As Primary Ciliary Dyskinesia (PCD) increasingly impacts populations worldwide and contributes to comorbidities such as diabetes, cardiovascular disease, and certain cancers, the demand for safer and more effective treatments is growing. According to DelveInsight, the PCD pipeline includes over four pharmaceutical and biotech companies actively developing more than four therapeutic candidates. These therapies are at various stages of clinical and preclinical development, reflecting substantial innovation and dedication to tackling this significant public health challenge.
DelveInsight’s “Primary Ciliary Dyskinesia Pipeline Insight 2025” report offers a comprehensive and strategic assessment of the current R&D landscape. It provides insights into clinical trial progress, emerging therapies, mechanisms of action, competitive positioning, and key initiatives by leading companies such as Parion Sciences, ReCode Therapeutics, Translate Bio, Santhera Pharmaceuticals, and ethris GmbH. The report serves as an essential resource for researchers, healthcare investors, and decision-makers seeking to understand the evolving PCD therapeutics market and the breakthroughs shaping its future.
Explore the Cutting-Edge Landscape of Primary Ciliary Dyskinesia Drug Development
Key Takeaways from the Primary Ciliary Dyskinesia Pipeline Report
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DelveInsight’s report on the Primary Ciliary Dyskinesia (PCD) pipeline highlights a dynamic landscape, with over four companies actively developing more than four therapeutic candidates for PCD treatment.
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In June 2024, the FDA granted Orphan Drug Designation to RCT1100, an inhaled mRNA therapy from ReCode Therapeutics, specifically targeting PCD caused by DNAI1 gene mutations.
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Leading companies in the PCD space—including Parion Sciences, ReCode Therapeutics, Translate Bio, Santhera Pharmaceuticals, and ethris GmbH—are actively investigating new drugs to enhance the treatment landscape. Promising pipeline candidates, such as P 1037 and others, are progressing through various stages of development.
Primary Ciliary Dyskinesia Overview:
Primary ciliary dyskinesia (PCD) is a rare genetic disorder, usually inherited in an autosomal recessive pattern, in which the tiny hair-like structures called cilia—responsible for moving mucus through the respiratory tract—do not function properly. Impaired ciliary activity prevents efficient clearance of mucus from the lungs, sinuses, and middle ears, allowing bacteria and other irritants to accumulate and cause recurrent respiratory infections. Symptoms often appear shortly after birth and may include coughing, gagging, choking, and lung collapse (atelectasis) due to respiratory distress. Over time, individuals with PCD frequently develop chronic infections in the lungs, sinuses, and ears, along with persistent coughing, excessive mucus production, and hearing loss.
Definitive diagnosis is typically achieved through genetic testing or microscopic examination of tissue samples from the lungs or sinuses, which can reveal structural abnormalities in the cilia using electron microscopy. Management focuses on maintaining lung function through airway clearance therapies, including routine sinus and ear irrigation. Treatment also involves antibiotics to fight infections, bronchodilators to ease breathing, corticosteroids to reduce inflammation, and mucolytics to thin and clear mucus.
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Primary Ciliary Dyskinesia Pipeline Analysis
The Primary Ciliary Dyskinesia pipeline insights report 2025, provides insights into:
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Provides comprehensive insights into key companies developing therapies in the Primary Ciliary Dyskinesia Market.
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Categorizes Primary Ciliary Dyskinesia therapeutic companies by development stage: early, mid, and late-stage.
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Highlights major companies involved in targeted therapy development, including both active and inactive (paused/discontinued) projects.
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Reviews emerging Primary Ciliary Dyskinesia drugs under development based on:
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Stage of development
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Primary Ciliary Dyskinesia Route of administration
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Target receptor
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Monotherapy vs. combination therapy
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Primary Ciliary Dyskinesia Mechanism of action
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Molecular type
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Offers detailed analysis of:
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Company-to-company and company-academia collaborations
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Primary Ciliary Dyskinesia Licensing agreements
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Funding and investment activities supporting future Primary Ciliary Dyskinesia market advancement.
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Unlock key insights into emerging Primary Ciliary Dyskinesia therapies and market strategies here: https://www.delveinsight.com/report-store/primary-ciliary-dyskinesia-pcd-pipeline-insight?utm_source=abnewswire&utm_medium=market&utm_campaign=kpr
Primary Ciliary Dyskinesia Emerging Drugs
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P 1037: Parion Sciences
Parion Sciences is developing P-1037, a novel inhibitor of the epithelial sodium channel (ENaC) designed to treat respiratory diseases such as primary ciliary dyskinesia (PCD), cystic fibrosis, and chronic obstructive pulmonary disease (COPD), all characterized by thick, dehydrated airway mucus. By blocking sodium channels on airway surfaces, ENaC inhibitors like P-1037 help rehydrate mucus, restore mucociliary clearance, and improve lung function.
Primary Ciliary Dyskinesia Pipeline Therapeutic Assessment
Primary Ciliary Dyskinesia Assessment by Product Type
• Mono
• Combination
• Mono/Combination
Primary Ciliary Dyskinesia By Stage
• 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
Primary Ciliary Dyskinesia Assessment by Route of Administration
• Oral
• Parenteral
• Intravenous
• Subcutaneous
• Topical
Primary Ciliary Dyskinesia Assessment by Molecule Type
• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy
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Table of Contents
1. Report Introduction
2. Executive Summary
3. Primary Ciliary Dyskinesia Current Treatment Patterns
4. Primary Ciliary Dyskinesia – DelveInsight’s Analytical Perspective
5. Therapeutic Assessment
6. Primary Ciliary Dyskinesia Late-Stage Products (Phase-III)
7. Primary Ciliary Dyskinesia Mid-Stage Products (Phase-II)
8. Early Stage Products (Phase-I)
9. Pre-clinical Products and Discovery Stage Products
10. Inactive Products
11. Dormant Products
12. Primary Ciliary Dyskinesia Discontinued Products
13. Primary Ciliary Dyskinesia Product Profiles
14. Primary Ciliary Dyskinesia Key Companies
15. Primary Ciliary Dyskinesia Key Products
16. Dormant and Discontinued Products
17. Primary Ciliary Dyskinesia Unmet Needs
18. Primary Ciliary Dyskinesia Future Perspectives
19. Primary Ciliary Dyskinesia Analyst Review
20. Appendix
21. Report Methodology
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