Cystic Fibrosis Pipeline
DelveInsight's, "Cystic Fibrosis Pipeline Insight 2023" report provides comprehensive insights about 75+ companies and 80+ pipeline drugs in the Cystic Fibrosis pipeline landscape. It covers the Cystic Fibrosis pipeline drug profiles, including Cystic Fibrosis clinical trials 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.
For Cystic Fibrosis Emerging drugs, the Cystic Fibrosis pipeline analysis report provides a 360° view of the therapeutics landscape by development point, product type, route of administration, molecule type, and MOA. The Cystic Fibrosis pipeline research covers business opportunities, challenges, future partnerships, strong competitors, and growth strategies.
In the Cystic Fibrosis Pipeline Report, a detailed description of the drug is given which includes the mechanism of action of the drug, Cystic Fibrosis clinical trials studies, Cystic Fibrosis NDA approvals (if any), and product development activities comprising the technology, Cystic Fibrosis collaborations, licensing, mergers and acquisition, funding, designations, and other product-related details.
Key Takeaways from the Cystic Fibrosis Pipeline Report
• Over 75+ Cystic Fibrosis companies are evaluating 80+ Cystic Fibrosis pipeline therapies in various stages of development, and their anticipated acceptance in the Cystic Fibrosis market would significantly increase market revenue.
• The leading Cystic Fibrosis Companies includes 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 Pipelines Therapies such as OligoG, Ensifentrine, MRT5005, CB280, KB407, SPL84231, and others.
• The Cystic Fibrosis Companies and academics are working to assess challenges and seek opportunities that could influence Cystic Fibrosis R&D. The Cystic Fibrosis pipeline therapies under development are focused on novel approaches to treat/improve Cystic Fibrosis.
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Cystic Fibrosis Overview
Cystic fibrosis is a progressive, genetic disease that causes long-lasting lung infections and limits the ability to breathe over time. More than 30,000 children and adults in the United States have CF (70,000 worldwide) and CF affects people of every racial and ethnic group. In people with CF, mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene cause the CFTR protein to become dysfunctional.
Recent Developmental Activities in the Cystic Fibrosis Treatment Landscape
• On March 2023, Insmed Incorporated announced a study of phase 2 clinical trials for Brensocatib. The main objective of the study is to evaluate the pharmacokinetics of brensocatib in participants with cystic fibrosis following once daily oral administration of study drug and to evaluate the safety of brensocatib compared to placebo in participants with cystic fibrosis (CF) over the 4-week treatment period.
• On March 2023, Aridis Pharmaceuticals Inc announced a study of phase 1 & 2 clinical trials for Inhaled AR- 501. This is a Phase 1/2a randomized, double-blind, two-part, dose-ascending, multicenter study of AR-501 (gallium citrate) solution, administered via inhalation, in healthy adult and P. aeruginosa infected cystic fibrosis (CF) subjects. Phase 1 of the study in HV subjects will consist of a single-ascending-dose (SAD) cohort, followed by the HV multiple-ascending-dose (MAD) cohort. Phase 2a of the study in CF subjects will consist of a MAD study design. The study will evaluate the safety and pharmacokinetic (PK) profile of single and repeat administrations of inhaled AR-501 solution in healthy adults, and the safety, PK and efficacy of repeat administrations of inhaled AR-501 solution in P. aeruginosa infected CF subjects.
For further information, refer to the detailed Cystic Fibrosis Drugs Launch, Cystic Fibrosis Developmental Activities, and Cystic Fibrosis News, click here for Cystic Fibrosis Ongoing Clinical Trial Analysis- www.delveinsight.com/sample-request/cystic-fibrosis-pipeline?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr
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.
Cystic Fibrosis Pipeline Therapeutics Assessment
There are approx. 75+ key companies which are developing the therapies for Cystic Fibrosis. The companies which have their Cystic Fibrosis drug candidates in the most advanced stage, i.e. phase II include, Algi Pharma.
Find out more about the Cystic Fibrosis Pipeline Segmentation, Therapeutics Assessment, Cystic Fibrosis Emerging Drugs @ Cystic Fibrosis Treatment Landscape- www.delveinsight.com/sample-request/cystic-fibrosis-pipeline?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr
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 Pipelines Therapies- OligoG, Ensifentrine, MRT5005, CB280, KB407, SPL84231, and others.
• Cystic Fibrosis Pipeline Segmentation: Product Type, Molecule Type, Route of Administration, Mechanism of Action
Dive deep into rich insights for drugs for Cystic Fibrosis Pipeline Companies and Therapies, click here @ Cystic Fibrosis Unmet Needs and Analyst Views- www.delveinsight.com/sample-request/cystic-fibrosis-pipeline?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr
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. 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- Key Companies
27. Appendix
Got Queries? Find out the related information on Cystic Fibrosis Mergers and acquisitions, Cystic Fibrosis Licensing Activities @ Cystic Fibrosis Recent Trends, and Future Perspectives- www.delveinsight.com/sample-request/cystic-fibrosis-pipeline?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr
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