Gene Therapy for Sickle-Cell Diseases - A Pipeline Analysis Report
- Published: Jul 2018
- Pages: 67
- SKU: IRTNTR23387
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This pipeline analysis report provides detailed insights into the clinical trials landscape of the gene therapy for sickle-cell diseases including molecules at pre-clinical and discovery stage. The report also offers comprehensive information about the therapeutic assessment of the pipeline molecules based on various segmentations such as therapy, route of administration, therapeutic modalities, and target.
Overview of the gene therapy for sickle-cell diseases
Sickle-cell diseases are a group of blood disorders caused by an abnormality in the hemoglobin in red blood cells. Sickle-cell diseases are mostly inherited from parents. The patients inherit one copy of the mutated hemoglobin gene from each parent in the chromosome 11. The people with one abnormal copy of the hemoglobin gene are called carriers, and they do not show the traits of the disease or develop the anemia. Sickle-cell diseases can lead to severe complications such as stroke, chronic pain, abortion, pulmonary hypertension, and osteomyelitis. The diseases are race- and geography-specific indications. The disease is more common in the Sub-Saharan regions, India, and the Middle-Eastern countries compared with other parts of the world. According to the National Library of Medicine, a part of the National Institutes of Health, sickle-cell diseases are more prevalent in African-Americans than in the Hispanic Americans, with a ratio of 3:1. The ratio of males to females with sickle-cell diseases is 1:1. As a result, with the rising incidences of sickle-cell diseases, the gene therapy for sickle-cell diseases is expected to increase considerably during the next few years.
According to this pipeline analysis report, most of the gene therapy molecules in the pipeline are being developed for sickle-cell diseases. Our market research analysts have also identified that most of these molecules are in the pre-clinical development stage and a considerable number of molecules have been discontinued from development.
Companies covered
This pipeline analysis report provides a detailed analysis of the companies that are involved in the development of gene therapy molecules for the treatment of sickle-cell diseases. 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 gene therapy molecules discontinued by companies.
Some of the companies covered in this pipeline analysis report are:
- Angiocrine Bioscience
- Calimmune
- Editas Medicine
- Genethon
- MaxCyte
Therapeutic assessment of the gene therapy for sickle-cell diseases by route of administration
- Intravenous
The intravenous route of administration (ROA) involves the application of the drug directly through the veins, which will have a more direct effect on the target cells.
Therapeutic assessment of the gene therapy for sickle-cell diseases by therapy
- Monotherapy
According to this pipeline analysis report, all the molecules that are currently in the gene therapy pipeline for sickle-cell diseases are being developed as monotherapy drugs and most of these molecules are in the pre-clinical stage of development.
Key questions answered in the report include
- What are the gene therapy molecules in the various development stages for sickle-cell diseases?
- What are the companies that are currently involved in the development of gene therapy molecules for sickle-cell diseases?
- 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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