DelveInsight Evaluates a Robust Cystic Fibrosis Pipeline as 75+ Companies to Set Foot in the Domain

DelveInsight Evaluates a Robust Cystic Fibrosis Pipeline as 75+ Companies to Set Foot in the Domain

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.


Request a sample and discover the recent breakthroughs happening in the Cystic Fibrosis Pipeline landscape @ Cystic Fibrosis Pipeline Outlook Report


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. When the protein is not working correctly, it’s unable to help move chloride — a component of salt — to the cell surface.


Recent Developmental Activities in the Cystic Fibrosis Treatment Landscape


  • In December 2021, AlgiPharma has been awarded up to NOK 16 million from the Norwegian Research Councils Innovation Project for the Industrial Sector program for the project “Therapeutic Alginates for Resistant and Recurrent Infections: Generating Eradication Therapies (TARRGET)”. The project grant awarded from the Research Council combined with the Norwegian government tax incentive scheme (SkatteFUNN) results in a total award value for AlgiPharma of about NOK 22 million (about EUR 2.2 MM / USD 2.5 MM).


  • AlgiPharma has received more than USD 46 million in research and development grants from EU’s 7th framework program, Horizon 2020, and Eurostars programs, the Norwegian Research Council, Innovate UK, US Army through Congressional Earmark funding, and the Cystic Fibrosis Foundation (CFF).


  • In November 2021, Calithera Biosciences shared interim safety and efficacy results from a Phase 1b, randomized, double-blind, placebo-controlled, dose-escalation trial evaluating CB-280, the company’s investigational arginase inhibitor, in adults with cystic fibrosis (CF). The data were shared in a poster presentation at the North American Cystic Fibrosis Foundation Conference (NACFC; Abstract 529).CB-280 demonstrated linear pharmacokinetics with plasma exposure increasing proportionally with dose. Complete and continuous target inhibition in plasma was achieved at the 100 mg dose and above. CB-280 also demonstrated robust pharmacodynamic effects, with rapid and significant dose-proportional increases in plasma arginine, the key driver of NO production.


  • In November 2021, Eloxx Pharmaceuticals announced positive topline results from the monotherapy arms of its Phase 2 clinical trial of ELX-02 in Class 1 cystic fibrosis (CF) patients with at least one G542X nonsense allele mutation. ELX-02 was well tolerated and achieved a statistically significant 5.4mmol/L reduction in sweat chloride in patients at the1.5mg/kg/day dose.


  • In October 2021, Boehringer Ingelheim, IP Group, the UK Cystic Fibrosis Gene Therapy Consortium (GTC, consisting of researchers from Imperial College London and the Universities of Oxford and Edinburgh) and Oxford Biomedica (OXB), today that Boehringer Ingelheim has exercised its options on intellectual property and know-how from the partners to progress and further accelerate the development of a potential, new treatment option for patients with CF. In the partnership, IP Group, acting on behalf of the three GTC host Universities, is granting exclusive global rights to develop, manufacture, register, and commercialize this lentiviral vector-based gene therapy for the treatment of cystic fibrosis. The GTC is additionally contributing its knowledge in pre-clinical research and clinical gene therapy development. OXB is adding its leading competence in manufacturing lentiviral vector-based therapies to Boehringer Ingelheim’s expertise in the development of novel breakthrough therapies for respiratory diseases.


  • In August 2021, Sanofi entered into a definitive agreement with Translate Bio (NASDAQ: TBIO), a clinical-stage mRNA therapeutics company, under which Sanofi will acquire all outstanding shares of Translate Bio for $38.00 per share in cash, which represents a total equity value of approximately $3.2 billion (on a fully diluted basis). The Sanofi and Translate Bio Boards of Directors unanimously approved the transaction.On the therapeutic side, Translate Bio has an early-stage pipeline in cystic fibrosis and other rare pulmonary diseases. In addition, discovery work is ongoing in diseases that affect the liver, and Translate Bio’s MRTTM platform may be applied to various classes of treatments, such as therapeutic antibodies or vaccines in areas such as oncology. Sanofi’s recent acquisition of Tidal Therapeutics expanded the company’s mRNA research capabilities in both immuno-oncology and inflammatory diseases. The Translate Bio acquisition further accelerates Sanofi’s efforts to develop transformative medicines using mRNA technology.


  • In August 2019, Path BioAnalytics Inc. (PBA) announced it had licensed rights to cavosonstat from Laurel Therapeutics. Cavosonstat is a novel CFTR modulator designed to correct a subset of CFTR mutations by increasing stability of the CFTR protein in the cell membrane through inhibition of S-nitrosoglutathione reductase (GSNOR) and preservation of S-nitrosoglutathione (GSNO).


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


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 Segmentation



  • Late stage products (Phase III)
  • Mid-stage products (Phase II)
  • Early-stage product (Phase I) along with the details of
  • Pre-clinical and Discovery stage candidates
  • Discontinued & Inactive candidates
  • Route of Administration


Route of Administration

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


Molecule Type

  • Oligonucleotide
  • Peptide
  • Small molecule
  • Product Type


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


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


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


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