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.
- Alnylam Pharmaceuticals
- BioMarin Pharmaceutical
- Spark Therapeutics
- 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?
PART 02: RESEARCH METHODOLOGY
PART 03: HEMOPHILIA GENE THERAPY: AN INSIGHT
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 08: THERAPEUTIC ASSESSMENT BY THERAPY
- Therapeutic assessment based on therapy
- Therapeutic assessment based on RoA
- Therapeutic assessment based on RoA in various stages of development
- Active companies: Category and parameters
- List of abbreviations
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
The author of the report recognizes the following companies in the hemophilia gene therapy: uniQure, Alnylam Pharmaceuticals, BioMarin Pharmaceutical, Spark Therapeutics, and Shire.
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. Due to the deficiency of these factors, the blood does not clot properly, and this condition creates complicated health issues.
Using a gene with the capacity to cure or prevent the progression of a disease is the basic concept of gene therapy technique. Different approaches are being tested by the researchers, including replacing a mutated gene that causes disease with a healthy copy of the gene, inactivating or knocking out a mutated gene that is malfunctioning, and introducing a new gene into the body to help fight a disease.
The use of gene therapy for the treatment of hemophilia is in many ways a promising option. Hemophilia is an ideal genetic disorder to treat by gene therapy, as it occurs due to a single genetic defect. The drawbacks associated with the majority of the marketed drugs (standard protein substitution therapy) intensified the interest in alternative treatments by gene therapy. However, gene therapy also has some drawbacks such as the risk of developing antibodies to the viral vectors used to deliver the genetic material to the cells in the individuals and inconsistent results in clinical trials with human test remains a matter of concern for the researchers.
The study was conducted using an objective combination of primary and secondary information including inputs from key participants in the industry.