Primary Ciliary Dyskinesia Pipeline constitutes 4+ key companies continuously working towards developing 4+ Primary Ciliary Dyskinesia treatment therapies, analyzes DelveInsight.
Primary Ciliary Dyskinesia Overview:
Primary ciliary dyskinesia (PCD) is an inherited autosomal recessive disorder that affects the function of tiny, hair-like structures called cilia in the respiratory tract. Dysfunctional cilia impair the clearance of mucus from the lungs, paranasal sinuses, and middle ears, resulting in recurrent respiratory infections as bacteria and irritants become trapped. Symptoms often appear shortly after birth and can include persistent coughing, choking, gagging, and neonatal respiratory distress such as lung atelectasis. People with PCD commonly experience chronic infections in the lungs, sinuses, and middle ears, along with excessive mucus production, persistent coughing, and hearing loss.
Diagnosis is typically confirmed through a lung or sinus biopsy or genetic testing, with ciliary structural abnormalities detectable via electron microscopy. Treatment aims to maintain lung function through airway clearance therapies, including regular cleaning of the sinuses and ear canals. Additional management approaches may involve antibiotics, bronchodilators, steroids, and mucolytic agents to relieve symptoms and prevent complications.
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"Primary Ciliary Dyskinesia Pipeline Insight 2025" report by DelveInsight provides a comprehensive analysis of the ongoing clinical development activities and growth prospects across the Primary Ciliary Dyskinesia Therapeutics Market.
Key Takeaways from the Primary Ciliary Dyskinesia Pipeline Report
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DelveInsight’s Primary Ciliary Dyskinesia (PCD) pipeline report highlights an active landscape, with over four companies developing more than four investigational therapies for PCD treatment.
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Key players in this space include Parion Sciences, ReCode Therapeutics, Translate Bio, Santhera Pharmaceuticals, ethris GmbH, and others, all working to advance new treatment options and improve the therapeutic landscape. Promising pipeline candidates in various stages of development include P 1037, among others.
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A notable development is ReCode Therapeutics’ investigational therapy, RCT1100. In June 2024, the FDA granted Orphan Drug Designation to RCT1100, recognizing its potential to address an unmet medical need. RCT1100 is an inhaled mRNA therapy designed to deliver DNAI1 mRNA to airway cells, aiming to restore normal ciliary function by correcting mutations in the DNAI1 gene, which account for roughly 7% of PCD cases. Preclinical studies have shown that RCT1100 promotes DNAI1 protein production and improves ciliary function in relevant models. ReCode is currently conducting a Phase 1 open-label trial to evaluate the safety and tolerability of single ascending doses of inhaled RCT1100 in patients with PCD caused by pathogenic DNAI1 mutations.
Primary Ciliary Dyskinesia Pipeline Analysis
The report provides insights into:
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The report provides detailed insights into the key companies that are developing therapies in the Primary Ciliary Dyskinesia Market.
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The report also evaluates different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Primary Ciliary Dyskinesia treatment.
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It analyzes the key companies involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
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It navigates the emerging drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
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Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement, and financing details for future advancement of the Primary Ciliary Dyskinesia market.
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Primary Ciliary Dyskinesia Emerging Drugs
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P 1037: Parion Sciences
Primary Ciliary Dyskinesia Companies
Over four leading companies are actively working on therapies for Primary Ciliary Dyskinesia, with Parion Sciences having the most advanced candidates currently in Phase II clinical trials.
DelveInsight’s report covers around 4+ products under different phases of clinical development like
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Late stage products (Phase III)
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Mid-stage products (Phase II)
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Early-stage product (Phase I) along with the details of
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Pre-clinical and Discovery stage candidates
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Discontinued & Inactive candidates
Primary Ciliary Dyskinesia pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
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Intravenous
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Subcutaneous
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Oral
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Intramuscular
Primary Ciliary Dyskinesia Products have been categorized under various Molecule types such as
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Monoclonal antibody
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Small molecule
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Peptide
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Primary Ciliary Dyskinesia Pipeline Therapeutic Assessment
• Primary Ciliary Dyskinesia Assessment by Product Type
• Primary Ciliary Dyskinesia By Stage
• Primary Ciliary Dyskinesia Assessment by Route of Administration
• Primary Ciliary Dyskinesia Assessment by Molecule Type
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Table of Content
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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About DelveInsight
DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.
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