Hemophilia Gene Therapy - A Pipeline Analysis Report
- Published: Oct 2017
- Pages: 105
- SKU: IRTNTR15465
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This pipeline analysis report identifies uniQure, Alnylam Pharmaceuticals, BioMarin Pharmaceutical, Spark Therapeutics, Shire, and Sangamo Therapeutics as some of the companies that are actively involved in the development of gene therapy molecules for hemophilia patients. This report presents a comprehensive analysis of the therapeutic assessment by therapy (monotherapy and combination therapy), by ROA (intravenous, subcutaneous, and unspecified), and by stage and ROA (phase III, phase I/II, phase I and IND, and pre-clinical). This pipeline analysis report also provides a detailed analysis of hemophilia gene therapy based on indications such as hemophilia A and hemophilia B.
Overview of the pipeline analysis report on hemophilia gene therapy
Hemophilia is an ideal genetic disorder and the drawbacks with the majority of the standard protein substitution therapy drugs resulted in the shift in interest towards the development of alternative treatments by gene therapy. Though the risk of developing antibodies to the viral vectors that are used to deliver the genetic material to the cells poses as a major drawback to gene therapy, the rising commercial importance of the gene therapy approach for the treatment of hemophilia resulted in a rise in the number of collaboration agreements between industries and research institutes. For instance, the acquisition of Baxalta helped the specialty pharmaceutical company become the largest hemophilia player and the company has announced that it has generated a revenue of more than USD 870 million in 2016.
According to Technavio’s pipeline analysis report on hemophilia gene therapy, the pipeline molecules in hemophilia gene therapy are currently in the pre-clinical development stage. SHP648, a high-performing next-generation FIX gene therapy candidate from one of the major Tier I companies Shire, is currently in the pre-clinical stage and the development of this therapeutic candidate is based on the AAV8-based gene therapy technology.
Key companies
Technavio’s analysts have identified thirteen companies that are actively involved in the development of gene therapy for hemophilia patients. Based on the companies’ revenue, this report has segregated the players into Tier 1, Tier 2, and Tier 3 companies. One of the major Tier 1 companies Alnylam Pharmaceuticals, is a leading player in the biotechnology industry and has registered an income of more than USD 410 million.
The Tier 1 companies covered in this report are –
- uniQure
- Alnylam Pharmaceuticals
- BioMarin Pharmaceutical
- Spark Therapeutics
- Shire
Segmentation of hemophilia gene therapy molecules by indication
- Hemophilia A
- Hemophilia B
It has been observed that the prevalence of hemophilia A is more in the US and the countries in EU5 such as France, Germany, Italy, Spain, and United Kingdom. According to this pipeline analysis report, most of the companies that are involved in the development of gene therapy for hemophilia patients are currently focusing on developing drugs for hemophilia A.
Therapeutic assessment of the hemophilia gene therapy molecules based on therapy
- Monotherapy
- Combination therapy
Monotherapy is the utilization of a single drug to treat a disease. Our research analysts have identified that all the hemophilia pipeline drugs that are currently in the pipeline are being developed only as a monotherapy agent and most of these molecules are still in the pre-clinical phase.
Key questions answered in the report include
- What are the different stages of the pipeline molecules in investigation?
- What are the molecules in the various development stages in the hemophilia gene therapy pipeline?
- What are the key companies that are currently involved in the development of gene therapy drugs for hemophilia?
- What are the molecules discontinued from the hemophilia gene therapy pipeline?
- What are the major regulatory authorities approving drugs in various regions?
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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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