Chapter 1. Research Background
1.1 Research Objectives
1.2 Definition
1.3 Research Scope
1.3.1 Pipeline analysis by phase
1.3.2 Pipeline analysis by route of administration
1.3.3 Pipeline analysis by molecule type
1.4 Key Stakeholders
Chapter 2. Research Methodology
2.1 Secondary Research
2.2 Primary Research
2.2.1 Breakdown of primary research respondents
2.2.1.1 By industry participant
2.2.1.2 By company type
Chapter 3. Executive Summary
Chapter 4. Pipeline Outlook
4.1 Overview
4.2 Key Drivers
4.2.1 Novel therapies in hemophilia pipeline
4.2.2 Technical advancements
4.3 Key Barriers
4.3.1 Lack of awareness about medication for the treatment of hemophilia
4.3.2 Less number of treatment options
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
Chapter 5. Hemophilia Pipeline Analysis by Phase (2023)
5.1 Filed
5.1.1 LR-769
5.1.1.1 Clinical trials
5.1.1.2 Clinical trial results
5.1.1.3 Strategic development
5.1.1.4 Technology
5.2 Phase III
5.2.1 Emicizumab
5.2.1.1 Clinical trials
5.2.1.2 Clinical trial results
5.2.1.3 Strategic development
5.2.1.4 Designation
5.2.1.5 Technology
5.2.2 NN7088
5.2.2.1 Clinical trials
5.2.2.2 Clinical trial results
5.2.2.3 Strategic development
5.2.2.4 Technology
5.2.3 Damoctocog alfa pegol
5.2.3.1 Clinical trials
5.3 Phase II
5.3.1 Fitusiran
5.3.1.1 Clinical trials
5.3.1.2 Clinical trial results
5.3.1.3 Strategic development
5.3.2 PF-06838435
5.3.2.1 Clinical trials
5.3.2.2 Strategic development
5.3.2.3 Designation
5.3.2.4 Technology
5.3.3 CSL689 rVIIa-FP
5.3.3.1 Pre-Clinical studies
5.3.3.2 Clinical trials
5.3.3.3 Clinical trial results
5.3.3.4 Designation
5.3.4 PF-06741086
5.3.4.1 Clinical trials
5.3.4.2 Clinical trial results
5.3.4.3 Designation
5.4 Phase I
5.4.1 SHP656
5.4.1.1 Clinical trials
5.4.1.2 Clinical results
5.4.1.3 Strategic development
5.4.1.4 Technology
5.4.2 BAY1093884
5.4.2.1 Clinical trials
5.4.2.2 Clinical trial results
5.4.3 Concizumab
5.4.3.1 Pre-Clinical studies
5.4.3.2 Clinical trials
5.4.4 Marzeptacog alfa (activated)
5.4.4.1 Clinical trials
5.4.4.2 Clinical trial results
5.4.4.3 Strategic development
5.4.4.4 Designation
5.4.5 ATX-F8117
5.4.5.1 Designation
5.4.6 BMN-270
5.4.6.1 Pre-Clinical studies
5.4.6.2 Clinical trials
5.4.6.3 Clinical trial results
5.4.6.4 Designation
5.4.7 NN7170
5.4.7.1 Clinical trials
5.4.7.2 Strategic development
5.4.7.3 Technology
5.4.8 SB-525 cDNA gene therapy
5.4.8.1 Clinical trials
5.4.8.2 Strategic development
5.4.8.3 Designation
5.4.9 SB-FIX
5.4.9.1 Pre-Clinical studies
5.4.9.2 Clinical trials
5.4.9.3 Technology
5.4.9.4 Designation
5.4.10 SPK-8011
5.4.10.1 Clinical trials
5.4.10.2 Technology
5.4.11 Factor IX
5.4.11.1 Pre-Clinical studies
5.4.12 AMT-060
5.4.12.1 Clinical trials
5.4.12.2 Clinical trial results
5.4.12.3 Strategic development
5.4.12.4 Technology
5.4.12.5 Designation
5.5 Pre-Clinical
5.6 Discovery
Chapter 6. Competitive Landscape
6.1 Key Players Benchmarking for Hemophilia Drug Pipeline
6.2 SWOT Analysis of Hemophilia Pipeline
Chapter 7. Company Profiles
7.1 F. Hoffmann-La Roche Ltd.
7.1.1 Business overview
7.1.2 Product and service offerings
7.2 Pfizer Inc.
7.2.1 Business overview
7.2.2 Product and service offerings
7.3 Bayer, AG
7.3.1 Business overview
7.3.2 Product and service offerings
7.4 Spark Therapeutics, Inc.
7.4.1 Business overview
7.4.2 Product and service offerings
7.5 Catalyst Biosciences, Inc.
7.5.1 Business overview
7.5.2 Product and service offerings
7.6 Alnylam Pharmaceuticals, Inc.
7.6.1 Business overview
7.6.2 Product and service offerings
7.7 Sangamo BioSciences, Inc.
7.7.1 Business overview
7.7.2 Product and service offerings
7.8 Dimension Therapeutics, Inc.
7.8.1 Business overview
7.8.2 Product and service offerings
7.9 uniQure N.V.
7.9.1 Business overview
7.9.2 Product and service offerings
Chapter 8. Appendix
8.1 Abbreviations
List of Tables
TABLE 1: PIPELINE ANALYSIS OF HEMOPHILIA, BY COMPANY (2023)
TABLE 2: DESCRIPTION OF LR-769
TABLE 3: CLINICAL TRIALS OF LR-769
TABLE 4: DESCRIPTION OF EMICIZUMAB
TABLE 5: CLINICAL TRIALS OF EMICIZUMAB
TABLE 6: DESCRIPTION OF NN7088
TABLE 7: CLINICAL TRIALS OF NN7088
TABLE 8: DESCRIPTION OF DAMOCTOCOG ALFA PEGOL
TABLE 9: CLINICAL TRIALS OF DAMOCTOCOG ALFA PEGOL
TABLE 10: DESCRIPTION OF FITUSIRAN
TABLE 11: CLINICAL TRIALS OF ALN-AT3SC
TABLE 12: DESCRIPTION OF PF-06838435
TABLE 13: CLINICAL TRIALS OF PF-06838435
TABLE 14: DESCRIPTION OF CSL689 RVIIA-FP
TABLE 15: CLINICAL TRIALS OF CSL689
TABLE 16: DESCRIPTION OF PF-06741086
TABLE 17: CLINICAL TRIALS OF PF-06741086
TABLE 18: DESCRIPTION OF SHP656
TABLE 19: CLINICAL TRIALS OF SHP656
TABLE 20: DESCRIPTION OF BAY1093884
TABLE 21: CLINICAL TRIALS OF BAY1093884
TABLE 22: DESCRIPTION OF CONCIZUMAB
TABLE 23: CLINICAL TRIALS OF CONCIZUMAB
TABLE 24: DESCRIPTION OF MARZEPTACOG ALFA (ACTIVATED)
TABLE 25: CLINICAL TRIALS OF MARZEPTACOG ALFA (ACTIVATED)
TABLE 26: DESCRIPTION OF ATX-F8117
TABLE 27: DESCRIPTION OF BMN-270
TABLE 28: CLINICAL TRIALS OF BMN-270
TABLE 29: DESCRIPTION OF NN7170
TABLE 30: CLINICAL TRIALS OF NN7170
TABLE 31: DESCRIPTION OF SB-525 CDNA GENE THERAPY
TABLE 32: CLINICAL TRIALS OF SB-525 CDNA GENE THERAPY
TABLE 33: DESCRIPTION OF SB-FIX
TABLE 34: CLINICAL TRIALS OF SB-FIX
TABLE 35: DESCRIPTION OF SPK-8011
TABLE 36: CLINICAL TRIALS OF SPK-8011
TABLE 37: DESCRIPTION OF FACTOR IX
TABLE 38: DESCRIPTION OF AMT-060
TABLE 39: CLINICAL TRIALS OF AMT-060
TABLE 40: F. HOFFMANN-LA ROCHE LTD. – AT A GLANCE
TABLE 41: PFIZER INC. – AT A GLANCE
TABLE 42: BAYER AG – AT A GLANCE
TABLE 43: SPARK THERAPEUTICS, INC. – AT A GLANCE
TABLE 44: CATALYST BIOSCIENCES, INC. – AT A GLANCE
TABLE 45: ALNYLAM PHARMACEUTICALS, INC. – AT A GLANCE
TABLE 46: SANGAMO BIOSCIENCES, INC. – AT A GLANCE
TABLE 47: DIMENSION THERAPEUTICS, INC. – AT A GLANCE
TABLE 48: UNIQURE N.V. – AT A GLANCE
List of Figures
FIG 1: RESEARCH METHODOLOGY FOR THE HEMOPHILIA PIPELINE ANALYSIS
FIG 2: SPLIT OF PRIMARY AND SECONDARY RESEARCH
FIG 3: BREAKDOWN OF PRIMARY RESEARCH RESPONDENTS, BY INDUSTRY PARTICIPANT
FIG 4: BREAKDOWN OF PRIMARY RESEARCH RESPONDENTS, BY COMPANY TYPE
FIG 5: ROLE OF CLOTTING FCATORS IN COAGUALTION
FIG 6: NUMBER OF HEMOPHILIA DRUG CANDIDATES UNDER DEVELOPMENT (2023)
FIG 7: HEMOPHILIA PIPELINE SPLIT, BY MOLECULE TYPE (2023)
FIG 8: HEMOPHILIA PIPELINE SPLIT, BY ROUTE OF ADMINISTRATION (2023)
FIG 9: KEY PLAYERS BENCHMARKING FOR HEMOPHILIA DRUG PIPELINE
FIG 10: SWOT ANALYSIS OF HEMOPHILIA PIPELINE
