Sections
| Title | Starting Page | Number of Pages |
|---|
| Table of Contents | 2 | 8 |
| List of Tables | 8 | 1 |
| List of Figures | 9 | 1 |
| Introduction | 10 | 1 |
| Global Markets Direct Report Coverage | 10 | 1 |
| Thalassemia Overview | 11 | 1 |
| Therapeutics Development | 12 | 2 |
| Pipeline Products for Thalassemia Overview | 12 | 1 |
| Pipeline Products for Thalassemia Comparative Analysis | 13 | 1 |
| Thalassemia Therapeutics under Development by Companies | 14 | 2 |
| Thalassemia Therapeutics under Investigation by Universities/Institutes | 16 | 1 |
| Thalassemia Pipeline Products Glance | 17 | 3 |
| Late Stage Products | 17 | 1 |
| Clinical Stage Products | 18 | 1 |
| Early Stage Products | 19 | 1 |
| Thalassemia Products under Development by Companies | 20 | 2 |
| Thalassemia Products under Investigation by Universities/Institutes | 22 | 1 |
| Thalassemia Companies Involved in Therapeutics Development | 23 | 22 |
| Acceleron Pharma, Inc. | 23 | 1 |
| Acetylon Pharmaceuticals, Inc. | 24 | 1 |
| Acino International AG | 25 | 1 |
| Agios Pharmaceuticals, Inc. | 26 | 1 |
| Alnylam Pharmaceuticals, Inc. | 27 | 1 |
| Bellicum Pharmaceuticals, Inc. | 28 | 1 |
| Bluebird bio, Inc. | 29 | 1 |
| Editas Medicine, Inc. | 30 | 1 |
| Emmaus Medical, Inc. | 31 | 1 |
| Errant Gene Therapeutics, LLC | 32 | 1 |
| Gamida Cell Ltd. | 33 | 1 |
| Gilead Sciences, Inc. | 34 | 1 |
| Incyte Corporation | 35 | 1 |
| Ionis Pharmaceuticals, Inc. | 36 | 1 |
| IRBM Science Park SpA | 37 | 1 |
| Johnson &Johnson | 38 | 1 |
| Kiadis Pharma N.V. | 39 | 1 |
| La Jolla Pharmaceutical Company | 40 | 1 |
| Merck &Co., Inc. | 41 | 1 |
| Pfizer Inc. | 42 | 1 |
| PharmaEssentia Corporation | 43 | 1 |
| Sangamo BioSciences, Inc. | 44 | 1 |
| Thalassemia Therapeutics Assessment | 45 | 9 |
| Assessment by Monotherapy Products | 45 | 1 |
| Assessment by Target | 46 | 2 |
| Assessment by Mechanism of Action | 48 | 2 |
| Assessment by Route of Administration | 50 | 2 |
| Assessment by Molecule Type | 52 | 2 |
| Drug Profiles | 54 | 76 |
| ACY-957 Drug Profile | 54 | 2 |
| AG-348 Drug Profile | 56 | 3 |
| ALN-TMP Drug Profile | 59 | 2 |
| ambrisentan Drug Profile | 61 | 2 |
| Antisense Oligonucleotides to Inhibit BCL11A and KLF1 for Hematological Disorders Drug Profile | 63 | 1 |
| ATIR-201 Drug Profile | 64 | 1 |
| BB-305 Drug Profile | 65 | 6 |
| benserazide Drug Profile | 71 | 2 |
| BPX-501 Drug Profile | 73 | 4 |
| BtX-13 Drug Profile | 77 | 1 |
| CNTO-530 Drug Profile | 78 | 1 |
| CordIn Drug Profile | 79 | 1 |
| Drug for Thalassemia and Hemochromatosis Drug Profile | 80 | 1 |
| Gene Therapy for Sickle Cell Disease and Thalassemia Drug Profile | 81 | 1 |
| glutamine Drug Profile | 82 | 3 |
| GSK-2696277 Drug Profile | 85 | 1 |
| IONISTMPRSS-6LRx Drug Profile | 86 | 1 |
| LJPC-401 Drug Profile | 87 | 1 |
| luspatercept Drug Profile | 88 | 6 |
| M-009 Drug Profile | 94 | 1 |
| M-012 Drug Profile | 95 | 2 |
| NiCord Drug Profile | 97 | 3 |
| PF-04447943 Drug Profile | 100 | 1 |
| Piggybac-HBB Drug Profile | 101 | 1 |
| PTG-300 Drug Profile | 102 | 1 |
| RNAi Gene Therapy for Sickle Cell Disease and Beta Thalassemia Drug Profile | 103 | 1 |
| ruxolitinib phosphate Drug Profile | 104 | 13 |
| SCD-101 Drug Profile | 117 | 1 |
| sirolimus Drug Profile | 118 | 1 |
| Small Molecule for Beta Thalassemia and Sickle Cell Anemia Drug Profile | 119 | 1 |
| Small Molecule to Inhibit Histone Deacetylase 1 for Beta-Thalassemia Drug Profile | 120 | 1 |
| Small Molecules to Inhibit PRMT5 for Haemoglobinopathies and Solid Tumours Drug Profile | 121 | 1 |
| sotatercept Drug Profile | 122 | 5 |
| Stem Cell Therapy for Beta Thalassemia Drug Profile | 127 | 1 |
| Stem Cell Therapy for Beta Thalassemia and Sickle Cell Disease Drug Profile | 128 | 1 |
| Thalagen Drug Profile | 129 | 1 |
| Thalassemia Dormant Projects | 130 | 1 |
| Thalassemia Product Development Milestones | 131 | 12 |
| Featured News &Press Releases | 131 | 1 |
| Jul 28, 2016: Protagonist Therapeutics Receives SBIR Funding for the Development of Injectable Hepcidin Mimetics for Treatment of Iron Overload Disorders | 131 | 1 |
| Jun 10, 2016: Acceleron and Celgene Announce Updated Results from an Ongoing Phase 2 Study of Luspatercept in Beta-Thalassemia Presented at the 21st Congress of the European Hematology Association | 131 | 1 |
| May 19, 2016: Agios to Present Clinical data from PKR Activator AG-348 at the 21st Congress of the European Hematology Association | 132 | 1 |
| May 19, 2016: Acceleron Announces Data Presentations with Luspatercept at the 21st Congress of the European Hematology Association | 133 | 1 |
| May 09, 2016: Merganser Biotech Announces Issuance of U.S. Patent 9,315,545 Covering Development Compound M012 | 134 | 1 |
| May 06, 2016: Drug-like Compound Shows Promise in Treating Two Blood Diseases | 134 | 1 |
| May 04, 2016: Acetylon Pharmaceuticals Publishes New Mechanism for the Treatment of Sickle Cell Disease/Beta-Thalassemia with Selective HDAC1/2 Inhibition in PLoS One | 135 | 1 |
| Feb 24, 2016: Merganser Biotech Initiates First Clinical Trial | 136 | 1 |
| Dec 07, 2015: Acetylon Presents Data on the Pharmacokinetics and HbE/HbG Hemoglobin Induction Following Once Daily Dosing of an Oral HDAC1,2 Inhibitor in Animal Models | 136 | 1 |
| Dec 02, 2015: Emmaus Life Sciences to Present Additional New Data from its Phase 3 Sickle Cell Disease Trial at 57th Annual ASH Meeting | 137 | 1 |
| Nov 05, 2015: Acceleron Announces New Data on Luspatercept at the 2015 American Society of Hematology Annual Meeting | 138 | 1 |
| Oct 23, 2015: Acceleron Highlights Phase 3 Studies, New Clinical Results and Research Strategies at Research and Development Day Event | 139 | 1 |
| Oct 19, 2015: La Jolla Pharmaceutical Doses First Patient in Phase 1 Clinical Trial of LJPC-401 in Patients at Risk of Iron Overload | 139 | 1 |
| Aug 11, 2015: La Jolla Pharmaceutical Company Announces FDA Acceptance of IND for LJPC-401 | 140 | 1 |
| Jun 12, 2015: Acceleron Announces New Luspatercept Phase 2 Clinical Results at the 20th Congress of the European Hematology Association | 141 | 2 |
| Appendix | 143 | 2 |
| Methodology | 143 | 1 |
| Coverage | 143 | 1 |
| Secondary Research | 143 | 1 |
| Primary Research | 143 | 1 |
| Expert Panel Validation | 143 | 1 |
| Contact Us | 143 | 1 |
| Disclaimer | 144 | 1 |
