【レポートの概要（一部）】
Table of Contents
Table of Contents 2
List of Tables 4
List of Figures 5
Introduction 6
Global Markets Direct Report Coverage 6
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) Overview 7
Therapeutics Development 8
Pipeline Products for Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) – Overview 8
Pipeline Products for Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) – Comparative Analysis 9
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) – Therapeutics under Development by Companies 10
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) – Therapeutics under Investigation by Universities/Institutes 11
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) – Pipeline Products Glance 12
Late Stage Products 12
Clinical Stage Products 13
Early Stage Products 14
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) – Products under Development by Companies 15
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) – Products under Investigation by Universities/Institutes 16
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) – Companies Involved in Therapeutics Development 17
Alnylam Pharmaceuticals, Inc. 17
Arcturus Therapeutics, Inc 18
Bsim2 19
GlaxoSmithKline plc 20
Isis Pharmaceuticals, Inc. 21
Pfizer Inc. 22
SOM Innovation Biotech SL 23
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) – Therapeutics Assessment 24
Assessment by Monotherapy Products 24
Assessment by Target 25
Assessment by Mechanism of Action 26
Assessment by Route of Administration 28
Assessment by Molecule Type 30
Drug Profiles 32
Antisense Oligonucleotide for Transthyretin Amyloidosis – Drug Profile 32
Product Description 32
Mechanism of Action 32
R&D Progress 32
ISIS-TTRRx – Drug Profile 33
Product Description 33
Mechanism of Action 33
R&D Progress 33
LUNAR-102 – Drug Profile 34
Product Description 34
Mechanism of Action 34
R&D Progress 34
Monoclonal Antibody to Inhibit ATTR for Familial Amyloid Polyneuropathy – Drug Profile 35
Product Description 35
Mechanism of Action 35
R&D Progress 35
patisiran – Drug Profile 36
Product Description 36
Mechanism of Action 36
R&D Progress 36
Small Molecules to Inhibit Transthyretin for Familial Amyloid Polyneuropathy – Drug Profile 37
Product Description 37
Mechanism of Action 37
R&D Progress 37
Small Molecules to Inhibit Transthyretin Related Amyloid Protein for Familial Amyloidotic Polyneuropathy – Drug Profile 38
Product Description 38
Mechanism of Action 38
R&D Progress 38
tafamidis meglumine – Drug Profile 39
Product Description 39
Mechanism of Action 39
R&D Progress 39
tolcapone – Drug Profile 41
Product Description 41
Mechanism of Action 41
R&D Progress 41
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) – Recent Pipeline Updates 42
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) – Dormant Projects 51
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade’s Disease) – Product Development Milestones 52
Featured News & Press Releases 52
Mar 02, 2015: Isis Pharmaceuticals Earns $15 Million From GSK for Advancing ISIS-TTR 52
Oct 13, 2014: Arcturus Therapeutics to Present Gene Knockdown Data in Non-Human Primates, Showing up to 94% Reduction in Gene Expression with a Single Low Dose 52
Oct 13, 2014: Alnylam Reports Six-Month Clinical Data from Patisiran Phase 2 Open-Label Extension (OLE) Study in Patients with Familial Amyloidotic Polyneuropathy (FAP) 52
Oct 09, 2014: Isis Pharmaceuticals Earns $18 Million From GSK for Advancing ISIS-TTR Rx 54
Jul 16, 2014: Isis Pharmaceuticals Earns $1 Million from GSK for Advancing ISIS-TTR Rx 54
Jun 24, 2014: Isis Pharmaceuticals Earns $1 Million From GSK for Advancing ISIS-TTR Rx 55
May 15, 2014: Isis Pharmaceuticals Earns $3.0 Million from GSK 55
Mar 21, 2014: Alnylam to Present Data from its RNAi Therapeutic Programs Targeting Transthyretin for the Treatment of TTR-Mediated Amyloidosis at XIVth International Symposium on Amyloidosis 56
Feb 14, 2014: Isis Announces Positive Opinion on European Orphan Drug Designation for ISIS-TTR Rx 56
Jan 10, 2014: SOM Biotech announces Orphan Drug Designation in US for SOM0226, a compound for the treatment of Transthyretin Amyloidosis 57
Appendix 58
Methodology 58
Coverage 58
Secondary Research 58
Primary Research 58
Expert Panel Validation 58
Contact Us 58
Disclaimer 59
【レポート販売概要】
■ タイトル：家族性アミロイドポリニューロパチー(トランスサイレチンアミロイドーシス)(Familial Amyloid Polyneuropathy)：治療薬開発パイプライン動向（2015年上半期版）■ 英文：Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) - Pipeline Review, H1 2015
■ 発行日：2015年3月19日
■ 調査会社：Global Markets Direct
■ 商品コード：GMDHC6428IDB
■ 調査対象地域：グローバル
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