脊髄性筋萎縮症(Spinal Muscular Atrophy (SMA))の治療薬パイプライン動向（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 6
List of Figures 7
Introduction 8
Global Markets Direct Report Coverage 8
Spinal Muscular Atrophy (SMA) Overview 9
Therapeutics Development 10
Pipeline Products for Spinal Muscular Atrophy (SMA) – Overview 10
Pipeline Products for Spinal Muscular Atrophy (SMA) – Comparative Analysis 11
Spinal Muscular Atrophy (SMA) – Therapeutics under Development by Companies 12
Spinal Muscular Atrophy (SMA) – Therapeutics under Investigation by Universities/Institutes 15
Spinal Muscular Atrophy (SMA) – Pipeline Products Glance 16
Late Stage Products 16
Clinical Stage Products 17
Early Stage Products 18
Unknown Stage Products 19
Spinal Muscular Atrophy (SMA) – Products under Development by Companies 20
Spinal Muscular Atrophy (SMA) – Products under Investigation by Universities/Institutes 22
Spinal Muscular Atrophy (SMA) – Companies Involved in Therapeutics Development 23
Bioblast Pharma Ltd. 23
Cellular Biomedicine Group, Inc. 24
Cytokinetics, Inc. 25
F. Hoffmann-La Roche Ltd. 26
Genethon 27
Genzyme Corporation 28
GMP-Orphan SAS 29
Isis Pharmaceuticals, Inc. 30
Longevity Biotech, Inc 31
Neurodyn Inc. 32
Neurotune AG 33
Nexgenix Pharmaceuticals, LLC 34
Novartis AG 35
OrphageniX, Inc. 36
Paratek Pharmaceuticals, Inc. 37
Pfizer Inc. 38
PTC Therapeutics, Inc. 39
Retrophin Inc. 40
Sarepta Therapeutics, Inc. 41
Trophos SA 42
Vybion, Inc. 43
Zambon Company S.p.A. 44
Spinal Muscular Atrophy (SMA) – Therapeutics Assessment 45
Assessment by Monotherapy Products 45
Assessment by Target 46
Assessment by Mechanism of Action 48
Assessment by Route of Administration 50
Assessment by Molecule Type 52
Drug Profiles 54
ALB-111 – Drug Profile 54
Product Description 54
Mechanism of Action 54
R&D Progress 54
Antisense Oligonucleotide for Spinal Muscular Atrophy and Myotonic Dystrophy – Drug Profile 56
Product Description 56
Mechanism of Action 56
R&D Progress 56
Antisense Oligonucleotide to Inhibit SMN2 gene for SMA – Drug Profile 57
Product Description 57
Mechanism of Action 57
R&D Progress 57
Antisense Oligonucleotides for Spinal Muscular Atrophy – Drug Profile 58
Product Description 58
Mechanism of Action 58
R&D Progress 58
ataluren – Drug Profile 59
Product Description 59
Mechanism of Action 59
R&D Progress 59
BBrm-01 – Drug Profile 62
Product Description 62
Mechanism of Action 62
R&D Progress 62
DNA Oligonucleotides for Spinal Muscular Atrophy – Drug Profile 63
Product Description 63
Mechanism of Action 63
R&D Progress 63
Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy – Drug Profile 64
Product Description 64
Mechanism of Action 64
R&D Progress 64
Gene Therapy to Activate SMN1 for Spinal Muscular Atrophy – Drug Profile 65
Product Description 65
Mechanism of Action 65
R&D Progress 65
INT-41 – Drug Profile 66
Product Description 66
Mechanism of Action 66
R&D Progress 66
LMI-070 – Drug Profile 68
Product Description 68
Mechanism of Action 68
R&D Progress 68
ND-602 – Drug Profile 69
Product Description 69
Mechanism of Action 69
R&D Progress 69
NT-1654 – Drug Profile 70
Product Description 70
Mechanism of Action 70
R&D Progress 70
nusinersen – Drug Profile 71
Product Description 71
Mechanism of Action 71
R&D Progress 71
NXD-30001 – Drug Profile 73
Product Description 73
Mechanism of Action 73
R&D Progress 73
olesoxime – Drug Profile 75
Product Description 75
Mechanism of Action 75
R&D Progress 75
Oligonucleotide to Activate SMN2 for Spinal Muscular Atrophy – Drug Profile 78
Product Description 78
Mechanism of Action 78
R&D Progress 78
PF-06687859 – Drug Profile 79
Product Description 79
Mechanism of Action 79
R&D Progress 79
PMO-25 – Drug Profile 81
Product Description 81
Mechanism of Action 81
R&D Progress 81
PTKSMA-2 – Drug Profile 82
Product Description 82
Mechanism of Action 82
R&D Progress 82
RE-003 – Drug Profile 83
Product Description 83
Mechanism of Action 83
R&D Progress 83
RG-7800 – Drug Profile 84
Product Description 84
Mechanism of Action 84
R&D Progress 84
scAAV9.CB.SMN – Drug Profile 86
Product Description 86
Mechanism of Action 86
R&D Progress 86
Small Molecules for CNS Disorders – Drug Profile 88
Product Description 88
Mechanism of Action 88
R&D Progress 88
sodium phenylbutyrate – Drug Profile 89
Product Description 89
Mechanism of Action 89
R&D Progress 89
Stem Cell Therapy for Amyotrophic Lateral Sclerosis and Spinal Muscular Atrophy – Drug Profile 90
Product Description 90
Mechanism of Action 90
R&D Progress 90
Synthetic Peptides for Central Nervous System Disorders and Diabetic Neuropathy – Drug Profile 91
Product Description 91
Mechanism of Action 91
R&D Progress 91
tirasemtiv – Drug Profile 92
Product Description 92
Mechanism of Action 92
R&D Progress 92
ZP-037 – Drug Profile 99
Product Description 99
Mechanism of Action 99
R&D Progress 99
Spinal Muscular Atrophy (SMA) – Recent Pipeline Updates 100
Spinal Muscular Atrophy (SMA) – Dormant Projects 119
Spinal Muscular Atrophy (SMA) – Product Development Milestones 120
Featured News & Press Releases 120
Nov 25, 2014: Isis Pharmaceuticals Initiates Phase 3 Study of ISIS-SMN Rx in Children With Spinal Muscular Atrophy 120
Nov 17, 2014: Cure SMA Presents Isis Pharmaceuticals with SMA Breakthrough Award 121
Oct 10, 2014: Isis Pharmaceuticals Reports Data From ISIS-SMN Rx Phase 2 Studies in Infants and Children With Spinal Muscular Atrophy 122
Oct 02, 2014: Isis Pharmaceuticals to Host Webcast to Discuss ISIS-SMN Rx Results Presented at the World Muscle Society Congress 125
Oct 02, 2014: AveXis Receives Orphan Drug Status from the US Food and Drug Administration for chariSMA for the Treatment of Spinal Muscular Atrophy 125
Aug 15, 2014: Isis Pharmaceuticals Earns $2M from the Advancement of ISIS-SMN Rx in Children with Spinal Muscular Atrophy 126
Aug 01, 2014: Isis Pharmaceuticals Initiates Phase 3 Study of ISIS-SMN Rx in Infants with Spinal Muscular Atrophy 127
Jun 13, 2014: Cytokinetics Announces Data Relating To Tirasemtiv Presented At The 2014 Annual Spinal Muscular Atrophy Conference 127
Jun 06, 2014: Cytokinetics Announces Two Presentations Relating To Tirasemtiv To Be Presented At The 2014 Annual Spinal Muscular Atrophy Conference 129
May 22, 2014: Gene therapy extends survival in an animal model of spinal muscular atrophy 129
Appendix 131
Methodology 131
Coverage 131
Secondary Research 131
Primary Research 131
Expert Panel Validation 131
Contact Us 132
Disclaimer 132