【レポートの概要（一部）】
Table of Contents
Table of Contents 2
List of Tables 5
List of Figures 6
Introduction 7
Global Markets Direct Report Coverage 7
Alpha- Antitrypsin Deficiency Overview 8
Therapeutics Development 9
Pipeline Products for Alpha- Antitrypsin Deficiency – Overview 9
Pipeline Products for Alpha- Antitrypsin Deficiency – Comparative Analysis 10
Alpha- Antitrypsin Deficiency – Therapeutics under Development by Companies 11
Alpha- Antitrypsin Deficiency – Therapeutics under Investigation by Universities/Institutes 13
Alpha- Antitrypsin Deficiency – Pipeline Products Glance 14
Late Stage Products 14
Clinical Stage Products 15
Early Stage Products 16
Alpha- Antitrypsin Deficiency – Products under Development by Companies 17
Alpha- Antitrypsin Deficiency – Products under Investigation by Universities/Institutes 19
Alpha- Antitrypsin Deficiency – Companies Involved in Therapeutics Development 20
Applied Genetic Technologies Corporation 20
Arrowhead Research Corporation 21
Carolus Therapeutics, Inc. 22
Cevec Pharmaceuticals GmbH 23
Dicerna Pharmaceuticals, Inc. 24
Grifols, S.A. 25
iBio, Inc. 26
Inhibrx 27
International Stem Cell Corporation 28
Intrexon Corporation 29
Kamada Ltd. 30
Polyphor Ltd. 31
Promethera Biosciences S.A. 32
Sangamo BioSciences, Inc. 33
Alpha- Antitrypsin Deficiency – Therapeutics Assessment 34
Assessment by Monotherapy Products 34
Assessment by Target 35
Assessment by Mechanism of Action 37
Assessment by Route of Administration 39
Assessment by Molecule Type 41
Drug Profiles 43
Alpha-1 Antitrypsin With rHuPH20 – Drug Profile 43
Product Description 43
Mechanism of Action 43
R&D Progress 43
alpha-1 proteinase inhibitor (human) second generation – Drug Profile 44
Product Description 44
Mechanism of Action 44
R&D Progress 44
ARC-AAT – Drug Profile 47
Product Description 47
Mechanism of Action 47
R&D Progress 47
CT-2009 – Drug Profile 48
Product Description 48
Mechanism of Action 48
R&D Progress 48
DC-AIM – Drug Profile 49
Product Description 49
Mechanism of Action 49
R&D Progress 49
Gene Therapy to Activate A1AT for Alpha 1-Antitrypsin Deficiency – Drug Profile 50
Product Description 50
Mechanism of Action 50
R&D Progress 50
Gene Therapy to Activate Alpha-1 Antitrypsin – Drug Profile 51
Product Description 51
Mechanism of Action 51
R&D Progress 51
HepaStem – Drug Profile 52
Product Description 52
Mechanism of Action 52
R&D Progress 52
POL-6014 – Drug Profile 54
Product Description 54
Mechanism of Action 54
R&D Progress 54
rAAV1-CB-hAAT – Drug Profile 56
Product Description 56
Mechanism of Action 56
R&D Progress 56
rAAV2-CB-hAAT – Drug Profile 57
Product Description 57
Mechanism of Action 57
R&D Progress 57
Recombinant A1PI – Drug Profile 58
Product Description 58
Mechanism of Action 58
R&D Progress 58
Recombinant Human Alpha-1 Antitrypsin – Drug Profile 60
Product Description 60
Mechanism of Action 60
R&D Progress 60
Recombinant Protein to Replace Alpha-1 Antitrypsin for Alpha Antitrypsin Deficiency – Drug Profile 61
Product Description 61
Mechanism of Action 61
R&D Progress 61
Recombinant Protein to Replace Alpha-1 Antitrypsin for Alpha-Antitrypsin Deficiency – Drug Profile 62
Product Description 62
Mechanism of Action 62
R&D Progress 62
RNAi Oligonucleotide for Alpha- Antitrypsin Deficiency – Drug Profile 63
Product Description 63
Mechanism of Action 63
R&D Progress 63
Stem Cell Therapy for Alpha-Antitrypsin Deficiency and Crigler-Najjar Syndrome – Drug Profile 65
Product Description 65
Mechanism of Action 65
R&D Progress 65
Alpha- Antitrypsin Deficiency – Recent Pipeline Updates 66
Alpha- Antitrypsin Deficiency – Dormant Projects 70
Alpha- Antitrypsin Deficiency – Discontinued Products 71
Alpha- Antitrypsin Deficiency – Product Development Milestones 72
Featured News & Press Releases 72
Jan 07, 2015: Kamada Reports Encouraging Discussions with European Co-Rapporteurs Regarding European Filing for Inhaled Alpha-1 Antitrypsin to Treat AATD 72
Nov 18, 2014: Arrowhead Files For Regulatory Permission To Begin Phase 1 Trial Of RNAi Therapeutic ARC-AAT 72
Nov 10, 2014: Arrowhead Presents Data on ARC-AAT at AASLD The Liver Meeting 2014 73
Sep 04, 2014: Kamada Reports Final Results from Phase 2/3 Clinical Trial of Inhaled Alpha-1 Antitrypsin to Treat Alpha-1 Antitrypsin Deficiency 74
Sep 03, 2014: Kamada to Announce Final Results from Phase 2/3 Clinical Trial of Inhaled AAT to Treat Alpha-1 Antitrypsin Deficiency on September 4, 2014 74
Jun 19, 2014: Arrowhead Announces New Clinical Candidate ARC-AAT for Treatment of Alpha-1 Antitrypsin Deficiency-Associated Liver Disease 75
May 16, 2014: Kamada Announces Preliminary Results from Phase II/III Pivotal Trial in Europe and Canada of Inhaled AAT to Treat Alpha-1 Antitrypsin Deficiency 76
May 15, 2014: Kamada to Announce Results from Phase 2/3 Clinical Trial of Inhaled AAT to Treat Alpha-1 Antitrypsin Deficiency on May 16 77
Mar 24, 2014: Kamada Announces Initiation of a Phase 2 U.S. Clinical Trial of Inhaled AAT to Treat Alpha-1 Antitrypsin Deficiency 78
Mar 21, 2014: Kamada to Report Top-Line Data from Phase 2/3 Clinical Trial of Inhaled AAT to Treat Alpha-1 Antitrypsin Deficiency by Late April or Early May 78
Appendix 80
Methodology 80
Coverage 80
Secondary Research 80
Primary Research 80
Expert Panel Validation 80
Contact Us 80
Disclaimer 81
【レポート販売概要】
■ タイトル：α1アンチトリプシン欠乏症(Alpha- Antitrypsin Deficiency)：治療薬開発パイプライン動向（2015年上半期版）■ 英文：Alpha- Antitrypsin Deficiency - Pipeline Review, H1 2015
■ 発行日：2015年2月24日
■ 調査会社：Global Markets Direct
■ 商品コード：GMDHC6282IDB
■ 調査対象地域：グローバル
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