Sections
| Title | Starting Page | Number of Pages |
|---|
| Table of Contents | 2 | 6 |
| List of Tables | 6 | 1 |
| List of Figures | 7 | 1 |
| Introduction | 8 | 1 |
| Global Markets Direct Report Coverage | 8 | 1 |
| Hemophilia B Overview | 9 | 1 |
| Therapeutics Development | 10 | 2 |
| Pipeline Products for Hemophilia B Overview | 10 | 1 |
| Pipeline Products for Hemophilia B Comparative Analysis | 11 | 1 |
| Hemophilia B Therapeutics under Development by Companies | 12 | 2 |
| Hemophilia B Therapeutics under Investigation by Universities/Institutes | 14 | 1 |
| Hemophilia B Pipeline Products Glance | 15 | 3 |
| Late Stage Products | 15 | 1 |
| Clinical Stage Products | 16 | 1 |
| Early Stage Products | 17 | 1 |
| Hemophilia B Products under Development by Companies | 18 | 1 |
| Hemophilia B Products under Investigation by Universities/Institutes | 19 | 1 |
| Hemophilia B Companies Involved in Therapeutics Development | 20 | 20 |
| Alnylam Pharmaceuticals, Inc. | 20 | 1 |
| Amarna Therapeutics B.V. | 21 | 1 |
| Bayer AG | 22 | 1 |
| Biogen Inc | 23 | 1 |
| Catalyst Biosciences, Inc. | 24 | 1 |
| China Biologic Products, Inc. | 25 | 1 |
| CSL Limited | 26 | 1 |
| Dimension Therapeutics, Inc. | 27 | 1 |
| Green Cross Corporation | 28 | 1 |
| MolMed S.p.A. | 29 | 1 |
| Novo Nordisk A/S | 30 | 1 |
| OPKO Biologics Ltd | 31 | 1 |
| Pfizer Inc. | 32 | 1 |
| Pharming Group N.V. | 33 | 1 |
| RegenxBio Inc. | 34 | 1 |
| rEVO Biologics, Inc. | 35 | 1 |
| Sangamo BioSciences, Inc. | 36 | 1 |
| Shire Plc | 37 | 1 |
| Spark Therapeutics, Inc. | 38 | 1 |
| UniQure N.V. | 39 | 1 |
| Hemophilia B Therapeutics Assessment | 40 | 7 |
| Assessment by Monotherapy Products | 40 | 1 |
| Assessment by Target | 41 | 2 |
| Assessment by Route of Administration | 43 | 2 |
| Assessment by Molecule Type | 45 | 2 |
| Drug Profiles | 47 | 50 |
| AMT-060 Drug Profile | 47 | 3 |
| BAX-335 Drug Profile | 50 | 2 |
| BAY-1093884 Drug Profile | 52 | 1 |
| CB-2679d Drug Profile | 53 | 1 |
| Cell Therapy for Hemophilia B Drug Profile | 54 | 1 |
| coagulation factor IX (human) Drug Profile | 55 | 1 |
| coagulation factor IX (recombinant) Drug Profile | 56 | 1 |
| coagulation factor IX (recombinant), albumin fusion protein Drug Profile | 57 | 5 |
| coagulation factor IX (recombinant), pegylated Drug Profile | 62 | 2 |
| coagulation factor VIIa (recombinant) Drug Profile | 64 | 2 |
| concizumab Drug Profile | 66 | 1 |
| CSL-689 Drug Profile | 67 | 2 |
| DTX-101 Drug Profile | 69 | 2 |
| fitusiran Drug Profile | 71 | 6 |
| Gene Therapy for Hemophilia B Drug Profile | 77 | 1 |
| Gene Therapy for Hemophilia B Drug Profile | 78 | 1 |
| Gene Therapy to Activate Factor IX for Hemophilia B Drug Profile | 79 | 1 |
| Gene Therapy to Activate Factor IX for Hemophilia B Drug Profile | 80 | 2 |
| LR-769 Drug Profile | 82 | 2 |
| marzeptacog alfa Drug Profile | 84 | 2 |
| MG-1113A Drug Profile | 86 | 1 |
| MOD-5014 Drug Profile | 87 | 3 |
| PF-06741086 Drug Profile | 90 | 1 |
| Recombinant Factor IX Replacement for Hemophilia B Drug Profile | 91 | 1 |
| SB-FIX Drug Profile | 92 | 2 |
| SPK-9001 Drug Profile | 94 | 2 |
| SVF-VIIa Drug Profile | 96 | 1 |
| Hemophilia B Dormant Projects | 97 | 1 |
| Hemophilia B Discontinued Products | 98 | 1 |
| Hemophilia B Product Development Milestones | 99 | 12 |
| Featured News &Press Releases | 99 | 1 |
| Sep 26, 2016: Alprolix obtains reimbursement across the United Kingdom | 99 | 1 |
| Sep 07, 2016: Catalyst Biosciences to Focus on Improved Factor IX Clinical Hemostasis Program | 99 | 1 |
| Sep 06, 2016: Sangamo BioSciences Receives Orphan Drug Designation From The FDA For SB-FIX, The First Application Of Therapeutic In Vivo Genome Editing | 100 | 1 |
| Jul 26, 2016: CSL Behring Presents Phase III Data for IDELVION for Hemophilia B at the World Federation of Hemophilia 2016 World Congress | 101 | 1 |
| Jul 25, 2016: Alnylam Announces New Positive Interim Phase 1 Study Results for Fitusiran, a Once-Monthly, Subcutaneous, Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia and Rare Bleeding Disorders | 102 | 3 |
| Jul 25, 2016: Alnylam to Report New Clinical Results with Fitusiran at the World Federation of Hemophilia 2016 World Congress | 105 | 1 |
| Jul 19, 2016: CSL Behring to Present New Data for IDELVION at the World Federation of Hemophilia 2016 World Congress | 105 | 1 |
| Jul 18, 2016: Biogen and Sobi to Showcase Long-Term Efficacy and Safety Data on ALPROLIX at World Federation of Hemophilia 2016 World Congress | 106 | 1 |
| Jun 06, 2016: Sobi: First Alprolix sales in the EU | 107 | 1 |
| May 24, 2016: Catalyst Biosciences and CMC Biologics Announce Manufacturing Agreement for Catalyst Next-Generation Factor VIIa Product CB 813d | 107 | 1 |
| May 16, 2016: Novo Nordisk files for regulatory approval in the US of long-acting factor IX for the treatment of hemophilia B | 107 | 1 |
| May 13, 2016: Alprolix (rFIXFc) approved in the EU for the treatment of hemophilia B | 108 | 1 |
| May 12, 2016: IDELVION (rFIX-FP) - European Commission Approval | 109 | 1 |
| May 11, 2016: CSL Behring's Innovative Therapy for Hemophilia B Patients, IDELVION, Receives 7 Years of Marketing Exclusivity from the U.S. Food and Drug Administration | 109 | 1 |
| May 11, 2016: European Commission Approves IDELVION --CSL Behring Novel Hemophilia B Treatment with Up to 14-day Dosing Intervals | 110 | 1 |
| Appendix | 111 | 2 |
| Methodology | 111 | 1 |
| Coverage | 111 | 1 |
| Secondary Research | 111 | 1 |
| Primary Research | 111 | 1 |
| Expert Panel Validation | 111 | 1 |
| Contact Us | 111 | 1 |
| Disclaimer | 112 | 1 |
