Primary ciliary dyskinesia - Pipeline Insight, 2021

Delveinsight
60 Pages - DELVE15426
$1,500.00

DelveInsight’s, “Primary ciliary dyskinesia - Pipeline Insight, 2021,” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Primary ciliary dyskinesia 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

Primary ciliary dyskinesia Understanding
Primary ciliary dyskinesia: Overview
Primary ciliary dyskinesia (PCD) is usually an autosomal recessive genetic condition in which the microscopic organelles (cilia) in the respiratory system have defective function. Ciliary dysfunction prevents the clearance of mucous from the lungs, paranasal sinuses and middle ears. Bacteria and other irritants in the mucous lead to frequent respiratory infections. Kartagener syndrome is a type of PCD associated with a mirror-image orientation of the heart and other internal organs (situs inversus). Rare cases of X-linked and autosomal dominant inheritance have been observed.

"Primary ciliary dyskinesia - Pipeline Insight, 2021" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Primary ciliary dyskinesia pipeline landscape is provided which includes the disease overview and Primary ciliary dyskinesia treatment guidelines. The assessment part of the report embraces, in depth Primary ciliary dyskinesia 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, Primary ciliary dyskinesia 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 Primary ciliary dyskinesia R&D. The therapies under development are focused on novel approaches to treat/improve Primary ciliary dyskinesia.
Primary ciliary dyskinesia Emerging Drugs Chapters
This segment of the Primary ciliary dyskinesia report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, 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.

Primary ciliary dyskinesia Emerging Drugs
• Lonodelestat: Santhera Pharmaceuticals
Lonodelestat (previously known as POL6014), a highly potent and selective peptide inhibitor of human neutrophil elastase (hNE), is in development for the treatment of cystic fibrosis. Neutrophil elastase is an enzyme associated with tissue inflammation, leading to degradation of the lung tissue in cystic fibrosis and several other pulmonary diseases. Lonodelestat has EU orphan drug designations (ODD) for the treatment of CF as well as for AATD and PCD in both EU and US. Santhera obtained the worldwide, exclusive rights from Polyphor AG to develop and commercialize lonodelestat in CF and other diseases.
Further product details are provided in the report……..

Primary ciliary dyskinesia: Therapeutic Assessment
This segment of the report provides insights about the different Primary ciliary dyskinesia drugs segregated based on following parameters that define the scope of the report, such as:
• Major Players in Primary ciliary dyskinesia
There are approx. 5+ key companies which are developing the therapies for Primary ciliary dyskinesia. The companies which have their Primary ciliary dyskinesia drug candidates in the most advanced stage, i.e. Phase I/II include, Santhera Pharmaceuticals.
• Phases

DelveInsight’s report covers around 5+ 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

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
• Oral
• Parenteral
• Intravitreal
• Subretinal
• Topical

• Molecule Type
Products have been categorized under various Molecule types such as
• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Gene therapy

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

Primary ciliary dyskinesia: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase III, II, I, preclinical and discovery stage. It also analyses Primary ciliary dyskinesia 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 Primary ciliary dyskinesia drugs.

Primary ciliary dyskinesia Report Insights
• Primary ciliary dyskinesia Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs
Primary ciliary dyskinesia 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 Primary ciliary dyskinesia drugs?
• How many Primary ciliary dyskinesia drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Primary ciliary dyskinesia?
• What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Primary ciliary dyskinesia 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 Primary ciliary dyskinesia and their status?
• What are the key designations that have been granted to the emerging drugs?

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Introduction
Executive Summary
Primary ciliary dyskinesia: Overview
• Causes
• Mechanism of Action
• Signs and Symptoms
• Diagnosis
• Disease Management
Pipeline Therapeutics
• Comparative Analysis
Therapeutic Assessment
• Assessment by Product Type
• Assessment by Stage and Product Type
• Assessment by Route of Administration
• Assessment by Stage and Route of Administration
• Assessment by Molecule Type
• Assessment by Stage and Molecule Type
Primary ciliary dyskinesia – DelveInsight’s Analytical Perspective
In-depth Commercial Assessment
• Primary ciliary dyskinesia companies’ collaborations, Licensing, Acquisition -Deal Value Trends
Primary ciliary dyskinesia Collaboration Deals
• Company-Company Collaborations (Licensing / Partnering) Analysis
• Company-University Collaborations (Licensing / Partnering) Analysis
Early Stage Products (Phase I/II)
• Comparative Analysis
Lonodelestat: Santhera Pharmaceuticals
• Product Description
• Research and Development
• Product Development Activities
Drug profiles in the detailed report…..
Preclinical Stage Products
• Comparative Analysis
Research programme: RNA therapeutics: ReCode Therapeutics
• Product Description
• Research and Development
• Product Development Activities
Drug profiles in the detailed report…..
Discovery Stage Products
• Comparative Analysis
Research programme: mRNA therapeutics: Translate Bio
• Product Description
• Research and Development
• Product Development Activities
Drug profiles in the detailed report…..
Inactive Products
• Comparative Analysis
Primary ciliary dyskinesia Key Companies
Primary ciliary dyskinesia Key Products
Primary ciliary dyskinesia- Unmet Needs
Primary ciliary dyskinesia- Market Drivers and Barriers
Primary ciliary dyskinesia- Future Perspectives and Conclusion
Primary ciliary dyskinesia Analyst Views
Primary ciliary dyskinesia Key Companies
Appendix

Table 1 Total Products for Primary ciliary dyskinesia
Table 2 Late Stage Products
Table 3 Mid Stage Products
Table 4 Early Stage Products
Table 5 Pre-clinical & Discovery Stage Products
Table 6 Assessment by Product Type
Table 7 Assessment by Stage and Product Type
Table 8 Assessment by Route of Administration
Table 9 Assessment by Stage and Route of Administration
Table 10 Assessment by Molecule Type
Table 11 Assessment by Stage and Molecule Type
Table 12 Inactive Products

Figure 1 Total Products for Primary ciliary dyskinesia
Figure 2 Late Stage Products
Figure 3 Mid Stage Products
Figure 4 Early Stage Products
Figure 5 Preclinical and Discovery Stage Products
Figure 6 Assessment by Product Type
Figure 7 Assessment by Stage and Product Type
Figure 8 Assessment by Route of Administration
Figure 9 Assessment by Stage and Route of Administration
Figure 10 Assessment by Molecule Type
Figure 11 Assessment by Stage and Molecule Type
Figure 12 Inactive Products

• Translate Bio
• ReCode Therapeutics
• Santhera Pharmaceuticals

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