Hemophilia Therapeutics Pipeline Analysis, 2017 - Clinical Trials & Results, Patent, Designation, Collaboration, and Other Developments

  • ID: 4340298
  • Report
  • 116 pages
  • P&S Market Research
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Disease Overview

Hemophilia is a genetic disorder characterized by excessive bleeding and delayed blood clotting. There are two types of hemophilia, hemophilia A (also known as classic hemophilia) and hemophilia B. According to the Centers for Disease Control and Prevention, the incidence of hemophilia stands at, one out of every 5,000 male births and as many as 20,000 males in the U.S., living with either of the two types of hemophilia, presently. The prevalence of hemophilia A is around four times high as compared to the prevalence of hemophilia B. It has been observed that the male population is more commonly affected by the disease, as compared to the female population, since hemophilia is inherited in an X-linked recessive genetic pattern, where females are generally carriers of the disease. Hemophilia A results from a deficiency of clotting Factor VIII, whereas hemophilia B (also known as the Christmas disease) is caused by a deficiency of clotting Factor IX, both of these are essential for the proper functioning of blood clotting. The cause of the deficiency of clotting factors is thought to be a mutation in one of the genes that codes for the synthesis of these clotting factor, which are required to form a blood clot.

Pipeline Analysis

Significant growth is expected in the therapeutic pipeline of hemophilia, mainly attributed to increasing collaboration between pharma as well as biotech companies, educational institutes and associations for the development of hemophilia therapeutics. Increasing prevalence of hemophilia and technological advancement are some of the major factors for the development of novel therapies for treatment of hemophilia. Positive clinical results of Phase III and Phase II drug candidates with lesser side effects are supporting the development of hemophilia therapeutics. Many awareness programs organized by pharma as well as biotechnology companies are supporting the development of hemophilia therapeutics pipeline. PolyXen is an enabling platform technology for protein drug delivery. Zinc finger nuclease mediated in vivo genome editing approach makes use of the endogenous albumin gene locus, a highly expressing and liver-specific site that can be edited with ZFNs to accept and express therapeutic genes. As of June 2017, the hemophilia pipeline comprises of approximately 53 drug candidates in the pipeline for the treatment of hemophilia in different stages of development.

Competitive Landscape

Some of the other key players developing drugs for the treatment of hemophilia include, Sangamo BioSciences, Inc., Caisson Biotech, Inc., XL-protein GmbH and others.

Scope for Customization

Customization is offered as per specific business requirements of clients. Illustrative customization within the scope of this report includes:
  • Market Forecast - Market analysis and forecast for the drug candidates in the latest stage of development
  • Company Profiles - Wider company coverage in terms of detailed analysis or additional company profiles
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1. Research Background
1.1 Research Objectives
1.2 Definition
1.3 Research Scope
1.4 Key Stakeholders

2. Research Methodology
2.1 Secondary Research
2.2 Primary Research

3. Executive Summary

4. Pipeline Outlook
4.1 Overview
4.2 Key Drivers
4.3 Key Barriers
4.4 Hemophilia Pipeline Analysis
4.4.1 Pipeline Analysis By Phase
4.4.2 Pipeline Analysis By Molecule Type
4.4.3 Pipeline Analysis By Route Of Administration
4.4.4 Pipeline Analysis By Company

5. Hemophilia Therapeutics Pipeline Analysis By Phase (2017)
5.1 Filed: Drug Profiles
5.1.1 Pre-Clinical Study
5.1.2 Pre-Clinical Results
5.1.3 Clinical Trials
5.1.4 Clinical Results
5.1.5 Strategic Development
5.1.6 Designation
5.1.7 Grants
5.1.8 Patent
5.1.9 Technology
5.2 Phase Iii: Drug Profiles
5.2.1 Pre-Clinical Study
5.2.2 Pre-Clinical Results
5.2.3 Clinical Trials
5.2.4 Clinical Results
5.2.5 Strategic Development
5.2.6 Designation
5.2.7 Grants
5.2.8 Patent
5.2.9 Technology
5.3 Phase Ii: Drug Profiles
5.3.1 Pre-Clinical Study
5.3.2 Pre-Clinical Results
5.3.3 Clinical Trials
5.3.4 Clinical Results
5.3.5 Strategic Development
5.3.6 Designation
5.3.7 Grants
5.3.8 Patent
5.3.9 Technology
5.4 Phase I: Drug Profiles
5.4.1 Pre-Clinical Study
5.4.2 Pre-Clinical Results
5.4.3 Clinical Trials
5.4.4 Clinical Results
5.4.5 Strategic Development
5.4.6 Designation
5.4.7 Grants
5.4.8 Patent
5.4.9 Technology
5.5 Pre-Clinical: Drug Candidates
5.6 Discovery: Drug Candidates

6 Competitive Landscape
6.1 Key Players Benchmarking For Hemophilia Drug Pipeline
6.2 Swot Analysis Of Hemophilia Pipeline

7 Company Profiles
7.1 Business Overview
7.2 Product And Service Offerings

8 Appendix
8.1 Abbreviations

List Of Tables
Pipeline Analysis Of Hemophilia, By Company (2017)
Description Of Filed Drug Candidates
Clinical Trials Of Filed Drug Candidates
Description Of Phase Iii Drug Candidates
Clinical Trials Of Phase Iii Drug Candidates
Description Of Phase Ii Drug Candidates
Clinical Trials Of Phase Ii Drug Candidates
Description Of Phase I Drug Candidates
Clinical Trials Of Phase I Drug Candidates
Pre-Clinical Drug Candidates
Discovery Drug Candidates
Companies- At A Glance

List Of Figures
Research Methodology For The Hemophilia Pipeline Analysis
Split Of Primary And Secondary Research
Breakdown Of Primary Research Respondents, By Industry Participant
Breakdown Of Primary Research Respondents, By Company Type
Role Of Clotting Fcators In Coagualtion
Number Of Hemophilia Drug Candidates Under Development (2017)
Hemophilia Pipeline Split, By Molecule Type (2017)
Hemophilia Pipeline Split, By Route Of Administration (2017)
Key Players Benchmarking For Hemophilia Drug Pipeline
Swot Analysis Of Hemophilia Pipeline
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