Primary Ciliary Dyskinesia Clinical and Non-Clinical Studies, Key Companies, Therapeutic Assessment, Emerging Therapies, Treatment Algorithm, and Pipeline Analysis | Parion Sciences, ReCode Therapeutics, Translate Bio, Santhera Pharmaceuticals, ethris Gmb

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 a typically inherited autosomal recessive disorder that affects the tiny, hair-like structures called cilia in the respiratory system, impairing their function. This dysfunction hinders the proper clearance of mucus from the lungs, paranasal sinuses, and middle ears, leading to recurrent respiratory infections due to trapped bacteria and irritants. Symptoms often manifest soon after birth and may include persistent coughing, choking, gagging, and neonatal respiratory distress, such as lung atelectasis. Individuals with PCD frequently suffer from chronic lung, sinus, and middle ear infections, along with excessive mucus production, chronic coughing, and hearing loss.

Diagnosis is confirmed through a biopsy of lung or sinus tissue or genetic testing, with structural abnormalities in the cilia being identifiable under an electron microscope. Treatment focuses on preserving lung health through airway clearance therapy, which may involve regular sinus and ear canal cleaning. Additional management strategies include the use of antibiotics, bronchodilators, steroids, and mucolytic medications to help alleviate 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

DelveInsight's Primary Ciliary Dyskinesia pipeline report depicts a robust space with 4+ active players working to develop 4+ pipeline therapies for Primary Ciliary Dyskinesia treatment.
Key Primary Ciliary Dyskinesia companies such as Parion Sciences, ReCode Therapeutics, Translate Bio, Santhera Pharmaceuticals, ethris GmbH, and others are evaluating new drugs for Primary Ciliary Dyskinesia to improve the treatment landscape.
Promising Primary Ciliary Dyskinesia pipeline therapies in various stages of development include P 1037, and others.

Recent breakthroughs in the Primary Ciliary Dyskinesia Pipeline Segment:

A notable advancement is ReCode Therapeutics' investigational therapy, RCT1100. In June 2024, the FDA granted Orphan Drug Designation to RCT1100, recognizing its potential to address this unmet medical need. RCT1100 is an inhaled mRNA therapy designed to deliver DNAI1 mRNA to airway cells, aiming to restore proper ciliary function by addressing mutations in the DNAI1 gene, which are responsible for approximately 7% of PCD cases. Preclinical studies have demonstrated that RCT1100 facilitates the production of the DNAI1 protein and improves ciliary function in relevant models. ReCode is currently enrolling patients in a Phase 1 open-label study to assess the safety and tolerability of a single ascending dose of inhaled RCT1100 in individuals with PCD caused by pathogenic mutations in the DNAI1 gene. ​

Primary Ciliary Dyskinesia Pipeline Analysis
The report provides insights into:
The report provides detailed insights into the key companies that are developing therapies in the Primary Ciliary Dyskinesia Market.
The report also evaluates different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Primary Ciliary Dyskinesia treatment.
It analyzes the key companies involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
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.
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.

Download our free sample page report on Primary Ciliary Dyskinesia pipeline insights @ https://www.delveinsight.com/sample-request/primary-ciliary-dyskinesia-pcd-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

Primary Ciliary Dyskinesia Emerging Drugs

P 1037: Parion Sciences

Primary Ciliary Dyskinesia Companies

Around four or more key companies are actively engaged in developing therapies for Primary Ciliary Dyskinesia. Among them, Parion Sciences has the most advanced drug candidates, currently in Phase II of clinical development.
DelveInsight's report covers around 4+ 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

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

Intravenous
Subcutaneous
Oral
Intramuscular

Primary Ciliary Dyskinesia Products have been categorized under various Molecule types such as

Monoclonal antibody
Small molecule
Peptide

Download Sample Pages to Get an in-depth Assessment of the Emerging Primary Ciliary Dyskinesia Therapies and Key Companies: Primary Ciliary Dyskinesia Clinical Trials and advancements @ https://www.delveinsight.com/report-store/primary-ciliary-dyskinesia-pcd-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

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

Download Primary Ciliary Dyskinesia Sample report to know in detail about the Primary Ciliary Dyskinesia treatment market @ Primary Ciliary Dyskinesia Therapeutic Assessment @ https://www.delveinsight.com/sample-request/primary-ciliary-dyskinesia-pcd-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

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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Contact Info:
Kritika Rehani
krehani@delveinsight.com
info@delveinsight.com
+14699457679

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.

This release was published on openPR.