Hemophilia Gene Therapy: An insight
Hemophilia is an inherited bleeding disorder. The gene responsible for hemophilia is present on the X chromosome and is inherited in a recessive manner. Hemophilia A, also called as factor VIII (FVIII) deficiency or classic hemophilia, is caused by missing or defective factor VIII. Most individuals, roughly 85%, have hemophilia A and produce too little or no factor VIII. On the other hand, hemophilia B, which is also called Christmas disease, is caused by a deficiency of factor IX.
Shire became the biggest hemophilia player after it bought Baxalta in June 2016, and the company reported total hemophilia revenue of $870.9 million in the FY2016.
Covered in this report
The report covers the present scenario and the growth prospects of the hemophilia gene therapy. The report presents a detailed picture of the market by way of study, synthesis, and summation of data from multiple sources.
Technavio's Hemophilia Gene Therapy - A Pipeline Analysis Report, has been prepared based on an in-depth market analysis with inputs from industry experts. The report covers the market landscape and its growth prospects during the forecast period. The report also includes a discussion of the key companies operating in this market.
Major companies
• uniQure
• Alnylam Pharmaceuticals
• BioMarin Pharmaceutical
• Spark Therapeutics
• Shire
Key questions answered in this report
• How will the market evolve during the forecast period?
• What are the major parameters impacting the market?
• What are the key market trends?
• What are the challenges to market growth?
• Who are the key companies in this market space?
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Table of Contents
PART 01: EXECUTIVE SUMMARY
PART 02: RESEARCH METHODOLOGY
PART 03: HEMOPHILIA GENE THERAPY: AN INSIGHT
• Introduction
PART 04: MAJOR REGULATORY AUTHORITIES
• US
• Europe
• Japan
• Australia
• China
PART 05: PIPELINE LANDSCAPE
PART 06: COMPARATIVE ANALYSIS
• Late-stage molecules (Phase III)
• Mid-stage molecules (Phase I/II)
• Early-stage molecules (Phase I and IND)
• Pre-clinical stage molecules
• Discontinued molecules
PART 07: INDICATION ANALYSIS
PART 08: THERAPEUTIC ASSESSMENT BY THERAPY
• Therapeutic assessment based on therapy
PART 09: THERAPEUTIC ASSESSMENT BY ROA
• Therapeutic assessment based on RoA
PART 10: THERAPEUTIC ASSESSMENT BY STAGE AND ROA
• Therapeutic assessment based on RoA in various stages of development
PART 11: KEY COMPANIES
• Active companies: Category and parameters
PART 12: APPENDIX
• List of abbreviations
List of Exhibits
Exhibit 01: Drug approval process by US FDA
Exhibit 02: Pipeline landscape
Exhibit 03: Pipeline molecules by vendors
Exhibit 04: Pipeline molecules in different developmental stages 2017
Exhibit 05: Pipeline molecules in different developmental stages (%) 2017
Exhibit 06: Overview: Fitusiran
Exhibit 07: Clinical trials description of Fitusiran
Exhibit 08: Overview: SB?525
Exhibit 09: Clinical trial description of SB-525
Exhibit 10: Overview: SB?FIX
Exhibit 11: Clinical trials description of SB?FIX
Exhibit 12: Overview: AMT?060
Exhibit 13: Clinical trials description of AMT-060 (AAV5-hFIX)
Exhibit 14: Overview: BMN 270
Exhibit 15: Clinical trials description of BMN 270
Exhibit 16: Overview: SPK?9001
Exhibit 17: Clinical trials description of SPK?9001
Exhibit 18: Overview: SPK-8011
Exhibit 19: Clinical trials description of SPK-8011
Exhibit 20: Overview: Gene therapy for hemophilia B
Exhibit 21: Clinical trials description of gene therapy for hemophilia B
Exhibit 22: Overview: DTX201
Exhibit 23: Overview: SHP654
Exhibit 24: Overview: SVFVIIa
Exhibit 25: Overview: Ex-vivo stem cell-LV-FVIII gene therapy
Exhibit 26: Overview: In-vivo AAV-FIX gene therapy
Exhibit 27: Overview: Research program for hemophilia A
Exhibit 28: Overview: Gene therapy for hemophilia
Exhibit 29: Overview: Factor VIII (rhFVIII)
Exhibit 30: Overview: Gene therapy for hemophilia A
Exhibit 31: Overview: SHP648
Exhibit 32: Overview: HepaStem
Exhibit 33: Discontinued hemophilia gene therapy pipeline molecules, 2017
Exhibit 34: Segmentation of hemophilia gene therapy pipeline molecules by indication 2017
Exhibit 35: An overview of hemophilia indication
Exhibit 36: Assessment of hemophilia gene therapy pipeline molecules by monotherapy products
Exhibit 37: Assessment of hemophilia gene therapy pipeline molecules by RoA
Exhibit 38: Assessment of hemophilia gene therapy pipeline molecules by stage and RoA
Exhibit 39: Active companies: Category and parameters 2017
Exhibit 40: Segmentation of companies 2017
Exhibit 41: Overview: Alnylam Pharmaceuticals 2017
Exhibit 42: Overview: Amarna Therapeutics 2017
Exhibit 43: Overview: BioMarin Pharmaceutical 2017
Exhibit 44: Overview: Bioverativ 2017
Exhibit 45: Overview: Dimension Therapeutics 2017
Exhibit 46: Overview: Expression Therapeutics 2017
Exhibit 47: Overview: Freeline Therapeutics 2017
Exhibit 48: Overview: Immusoft 2017
Exhibit 49: Overview: Pharming Group 2017
Exhibit 50: Overview: Promethera 2017
Exhibit 51: Overview: Sangamo Therapeutics 2017
Exhibit 52: Overview: Shire 2017
Exhibit 53: Overview: Spark Therapeutics 2017
Exhibit 54: Overview: uniQure 2017
