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Hemochromatosis/Iron Overload Therapeutics - Pipeline Analysis 2018, Clinical Trials & Results, Patents, Designations, Collaborations, and Other Developments

  • ID: 4517552
  • Report
  • 67 pages
  • Prescient & Strategic Intelligence Private Limited
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Hemochromatosis or iron overload refer to the accumulation of excessive iron in the body. Iron chelation therapy is needed to remove this excess iron from the body. Hemochromatosis may be caused due to genetic disorders or acquired by multiple blood transfusions or consuming high levels of supplemental iron. High levels of iron in the body may damage the vital organs in the body.

Insights on Pipeline Segments

According to the research findings, a majority of the drug candidates for the treatment of hemochromatosis are being developed to be administered by the subcutaneous route, which facilitates the absorption of the drug as iron chelator drugs such as deferoxamine, that are poorly absorbed from the gastrointestinal tract.

Hemochromatosis Therapeutics Pipeline is Driven by the Availability of the Limited Drug Options

As of now, there are three marketed iron chelator drugs available for the treatment of hemochromatosis namely; Deferasirox, Deferoxamine and Deferiprone. Limited competition gives companies investing in the hemochromatosis therapeutics pipeline an opportunity to gain market share. Thus, driving the growth of the hemochromatosis therapeutics pipeline.

Hemochromatosis Therapeutics Pipeline Involves Development of Gene Suppressor Therapy

Hemochromatosis most of the times develops as a hereditary disease. Therefore, it has been observed that the companies are developing drug candidates that suppresses the expression of genes. For instance, ALN-TMP by Alnylam Pharmaceuticals Inc. is under the Pre-Clinical phase of development for the treatment of hemochromatosis. The drug candidate is a Tmprss6 gene suppressor being developed using RNA interference (RNAi) technology.

Some of the key players involved in the development of hemochromatosis therapeutics include La Jolla Pharmaceutical Company, Silence Therapeutics plc, and Shire Plc.
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1. Research Background
1.1 Research Objectives
1.2 Definition
1.3 Research Scope
1.3.1 Pipeline Analysis by Phase
1.3.2 Pipeline Analysis by Molecule Type
1.3.3 Pipeline Analysis by Route of Administration
1.4 Key Stakeholders

2. Research Methodology
2.1 Secondary Research
2.2 Primary Research
2.2.1 Breakdown of Primary Research Respondents by industry participant by company type

3. Executive Summary

4. Pipeline Outlook
4.1 Disease Overview
4.2 Classification of Hemochromatosis
4.3 Symptoms
4.4 Diagnosis
4.5 Causes of Hemochromatosis
4.6 Treatment
4.7 Epidemiology
4.8 Key Drivers
4.8.1 Limited Number of Drugs
4.8.2 Side Effects of the Current Treatments
4.8.3 Cost of Current Treatments
4.9 Key Barriers
4.9.1 Underdiagnoses of the disease
4.10 Hemochromatosis Therapeutics Pipeline Analysis
4.10.1 Pipeline Analysis by Phase
4.10.2 Pipeline Analysis by Molecule Type
4.10.3 Pipeline Analysis by Route of Administration
4.10.4 Pipeline Analysis by Company

5. Hemochromatosis Therapeutics Pipeline Analysis by Phase (2018)
5.1 Phase II
5.1.1 XX Clinical Trials
5.2 Phase I
5.2.1 K-DFX Patents Technology
5.2.2 XX Clinical trials Patents Drug designation
5.2.3 XX Clinical Trials Patents Technology
5.2.4 XX Clinical Trials
5.3 Pre-Clinical
5.3.1 VIT 2763
5.3.2 XX Pre-clinical results Technology
5.3.3 XX Patents Technology
5.3.4 XX Patents

6. Clinical Trials Review
6.1 Clinical Trials by Region
6.2 Clinical Trials by Trial Phase

7. Competitive Landscape
7.1 Key Players Benchmarking for Hemochromatosis Therapeutics Pipeline
7.2 SWOT Analysis of Hemochromatosis Therapeutics Pipeline
7.2.1 Strengths
7.2.2 Weaknesses
7.2.3 Opportunities
7.2.4 Threats

8. Company Profiles
8.1 Ionis Pharmaceuticals Inc.
8.1.1 Business Overview
8.1.2 Product Offerings
8.2 Company 2
8.2.1 Business Overview
8.2.2 Product Offerings
8.3 Company 3
8.3.1 Business Overview
8.3.2 Product Offerings
8.4 Company 4
8.4.1 Business Overview
8.4.2 Product Offerings
8.5 Company 5
8.5.1 Business Overview
8.5.2 Product Offerings
8.6 Company 6
8.6.1 Business Overview
8.6.2 Product Offerings
8.7 Company 7
8.7.1 Business Overview
8.7.2 Product Offerings

9. Appendix
9.1 Abbreviations
9.2 Related Reports

List of Tables
Table 1 Pipeline Analysis of Hemochromatosis Therapeutics, by Company (2018)
Table 2 Description of Xx
Table 3 Clinical Trials of Xx
Table 4 Description of K-Dfx
Table 5 Description of Xx
Table 6 Clinical Trials of Xx
Table 7 Description of Xx
Table 8 Clinical Trial of Xx
Table 9 Description of Xx
Table 10 Clinical Trial of Xx
Table 11 Description of Vit 2763
Table 12 Description of Xx
Table 13 Description of Xx
Table 14 Patents by Xx
Table 15 Description of Xx
Table 16 Ionis Pharmaceuticals, Inc. - At a Glance
Table 17 Company 2 - At a Glance
Table 18 Company 3 - At a Glance
Table 19 Company 4 - At a Glance
Table 20 Company 5 - At a Glance
Table 21 Company 6 - At a Glance
Table 22 Company 7 - At a Glance

List of Figures
Fig 1: Research Methodology
Fig 2: Breakdown of Primary Research Respondents, by Industry Participant
Fig 3: Breakdown of Primary Research Respondents, by Company Type
Fig 4: Hemochromatosis Drug Candidates In Development (2018)
Fig 5: Hemochromatosis Pipeline Split, by Molecule Type (2018)
Fig 6: Hemochromatosis Pipeline Split, by Route of Administration (2018)
Fig 7: Breakdown of Clinical Trials, by Region
Fig 8: Breakdown of Clinical Trials, by Trial Phase
Fig 9: Key Players Benchmarking
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