Cystic fibrosis- Pipeline Insight, 2025

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 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.

Geography Covered:

• Global coverage

Cystic fibrosis: Understanding

Cystic fibrosis: Overview

Cystic fibrosis (CF) is a recessive genetic disease caused by a mutation in the epithelial chloride channel-cystic fibrosis transmembrane conductance regulator (CFTR). CF is a predominant genetic disorder with a disease severity ranging from mild to life-threatening. The number of CF patients in a population varies depending on ethnicity. 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.

People with CF can have a variety of symptoms, including: Very salty-tasting skin, persistent coughing, at times with phlegm, frequent lung infections including pneumonia or bronchitis, wheezing or shortness of breath, poor growth or weight gain in spite of a good appetite, frequent greasy, bulky stools or difficulty with bowel movements, nasal polyps , chronic sinus infections and many more. Cystic fibrosis is a complex disease. The types of symptoms and how severe they are can differ widely from person to person.

Cystic fibrosis (CF) is a genetic disorder that affects the lungs, pancreas, and other organs by causing thick, sticky mucus buildup, leading to respiratory and digestive issues. It is caused by mutations in the CFTR gene, which disrupts chloride ion transport. Treatment focuses on managing symptoms and improving quality of life, including airway clearance techniques, inhaled medications (mucolytics, bronchodilators), pancreatic enzyme supplements, and CFTR modulators like Trikafta (elexacaftor/tezacaftor/ivacaftor). Advances in gene therapy are being explored for future cures.

In most cases, CF is diagnosed during childhood. Doctors diagnose CF with a thorough evaluation and by using different tests. These include:

Sweat Test: The sweat test measures the amount of chloride in the body's sweat, which is higher in people who have CF. In the test, your healthcare provider spreads a chemical called pilocarpine on your skin, then applies a small amount of electric stimulation to encourage the sweat glands to produce sweat. Your provider then collects the sweat in a plastic coil or on a piece of filter paper or gauze. People of any age can have a sweat test. It's not painful and does not use a needle. This is the most conclusive test for CF.

Genetic tests: Blood samples are tested for the genes that cause CF.

Sinus X-rays: As with chest X-rays, sinus X-rays can confirm CF in people who show certain symptoms. Other forms of testing are used along with sinus X-rays.

Sputum culture: The healthcare provider takes a sample of the patients sputum (spit) and tests it for bacteria. Certain bacteria, such as Pseudomonas, are most commonly found in people who have CF.

There is no cure for cystic fibrosis. The major focus of management is keeping airways clear. The patient can use devices that fit into their mouth or therapy vests that rely on vibrations to loosen mucus. People with cystic fibrosis have nutritional needs that aren't the same as the needs of people without CF. People with CF may need 1.5 to 2 times the number of calories as people without CF. You need the extra calories if you have CF because you use more energy than other people to breathe, fight lung infections and maintain your strength. Another common misbelief is that salt (sodium) is unhealthy for all people. This isn't true for children and adults with CF. People with CF lose a lot of salt in their sweat. Although there's not a set standard, healthcare providers generally tell people with CF to eat salty foods. This is true especially during hot, humid weather and exercise.

"Cystic fibrosis- Pipeline Insight, 2025" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Cystic fibrosis pipeline landscape is provided which includes the disease overview and Cystic fibrosis treatment guidelines. The assessment part of the report embraces, in depth Cystic fibrosis commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, 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.

Report Highlights:

• The companies and academics are working to assess challenges and seek opportunities that could influence Cystic fibrosis R&D. The therapies under development are focused on novel approaches to treat/improve Cystic fibrosis.

Cystic fibrosis Emerging Drugs Chapters

This segment of the Cystic fibrosis report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, II/III I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Cystic fibrosis Emerging Drugs

• 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(R)). 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.

Cystic fibrosis: Therapeutic Assessment

This segment of the report provides insights about the different Cystic fibrosis drugs segregated based on following parameters that define the scope of the report, such as:

• Major Players in Cystic fibrosis

There are approx. 50+ 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, Armata Pharmaceuticals.

• Phases

DelveInsight's report covers around 55+ products under different phases of clinical development like

• 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

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
• Molecule Type

Products have been categorized under various Molecule types such as

• Monoclonal antibody
• Small molecule
• Peptide
• Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Cystic fibrosis: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Cystic fibrosis therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Cystic fibrosis drugs.

Cystic fibrosis Report Insights

• Cystic fibrosis Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Cystic fibrosis Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions:

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Cystic fibrosis drugs?
• How many Cystic fibrosis drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Cystic fibrosis?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Cystic fibrosis therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Cystic fibrosis and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Verona Pharma
• Armata Pharmaceuticals
• 4D Molecular Therapeutics
• Spirovant Sciences
• Vertex Pharmaceuticals
• Krystal Biotech
• Porosome Therapeutics
• SpliSense
• Enterprise Therapeutics Ltd

Key Products

• Ensifentrine
• AP-PA02
• 4D-710
• SP-101
• VX-522
• KB407
• CF PT201
• SPL23
• VX-828
• SPL84
• ETD001