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Global Hemophilia Gene Therapy Market Size, Status and Forecast 2025

Global Hemophilia Gene Therapy Market Size, Status and Forecast 2025

Hemophilia is a genetic disorder that impairs blood clotting. When one of the genes that encode for blood clotting factors is absent or deficient, the smallest injury can trigger serious bleeding episodes. Since these genes are located on the X chromosome, the disease is much more common in males than females.

About 80% of them have hemophilia A, which affects the clotting factor VIII. The second most common form, hemophilia B, is due to a deficiency of the clotting factor IX. Several biotechs are racing to launch the first gene therapy for hemophilia. Currently, uniQure in the Netherlands and Spark Therapeutics in the US have the most advanced programs. Spark scored a victory in December when it presented Phase I/II for its candidate SPK-9001. The gene therapy was able to reduce annual bleeding episodes by 97%, as compared to its competitor uniQure¡¯s candidate, AMT-060. However, uniQure has fought back. The company added a modification in its gene therapy that is known to increase clotting activity by 8- to 9- fold. This improved version has already been cleared to start a Phase III clinical trial in both Europe and the US.

Hemophilia B has traditionally been the main focus of biotechs, since most big pharma efforts were focused on the bigger hemophilia A market. In addition, applying gene therapy to hemophilia A is more challenging; The gene coding for the factor IX protein missing in hemophilia B is simply smaller than that for factor VIII missing in hemophilia A, and therefore easier to fit in the viral vectors used for gene delivery. US-based BioMarin is leading the development of a gene therapy. To overcome the size limit, the company has deleted a region from the factor VIII protein that is not necessary for clotting.

Hemophilia treatment is currently in the pre-clinical stage. And the multiple treatments that are underway might significantly improve the quality of life of patients with hemophilia, by getting rid of frequent infusions and hospital visits, and transitioning patients from severe to mild hemophilia.

The major players in global Hemophilia Gene Therapy market include
Spark Therapeutics
Ultragenyx
Shire PLC
Sangamo Therapeutics
Bioverativ
BioMarin
uniQure
Freeline Therapeutics

On the basis on the end users/applications, this report covers
Hemophilia A Gene Therapy
Hemophilia B Gene Therapy


1 Hemophilia Market Overview
1.1 Overview Hemophilia
1.2 Hemophilia Types
1.2.1 Hemophilia A
1.2.2 Hemophilia B
1.3 Causes of Hemophilia
1.4 Global Hemophilia Distribution
1.4.1 Global Hemophilia Distribution
1.4.2 Severity of Hemophilia
1.5 A Brief History of Hemophilia Treatment
1.6 Hemophilia Gene Therapy Companies
2 Gene Therapy
2.1 Gene Therapy Introduction
2.2 How does Gene Therapy work
2.3 Safety Protection
2.4 Development
3 Regulations of Gene Therapies
3.1 USA Regulations
3.1.1 U.S. Biologic Products Development Process
3.1.2 Human Clinical Trials under an IND
3.1.3 Additional Regulation for Gene Therapy Clinical Trials
3.1.4 Compliance with cGMP Requirements
3.1.5 U.S. Review and Approval Processes
3.1.6 Orphan Drug Designation
3.1.7 Expedited Development and Review Programs
3.1.8 Post-approval Requirements
3.1.9 U.S. Patent Term Restoration and Marketing Exclusivity
3.2 European Union Regulation and Exclusivity
3.3 Other Healthcare Laws and Regulations
4 Spark Therapeutics
4.1 Company Profile
4.2 Products Introduction
4.2.1 SPK-8011
4.2.2 SPK-9001
4.3 Development Plan
5 Ultragenyx
5.1 Company Profile
5.2 Products Introduction
5.2.1 DTX201
5.2.2 DTX101
5.3 Development Plan
5.4 Ultragenyx Acquire Dimension Therapeutics (Oct. 2017)
6 Shire PLC
6.1 Company Profile
6.2 Products Introduction
6.2.1 SHP654£¨BAX 888£©
6.3 Development Plan
6.4 Shire Acquire Baxalta (Jun. 2016)
7 Sangamo Therapeutics
7.1 Company Profile
7.2 Products Introduction
7.2.1 SB-525 ¨C Hemophilia A
7.2.2 SB-FIX ¨C Hemophilia B
7.3 Research and Development Expenses
7.4 Collaboration with Pfizer
8 Bioverativ
8.1 Company Profile
8.2 Products Introduction
8.3 Collaboration with San Raffaele-TIGET
8.4 Acquired by Sanofi
9 UniQure
9.1 Company Profile
9.2 Product Information
9.2.1 Hemophilia B Gene Therapy
9.2.2 Hemophilia A Gene Therapy
9.3 Hemophilia B Gene Therapy Study Type
10 Freeline Therapeutics
10.1 Company Profile
10.2 Product Information
10.3 Freeline Therapeutics Timeline
11 BioMarin
11.1 Company Profile
11.2 Product Information
11.3 BioMarin Provides 2 Years of Clinical Data
12 Research Findings and Conclusion
13 Methodology and Data Source
13.1 Methodology/Research Approach
13.1.1 Research Programs/Design
13.1.2 Market Size Estimation
13.1.3 Market Breakdown and Data Triangulation
13.2 Data Source
13.2.1 Secondary Sources
13.2.2 Primary Sources
13.3 Disclaimer
13.4 Author List
List of Tables and Figures
Figure Impaired Blood Clotting in Hemophilia
Table Severity of Hemophilia
Figure Hemophilia Types Share
Figure Causes of Hemophilia
Figure Global Hemophilia Distribution in 2016
Table Demographics of Hemophilia
Figure Severity of Hemophilia A-Males
Figure Severity of Hemophilia B-Males
Figure Severity of Hemophilia A-Females
Figure Severity of Hemophilia B-Females
Table Hemophilia A Gene Therapy Companies
Table Hemophilia B Gene Therapy Companies
Figure Picture of Gene Therapy
Figure Gene Therapy Process
Table Spark Therapeutics Company Profile
Figure Product Pipeline
Table Research and Development Expense of SPK-8011 and SPK-9001
Figure Preliminary SPK©\8011 Phase 1/2 data in hemophilia B
Figure Preliminary SPK©\9001 Phase 1/2 data in hemophilia B
Figure Spark Therapeutics Revenue from Collaboration Agreements
Table Ultragenyx Company Profile
Table Shire PLC Company Profile
Figure Product Pipeline
Table Sangamo Therapeutics Company Profile
Figure Product Pipeline of Hemophilia
Table Sangamo Therapeutics Research and Development Expenses of Hemophilia Gene Therapy
Table Bioverativ Company Profile
Figure Product Pipeline of Hemophilia
Table Timeline of Bioverativ
Table UniQure Company Profile
Table UniQure Hemophilia B Gene Therapy Development Timeline
Figure UniQure Hemophilia B Gene Therapy Milestones in 2018
Table UniQure Pivotal Study Program
Table UniQure Goal of Hemophilia B Gene Therapy
Table Freeline Therapeutics Company Profile
Table Freeline Therapeutics Timeline
Table BioMarin Company Profile
Table BioMarin Hemophilia A Gene Therapy Development

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