ファンコニ貧血(FA)のパイプラインインサイト(~2029)

◆英語タイトル:Fanconi Anemia (FA) - Pipeline Insight, 2019
◆商品コード:DATA00109032
◆発行会社(リサーチ会社):DelveInsight
◆発行日:2019年12月
◆ページ数:60
◆レポート言語:英語
◆レポート形式:PDF
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【レポートの概要】

Fanconi Anemia (FA) – Pipeline Insight, 2019 report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Fanconi Anemia pipeline landscape is provided which includes the disease overview and Fanconi Anemia treatment guidelines. The assessment part of the report embraces, in depth Fanconi Anemia 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, Fanconi Anemia collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Fanconi Anemia Understanding
According to the Cancer and Blood Disorders Center, Fanconi Anemia (Fanconi hypoplastic anemia, Fanconi pancytopenia, Fanconi panmyelopathy) is a rare inherited disease characterized by multiple physical abnormalities, bone marrow failure, and a higher than normal risk of cancer. Researchers have shown that mutations in one of at least 15 different genes can cause FA. The proteins normally produced by these genes form a kind of cellular “machine” that helps detect and repair damaged DNA in blood stem cells and other cells in the body, in FA this damaged DNA repair is slowed. Therefore, blood stem cells (in the bone marrow) accumulate damaged DNA and do not survive. FA is usually discovered between birth and age 10-15 years; however, there also have been cases identified in adulthood. FA occurs equally in males and females. It has been identified in all ethnic groups. Researchers continue to clone and characterize the genes responsible for FA, which is bringing considerable progress in the diagnosis and understanding of this disease. It is more common in male as compared to female.

Fanconi Anemia Pipeline Development Activities
The report provides insights into different therapeutic candidates in discovery and preclinical, phase 1, phase 2, and phase 3 stage. Drugs under development as a monotherapy or combination therapy are also included. It also analyses key players involved in FA targeted therapeutics development with respective active and dormant or discontinued projects. FA pipeline report covers 7+ companies. Some of the key players include Rocket pharma (RP-L102), Bellicum Pharmaceuticals (BPX-501), etc.

The report is built using data and information traced from the researcher’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university web sites and industry-specific third party sources, etc.

Fanconi Anemia Analytical Perspective by DelveInsight
• In-depth Fanconi Anemia Commercial Assessment of products
This report provides an in-depth Commercial Assessment of therapeutic drugs have been included which comprises of collaborations, Licensing, Acquisition –Deal Value Trends. The sub-segmentation is described in the report which includes Company-Company Collaborations (Licensing / Partnering), Company-Academia Collaborations, and Acquisition analysis in both Graphical and tabulated form.
• Fanconi Anemia Clinical Assessment of products
The report comprises of comparative clinical assessment of products by development stage, product type, route of administration, molecule type, and MOA type across this indication.

Scope of the report
• The Fanconi Anemia report provides an overview of therapeutic pipeline activity for Fanconi Anemia across the complete product development cycle including all clinical and non-clinical stages
• It comprises of detailed profiles of Fanconi Anemia therapeutic products with key coverage of developmental activities including technology, collaborations, licensing, mergers and acquisition, funding, designations and other product related details
• Detailed Fanconi Anemia Research and Development progress and trial details, results wherever available, are also included in the pipeline study
• Therapeutic assessment of the active pipeline products by development stage, product type, route of administration, molecule type, and MOA type
• Coverage of dormant and discontinued pipeline projects along with the reasons if available across Fanconi Anemia

Reasons to Buy
• Establish a comprehensive understanding of the current pipeline scenario across Fanconi Anemia to formulate effective R&D strategies
• Assess challenges and opportunities that influence Fanconi Anemia R&D
• Develop strategic initiatives by understanding the focus areas of leading companies.
• Gather impartial perspective of strategies of the emerging competitors having potentially lucrative portfolio in this space and create effective counter strategies to gain competitive advantage
• Get in detail information of each product with updated information on each project along with key milestones
• Devise Fanconi Anemia in licensing and out licensing strategies by identifying prospective partners with progressing projects for Fanconi Anemia to enhance and expand business potential and scope
• Our extensive domain knowledge on therapy areas support the clients in decision-making process regarding their therapeutic portfolio by identifying the reason behind the inactive or discontinued drugs*Please Note:* The report will be delivered in 2 business days upon order confirmation.

【レポートの目次】

1. Report Introduction
2. Fanconi Anemia (Fanconi hypoplastic anemia, Fanconi pancytopenia, Fanconi panmyelopathy)
2.1. Fanconi Anemia Disease Overview
2.2. Fanconi Anemia History
2.3. Fanconi Anemia Symptoms
2.4. Fanconi Anemia Causes
2.5. Fanconi Anemia Pathophysiology
2.6. Fanconi Anemia Diagnosis
2.6.1. Diagnostic Guidelines
3. Fanconi Anemia Current Treatment Patterns
3.1. Treatment Guidelines
4. Fanconi Anemia – DelveInsight’s Analytical Perspective
4.1. In-depth Commercial Assessment
4.1.1. Fanconi Anemia companies collaborations, Licensing, Acquisition –Deal Value Trends
4.1.1.1. Assessment Summary
4.1.2. Fanconi Anemia Collaboration Deals
4.1.2.1. Company-Company Collaborations (Licensing / Partnering) Analysis
4.1.2.2. Fanconi Anemia Acquisition Analysis
4.2. Clinical Assessment of Pipeline Drugs
4.2.1. Assessment by Phase of Development
4.2.2. Assessment by Product Type (Mono / Combination)
4.2.2.1. Assessment by Stage and Product Type
4.2.3. Assessment by Route of Administration
4.2.3.1. Assessment by Stage and Route of Administration
4.2.4. Assessment by Molecule Type
4.2.4.1. Assessment by Stage and Molecule Type
4.2.5. Assessment by MOA
4.2.5.1. Assessment by Stage and MOA
5. Fanconi Anemia Pipeline Therapeutics
5.1. Late Stage Products (Phase-III)
5.1.1. Comparative Analysis
5.2. Mid Stage Products (Phase-II)
5.2.1. Comparative Analysis
5.3. Early Stage Products (Phase-I)
5.3.1. Comparative Analysis
5.4. Pre-clinical and Discovery Stage Products
5.4.1. Comparative Analysis
5.5. Inactive Products
6. Fanconi Anemia – Products Analysis
6.1. Product Profiles
6.1.1. RP-L102- Rocket pharma
6.1.1.1. Product Description
6.1.1.1.1. Product Overview
6.1.1.1.2. Mechanism of Action
6.1.1.2. Research and Development
6.1.1.2.1. Clinical Studies
6.1.1.2.1.1. Detailed Study Description
6.1.1.2.1.2. Study Results
6.1.1.2.1.3. Clinical Trials: Tabular View
6.1.1.3. Product Development Activities
6.1.1.3.1. Tabulated Product Summary
6.1.1.3.1.1. General Description Table
6.1.2. BPX-501- Bellicum Pharmaceuticals
6.1.2.1. Product Description
6.1.2.1.1. Product Overview
6.1.2.1.2. Mechanism of Action
6.1.2.2. Research and Development
6.1.2.2.1. Clinical Studies
6.1.2.2.1.1. Detailed Study Description
6.1.2.2.1.2. Study Results
6.1.2.2.1.3. Clinical Trials: Tabular View
6.1.2.3. Product Development Activities
6.1.2.3.1. Tabulated Product Summary
6.1.2.3.1.1. General Description Table
To be continued in the report
7. Recent Technologies
8. Fanconi Anemia Key Companies
8.1. Abeona Therapeutics
8.2. Rocket Pharmaceuticals
8.3. CIEMAT
8.4. BioLineRx
8.5. Foresee Pharmaceuticals
8.6. Novartis
8.7. Bellicum Pharmaceuticals
9. Fanconi Anemia Key Products
9.1. Research programme: rare haematological disorder gene therapies
9.2. Research programme: gene therapies
9.3. RP L101
9.4. RP L102
9.5. Motixafortide
9.6. FP 045
9.7. Eltrombopag
9.8. BPX-501 T cells
10. Dormant and Discontinued Products
10.1. Dormant Products
10.1.1. Reasons for being dormant
10.2. Discontinued Products
10.2.1. Reasons for the discontinuation
11. Fanconi Anemia – Unmet Needs
12. Fanconi Anemia – Future Perspectives
13. Appendix
14. Report Methodology
14.1. Secondary Research
14.2. Expert Panel Validation
Table 1. Diagnostic Guidelines
Table 2. Treatment Guidelines
Table 3. Assessment Summary
Table 4. Company-Company Collaborations (Licensing / Partnering) Analysis
Table 5. Fanconi Anemia Acquisition Analysis
Table 6. Assessment by Phase of Development
Table 7. Assessment by Product Type (Mono / Combination)
Table 8. Assessment by Stage and Product Type
Table 9. Assessment by Route of Administration
Table 10. Assessment by Stage and Route of Administration
Table 11. Assessment by Molecule Type
Table 12. Assessment by Stage and Molecule Type
Table 13. Assessment by MOA
Table 14. Assessment by Stage and MOA
Table 15. Late Stage Products (Phase-III)
Table 16. Mid Stage Products (Phase-II)
Table 17. Early Stage Products (Phase-I)
Table 18. Pre-clinical and Discovery Stage Products
Table 19. Inactive Products
Table 20. Dormant Products
Table 21. Discontinued Products
Figure 1. Disease Overview
Figure 2. History
Figure 3. Symptoms
Figure 4. Causes
Figure 5. Pathophysiology
Figure 6. Diagnostic Guidelines
Figure 7. Treatment Guidelines
Figure 8. Fanconi Anemia companies collaborations, Licensing, Acquisition –Deal Value Trends
Figure 9. Company-Company Collaborations (Licensing / Partnering) Analysis
Figure 10. Fanconi Anemia Acquisition Analysis
Figure 11. Assessment by Phase of Development
Figure 12. Assessment by Product Type (Mono / Combination)
Figure 13. Assessment by Stage and Product Type
Figure 14. Assessment by Route of Administration
Figure 15. Assessment by Stage and Route of Administration
Figure 16. Assessment by Molecule Type
Figure 17. Assessment by Stage and Molecule Type
Figure 18. Assessment by MOA
Figure 19. Assessment by Stage and MOA
Figure 20. Late Stage Products (Phase-III)
Figure 21. Mid Stage Products (Phase-II)
Figure 22. Early Stage Products (Phase-I)
Figure 23. Pre-clinical and Discovery Stage Products
Figure 24. Inactive Products
Figure 25. Dormant Products
Figure 26. Discontinued Products
Figure 27. Unmet Needs



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