ライソゾーム病(Lysosomal Storage Disorder)：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 6
List of Figures 7
Introduction 8
Global Markets Direct Report Coverage 8
Lysosomal Storage Disorder Overview 9
Therapeutics Development 10
Pipeline Products for Lysosomal Storage Disorder – Overview 10
Pipeline Products for Lysosomal Storage Disorder – Comparative Analysis 11
Lysosomal Storage Disorder – Therapeutics under Development by Companies 12
Lysosomal Storage Disorder – Therapeutics under Investigation by Universities/Institutes 14
Lysosomal Storage Disorder – Pipeline Products Glance 15
Late Stage Products 15
Clinical Stage Products 16
Early Stage Products 17
Lysosomal Storage Disorder – Products under Development by Companies 18
Lysosomal Storage Disorder – Products under Investigation by Universities/Institutes 20
Lysosomal Storage Disorder – Companies Involved in Therapeutics Development 21
Amicus Therapeutics, Inc. 21
AngioChem Inc. 22
BBB Therapeutics B.V. 23
Chiesi Farmaceutici SpA 24
Fate Therapeutics, Inc. 25
Minoryx Therapeutics s.l. 26
Neuralstem, Inc. 27
Nuo Therapeutics, Inc. 28
Orphazyme ApS 29
Oxyrane Belgium NV 30
REGiMMUNE Corporation 31
Sagetis Biotech, S.L. 32
Sangamo BioSciences, Inc. 33
Synageva BioPharma Corp. 34
Ultragenyx Pharmaceutical Inc. 35
Lysosomal Storage Disorder – Therapeutics Assessment 36
Assessment by Monotherapy Products 36
Assessment by Target 37
Assessment by Mechanism of Action 39
Assessment by Route of Administration 41
Assessment by Molecule Type 43
Drug Profiles 45
2B3-201 – Drug Profile 45
Product Description 45
Mechanism of Action 45
R&D Progress 45
ALD-601 – Drug Profile 47
Product Description 47
Mechanism of Action 47
R&D Progress 47
Chaperone-ERT Combinations – Drug Profile 48
Product Description 48
Mechanism of Action 48
R&D Progress 48
Delta-tocopherol – Drug Profile 49
Product Description 49
Mechanism of Action 49
R&D Progress 49
FT-1050 – Drug Profile 50
Product Description 50
Mechanism of Action 50
R&D Progress 50
ML-SA1 – Drug Profile 53
Product Description 53
Mechanism of Action 53
R&D Progress 53
NSI-566 – Drug Profile 54
Product Description 54
Mechanism of Action 54
R&D Progress 54
Orph-001 – Drug Profile 56
Product Description 56
Mechanism of Action 56
R&D Progress 56
Recombinant Enzyme for Farber Disease and Cystic Fibrosis – Drug Profile 57
Product Description 57
Mechanism of Action 57
R&D Progress 57
Recombinant Enzyme for LSD-2 Disease – Drug Profile 58
Product Description 58
Mechanism of Action 58
R&D Progress 58
Recombinant Enzyme for LSD-3 Disease – Drug Profile 59
Product Description 59
Mechanism of Action 59
R&D Progress 59
Recombinant Enzyme for LSD-4 Disease – Drug Profile 60
Product Description 60
Mechanism of Action 60
R&D Progress 60
Recombinant Enzyme for LSD-5 Disease – Drug Profile 61
Product Description 61
Mechanism of Action 61
R&D Progress 61
Recombinant Enzyme for Lysosomal Storage Disorder – Drug Profile 62
Product Description 62
Mechanism of Action 62
R&D Progress 62
Recombinant Enzyme to Activate LRP1 for Lysosomal Storage Disorder – Drug Profile 63
Product Description 63
Mechanism of Action 63
R&D Progress 63
RGI-5000 – Drug Profile 64
Product Description 64
Mechanism of Action 64
R&D Progress 64
SAG-002 – Drug Profile 65
Product Description 65
Mechanism of Action 65
R&D Progress 65
SBLSD-3 – Drug Profile 66
Product Description 66
Mechanism of Action 66
R&D Progress 66
SBLSD-4 – Drug Profile 67
Product Description 67
Mechanism of Action 67
R&D Progress 67
sebelipase alfa – Drug Profile 68
Product Description 68
Mechanism of Action 68
R&D Progress 68
Small Molecule to Activate Beta-Galactosidase for GM1 Gangliosidosis and Gaucher’s disease – Drug Profile 70
Product Description 70
Mechanism of Action 70
R&D Progress 70
Small Molecules for GM1-gangliosidosis And Morquio B – Drug Profile 71
Product Description 71
Mechanism of Action 71
R&D Progress 71
Small Molecules for Lysosomal Storage Disorders – Drug Profile 72
Product Description 72
Mechanism of Action 72
R&D Progress 72
Stem Cell Therapy for Lysosomal Storage Disorder – Drug Profile 73
Product Description 73
Mechanism of Action 73
R&D Progress 73
UX-002 – Drug Profile 74
Product Description 74
Mechanism of Action 74
R&D Progress 74
UX-004 – Drug Profile 75
Product Description 75
Mechanism of Action 75
R&D Progress 75
ZA-011 – Drug Profile 76
Product Description 76
Mechanism of Action 76
R&D Progress 76
Lysosomal Storage Disorder – Recent Pipeline Updates 77
Lysosomal Storage Disorder – Dormant Projects 94
Lysosomal Storage Disorder – Product Development Milestones 95
Featured News & Press Releases 95
Feb 23, 2015: FDA Accepts BLA Filing For Synageva’s Kanuma (Sebelipase Alfa); Grants Priority Review And Assigns PDUFA Date 95
Feb 18, 2015: Synageva European Patent Issued For Treatment Of LAL Deficiency 95
Feb 11, 2015: Synageva BioPharma Announces Presentations At 11th Annual World Symposium 96
Dec 23, 2014: Synageva BioPharma Announces Sebelipase Alfa Marketing Authorization Application Validated By The EMA 97
Dec 02, 2014: Synageva BioPharma Completes Rolling Submission Of Biologics License Application To The FDA For Sebelipase Alfa And Submits Marketing Authorization Application To European Medicines Agency 97
Nov 10, 2014: Synageva BioPharma Presents New Data From Phase 3 Study Of Sebelipase Alfa In Children And Adults With LAL Deficiency At The AASLD Meeting 98
Oct 23, 2014: Synageva BioPharma Presents Sebelipase Alfa Data At The NASPGHAN Meeting 100
Oct 21, 2014: Synageva BioPharma Starts Rolling Submission Of A Biologics License Application To The FDA For Sebelipase Alfa 101
Oct 09, 2014: Sebelipase Alfa Phase 3 Data In Children And Adults With LAL Deficiency Selected For Oral Presentation During The Late-Breaking Session At The Liver Meeting 102
Jul 29, 2014: Fate Therapeutics Announces FDA Clearance of IND for Clinical Development of PROHEMA in Inherited Metabolic Disorders 102
Appendix 104
Methodology 104
Coverage 104
Secondary Research 104
Primary Research 104
Expert Panel Validation 104
Contact Us 104
Disclaimer 105