サラセミア(Thalassemia)：治療薬開発パイプライン動向（2015年上半期版）...市場調査レポートについてご紹介

Table of Contents
Table of Contents 2
List of Tables 5
List of Figures 6
Introduction 7
Global Markets Direct Report Coverage 7
Thalassemia Overview 8
Therapeutics Development 9
Pipeline Products for Thalassemia – Overview 9
Pipeline Products for Thalassemia – Comparative Analysis 10
Thalassemia – Therapeutics under Development by Companies 11
Thalassemia – Therapeutics under Investigation by Universities/Institutes 12
Thalassemia – Pipeline Products Glance 13
Late Stage Products 13
Clinical Stage Products 14
Early Stage Products 15
Thalassemia – Products under Development by Companies 16
Thalassemia – Products under Investigation by Universities/Institutes 18
Thalassemia – Companies Involved in Therapeutics Development 19
Acceleron Pharma, Inc. 19
Acetylon Pharmaceuticals, Inc. 20
Acino Pharma AG 21
Alnylam Pharmaceuticals, Inc. 22
Bluebird bio, Inc. 23
Emmaus Medical, Inc. 24
Errant Gene Therapeutics, LLC 25
Gamida Cell Ltd. 26
Incyte Corporation 27
IRBM Science Park SpA 28
Isis Pharmaceuticals, Inc. 29
Johnson & Johnson 30
Protagonist Therapeutics Inc. 31
Sangamo BioSciences, Inc. 32
Thalassemia – Therapeutics Assessment 33
Assessment by Monotherapy Products 33
Assessment by Target 34
Assessment by Mechanism of Action 36
Assessment by Route of Administration 38
Assessment by Molecule Type 40
Drug Profiles 42
ACY-957 – Drug Profile 42
Product Description 42
Mechanism of Action 42
R&D Progress 42
ALN-TMP – Drug Profile 43
Product Description 43
Mechanism of Action 43
R&D Progress 43
Antisense Oligonucleotides to Inhibit BCL11A and KLF1 for Hematological Disorders – Drug Profile 44
Product Description 44
Mechanism of Action 44
R&D Progress 44
BB-305 – Drug Profile 45
Product Description 45
Mechanism of Action 45
R&D Progress 45
BtX-13 – Drug Profile 47
Product Description 47
Mechanism of Action 47
R&D Progress 47
CNTO-530 – Drug Profile 48
Product Description 48
Mechanism of Action 48
R&D Progress 48
Gene Therapy for Sickle Cell Disease and Beta Thalassemia – Drug Profile 49
Product Description 49
Mechanism of Action 49
R&D Progress 49
Gene Therapy for Sickle Cell Disease and Thalassemia – Drug Profile 50
Product Description 50
Mechanism of Action 50
R&D Progress 50
glutamine – Drug Profile 51
Product Description 51
Mechanism of Action 51
R&D Progress 51
luspatercept – Drug Profile 53
Product Description 53
Mechanism of Action 53
R&D Progress 53
NiCord – Drug Profile 54
Product Description 54
Mechanism of Action 54
R&D Progress 54
PB-04 – Drug Profile 55
Product Description 55
Mechanism of Action 55
R&D Progress 55
Peptide Agonists of Hepcidin for Beta-Thalassemia and Juvenile Hemochromatosis – Drug Profile 56
Product Description 56
Mechanism of Action 56
R&D Progress 56
Project x-1450 – Drug Profile 57
Product Description 57
Mechanism of Action 57
R&D Progress 57
ruxolitinib phosphate – Drug Profile 58
Product Description 58
Mechanism of Action 58
R&D Progress 58
Small Molecule to Inhibit Histone Deacetylase for Beta-Thalassemia – Drug Profile 63
Product Description 63
Mechanism of Action 63
R&D Progress 63
sotatercept – Drug Profile 64
Product Description 64
Mechanism of Action 64
R&D Progress 64
Stem Cell Therapy for Beta Thalassemia – Drug Profile 66
Product Description 66
Mechanism of Action 66
R&D Progress 66
Synthetic Peptides Hematological and Genetic Disorders – Drug Profile 67
Product Description 67
Mechanism of Action 67
R&D Progress 67
Thalagen – Drug Profile 69
Product Description 69
Mechanism of Action 69
R&D Progress 69
Thalassemia – Recent Pipeline Updates 70
Thalassemia – Dormant Projects 90
Thalassemia – Product Development Milestones 91
Featured News & Press Releases 91
Feb 04, 2015: Sangamo BioSciences Announces FDA Acceptance of IND to Initiate Clinical Trial of its Novel ZFP Therapeutic for Beta-Thalassemia 91
Dec 08, 2014: Acetylon Pharmaceuticals Presents Positive Preclinical Data on Selective HDAC1/2 Inhibition for the Treatment of Sickle Cell Disease/ß-thalassemia 92
Dec 07, 2014: New Data from Luspatercept Phase 2 Clinical Trial in Beta-Thalassemia Presented at the 56th American Society of Hematology (ASH) Annual Meeting and Exposition 92
Nov 06, 2014: Acceleron Announces New Clinical Data Presentations on luspatercept at the 2014 American Society of Hematology Annual Meeting 94
Nov 06, 2014: Celgene To Present Phase II trial Results Update On Luspatercept at American Society of Hematology Annual Meeting 95
Nov 06, 2014: Acceleron Announces New Clinical Data Presentations on sotatercept at the 2014 American Society of Hematology Annual Meeting 95
Oct 01, 2014: Acceleron to Host Educational Webcast on Luspatercept 96
Oct 01, 2014: Acceleron to Host Educational Webcast on Beta-thalassemia 96
Jun 14, 2014: Celgene and Acceleron announces interim data from ACE-536 at the 19th European Hematology Association annual congress 96
Jun 14, 2014: Studies Presented at 19th European Hematology Association Annual Congress Evaluate Activin Receptor Ligand Trap Programs in Beta-Thalassemia 97
Appendix 99
Methodology 99
Coverage 99
Secondary Research 99
Primary Research 99
Expert Panel Validation 99
Contact Us 99
Disclaimer 100