, Fast Track and Rare Pediatric Disease designation. It is in Phase1/2 stage of development for the treatment of Cystic Fibrosis

 

• CB280: Calithera Biosciences

CB-280 is a potent and selective oral inhibitor of arginase. Arginase plays an important role in the pathophysiology of CF airway disease. Sputum from patients with CF has elevated arginase activity leading to diminished arginine levels. Reduced arginine is thought to exacerbate pulmonary disease in CF by impairing the production of nitric oxide, leading to a diminished anti-microbial immune response and impaired airway function. It is known that the airways of patients with CF have lower than normal nitric oxide production, and lower nitric oxide levels directly correlate with worsened lung function and increased colonization with pathogens, including Pseudomonas aeruginosa. It is in Phase1 stage of development for the treatment of Cystic Fibrosis.

 

• KB407 : Krystal Biotech

KB407 is a 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. It is in Pre-Clinical stage of development for the treatment of Cystic Fibrosis.

 

• SPL84231: SpliSense

SPL84-23-1, is designed to be mainly delivered via inhalation, effectively penetrates the target cells in the lungs, and binds to the target region, thereby preventing the inclusion of 84 intronic nucleotides as a cryptic exon, and generating a fully functioning CFTR protein. It is in Pre-Clinical stage of development for the treatment of Cystic Fibrosis.

 

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Cystic Fibrosis Companies

Krystal Biotech, Vertex Pharmaceuticals, Translate Bio, Novartis, Algi Pharma, Verona Pharma, Atlantic Healthcare, Calithera Biosciences, Horizon Therapeutics, Santhera Pharmaceuticals, Reveragen Biopharma, Spli Sense, GlaxoSmithKline, EmphyCorp, Abbvie, Galapagos NV, Vertex Pharmaceuticals, PathBio Analytics, AstraZeneca, AxentisPharma AG, 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

• Intra-articular
• Intraocular
• Intrathecal
• Intravenous
• Ophthalmic
• Oral
• Parenteral
• Subcutaneous
• Topical
• Transdermal
• Molecule Type

 

Products have been categorized under various Molecule types such as

• Oligonucleotide
• Peptide
• Small molecule
• Product Type

 

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Scope of the Cystic Fibrosis Pipeline Report

• Coverage- Global
• Cystic Fibrosis Companies- Krystal Biotech, Vertex Pharmaceuticals, Translate Bio, Novartis, Algi Pharma, Verona Pharma, Atlantic Healthcare, Calithera Biosciences, Horizon Therapeutics, Santhera Pharmaceuticals, Reveragen Biopharma, Spli Sense, GlaxosmithKline, EmphyCorp, Abbvie, Galapagos NV, Vertex Pharmaceuticals, PathBio Analytics, AstraZeneca, AxentisPharma AG, and others.
• Cystic Fibrosis Pipeline Therapies- Ciprofloxacin (Cipro Inhale, BAYQ3939), P-1037, Hypertonic Saline, VX-371 + HS, MP-376 (Levofloxacin solution for Inhalation), 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

 

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Table of Content

1. Introduction
2. Cystic Fibrosis Executive Summary
3. Cystic Fibrosis: Overview
4. Cystic Fibrosis Pipeline Therapeutics
5. Cystic Fibrosis 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. OligoG : Algi pharma
12. Drug profiles in the detailed report…..
13. Early Stage Products (Phase I)
14. CB280:Calithera Biosciences
15. Drug profiles in the detailed report…..
16. Preclinical and Discovery Stage Products
17. SPL84231: Spli Sense
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 Companies
27. Appendix

 

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