Thalassemia - A Pipeline Analysis Report
- Published: Jul 2018
- Pages: 80
- SKU: IRTNTR22730
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This pipeline analysis report provides detailed insights into the drug development landscape of the drug development for Thalassemia including molecules at preclinical, discovery, and other developmental stages. The report also offers comprehensive information about the therapeutic assessment of the pipeline molecules based on various segmentation such as indications coverage, therapies employed, route of administration, therapeutic modality, targets for drug development, and mechanism of action.
Overview of the drug development pipeline for Thalassemia
Thalassemia is an inherited disorder that can be inherited from one of the parents or both the parents. The two types of Thalassemia include alphas-thalassemia and beta-thalassemia. Patients suffering from this condition experience anemia, which is caused by excessive destruction of red blood cells. Fatigue, abdominal swelling, dark urine, pale skin, weakness are some of the symptoms that are experienced by patients suffering from Thalassemia. Technavio’s market research analysts have predicted that with the introduction of regular blood transfusion, gene transference, and cell replacement as some of the most promising approaches to treat Thalassemia, the global Thalassemia market will witness growth in the forthcoming years.
According to this pipeline analysis report, most of the drug development molecules in the pipeline are under the pre-clinical developmental stage. For instance, sponsors such as Aevi Genomic Medicine developed TARGTEPO and Agios developed AG-348, which are under pre-clinical development stage. The pipeline landscape in the pipeline analysis report mentions the percentage of drug development molecules that are under phase I, phase I/II, and phase II stages. Our market research analysts have also identified that a small amount of drug molecules that are being developed comes under phase III and discovery developmental stages.
Companies covered
This pipeline analysis report provides a detailed analysis of the companies that are involved in the development of drug molecules for the treatment of Thalassemia. In addition to providing information on the various stages of molecules developed by companies for different indications, this pipeline analysis report also provides information about the drug development molecules discontinued by companies.
Some of the companies covered in this pipeline analysis report are –
- Celgene
- Invenux
- Rare Partners
Therapeutic assessment of the drug development pipeline for Thalassemia by route of administration
- Oral
- Subcutaneous
- Intravenous
- Intraosseous
The oral route of administration (ROA) involves the administration of drug substances through the mouth cavity. It has been observed that the majority of total therapeutics are being developed for oral administration.
Therapeutic assessment of the drug development pipeline for Thalassemia by therapeutic modality
- Gene therapy
- Small molecule
- Protein
- Biological
- Plant derived
According to this pipeline analysis report, the majority of molecules that are currently in the drug development pipeline for Thalassemia are being developed as small molecules. Small molecules are chemically manufactured active substances that can enter cells easily because of their low molecular weight.
Key questions answered in the report include
- What are the drug development molecules in the various development stages for Thalassemia?
- What are the companies that are currently involved in the development of drug development molecules for Thalassemia?
- Insight into discontinued/inactive molecules with appropriate reasoning?
- What are the major regulatory authorities approving drugs in various regions?
- Detailed profiling of each active molecule
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PART 01: EXECUTIVE SUMMARY
PART 02: SCOPE OF THE REPORT
PART 03: RESEARCH METHODOLOGY
PART 04: INTRODUCTION
PART 05: MAJOR REGULATORY AUTHORITIES
- US
- Europe
- China
PART 06: PIPELINE LANDSCAPE
PART 07: COMPARATIVE ANALYSIS
- Discovery stage molecules
- Pre-clinical stage molecules
- Inactive and discontinued molecules
PART 08: INDICATION ANALYSIS
PART 09: THERAPEUTIC ASSESSMENT (THERAPY BASED)
PART 10: THERAPEUTIC ASSESSMENT (ROA BASED)
PART 11: THERAPEUTIC ASSESSMENT BY TARGET
PART 12: KEY COMPANIES
- Active companies: Category and parameters
PART 13: APPENDIX
- List of abbreviations
Research Framework
Technavio presents a detailed picture of the market by way of study, synthesis, and summation of data from multiple sources. The analysts have presented the various facets of the market with a particular focus on identifying the key industry influencers. The data thus presented is comprehensive, reliable, and the result of extensive research, both primary and secondary.
INFORMATION SOURCES
Primary sources
- Manufacturers and suppliers
- Channel partners
- Industry experts
- Strategic decision makers
Secondary sources
- Industry journals and periodicals
- Government data
- Financial reports of key industry players
- Historical data
- Press releases

DATA ANALYSIS
Data Synthesis
- Collation of data
- Estimation of key figures
- Analysis of derived insights
Data Validation
- Triangulation with data models
- Reference against proprietary databases
- Corroboration with industry experts

REPORT WRITING
Qualitative
- Market drivers
- Market challenges
- Market trends
- Five forces analysis
Quantitative
- Market size and forecast
- Market segmentation
- Geographical insights
- Competitive landscape
Key Questions Answered
- What are the key global market and the regional market share?
- What are the revenue-generating key market segments?
- What are the key factors driving and challenging this market’s growth?
- Who are the key market vendors and their growth strategies?
- What are the latest trends influencing the growth of this market?
- What are the variables influencing the market growth in the primary regions?
- What are the factors influencing the growth of the parent market?
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