【レポートの概要（一部）】
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
【レポート販売概要】
■ タイトル：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）■ 英文：Lysosomal Storage Disorder - Pipeline Review, H1 2015
■ 発行日：2015年3月11日
■ 調査会社：Global Markets Direct
■ 商品コード：GMDHC6265IDB
■ 調査対象地域：グローバル
- アウトオブバンド認証（OOBA）の世界市場予測（～2023年）Mandate to follow regulatory and data protection laws is expected to drive the out of band authentication marketThe OOBA market size is expected to grow from USD 533 million in 2018 to USD 1,532 million by 2023, at a Compound Annual Growth Rate (CAGR) of 23.5% during the forecast period. The factors expected to drive the OOBA market are the growing need for frictionless OOBA solutions among organi …
- ソフトウェア・ディファインド・ストレージ（SDS）の世界市場2016-2020About Software Defined Storage (SDS) Software-defined storage (SDS) is a central part of the software-defined data center (SDDC), which is an emerging information technology (IT). It has been designed for organizations to store business data and to enable the speedy delivery of IT services. In an SDS framework, storage services are delivered as a software layer that can be abstracted from the unde …
- 2014年戦略提言：日本のHIV/AIDS診断・検査市場Complete report $2,300. DataPack (test volumes, sales forecasts, supplier shares) $1,500.Summary This report presents detailed analyses of the AIDS diagnostics market in Japan. The report provides test volume and sales forecasts for HIV-1/2, HIV Ag, NAT, Western Blot and other confirmatory tests performed in the following market segments: - Hospitals …
- 水産飼料成分・製品の世界市場About Aquatic Feed Formulated aqua feed, which contains vital ingredients for aquatic species such as fish, crustaceans, and mollusks, is mostly available in the form of pellets. Depending on the fish species, these pellets vary in size. Additionally, depending on where different fish species are reared, the composition and properties of these feed pellets can be altered so that they can either fl …
- バスバーの世界市場2016-2020About Busbar A busbar is a metallic strip or bar capable of conducting electricity. It is an essential component of the electrical power grid that enables the grid to conduct electricity while reducing power loss simultaneously. The materials used for busbar construction and its dimensional specifications determine the maximum current it can safely conduct. Low power rating busbars are used in ele …
- PA Resources AB：石油・ガスバリューチェーン分析レポート2013PA Resources AB Analysis Across the Oil and Gas Value Chain Report, 2013 Update Summary "PA Resources AB" Analysis Across the Oil and Gas Value Chain is an essential source for data, analysis and strategic insight into “PA Resources AB”. The report provides key information relating to oil and gas assets of the company along with its operations across the value chain. The report examines the compan …
- ミオクローヌス発作治療のグローバル臨床試験動向（2014年H2）Myoclonic Seizures Global Clinical Trials Review, H2, 2014 Summary GlobalData's clinical trial report, “Myoclonic Seizures Global Clinical Trials Review, H2, 2014" provides data on the Myoclonic Seizures clinical trial scenario. This report provides elemental information and data relating to the clinical trials on Myoclonic Seizures. It includes an overview of the trial numbers and their recruitme …
- コンテンツデリバリネットワーク（CDN）の世界市場：主要ベンダー、市場現状、展望Next generation applications, content, and commerce offerings are driving the need for flexible and cost effective Content Delivery Network (CDN) solutions, which core services such as aggregation, management, and distribution of content as well as a variety of value-added functionality. The market need for CDN solutions correlates directly with the explosion of broadband networks, smart mobile ph …
- ユーカリ油の世界市場2015Eucalyptus Oil (CAS 8000-48-4) Market Research Report 2015 aims at providing comprehensive data on Eucalyptus Oil market globally and regionally (Europe, Asia, North America, Latin America etc.) The report includes Eucalyptus Oil description, covers its application areas, manufacturing methods, patterns. It captures Eucalyptus Oil market trends, pays close attention to Eucalyptus Oil manufacturers …
- 苦味抑制剤およびフレーバーキャリアの世界市場予測（～2023年）“The bitterness suppressors and flavor carriers market is projected to grow at a CAGR of 4.9%, and the key drivers include the growing functional foods market, growth in R&D for enhanced application of encapsulation technology, and increasing applications in the pharmaceutical industry.” The bitterness suppressors and flavor carriers market is estimated at USD 192 million in 2018 and is projected …
- 世界のキセノンライト市場2015The Global Xenon Lights Industry 2015 Market Research Report is a professional and in-depth study on the current state of the Xenon Lights industry. The report provides a basic overview of the industry including definitions, classifications, applications and industry chain structure.The Xenon Lights market analysis is provided for the international markets including development trends, competitive …
- 世界の幹細胞（ステムセル）治療に関する制度動向、パイプライン分析、市場予測（～2020）The global stem cell therapy market is estimated to grow at a lower CAGR in developed countries as compared to emerging countries such as China, India, and Korea in the forecast period (2015-2020). Factors such as increasing funding from various governments and private organizations focus on stem cell research by the growing industry, and rising global awareness about stem cell therapies through v …
- ホワイトチョコレートの世界市場2019-2023About this market The rising adoption of white chocolate in diverse application sectors will continuously impact the market growth positively. Owing to factors including negligible caffeine content and smoother buttery texture compared to other variants, there has been an increasing awareness about the different applications of white chocolate among the consumers. This will eventually, foster the …
- 世界のドリルパイプ（掘削工具）市場This report estimates the drill pipe market in terms of value and volume. The value of this market is estimated in terms of $million and the volume is estimated in terms of thousand short tons. It has been further broken down into geographical regions and is further split on the basis of application and grade. Drill pipes are required for drilling activities in order to transmit the torque generat …
- 自然言語処理（NLP）の世界市場2015-2019About NLP Natural language processing is a computer application that can understand human language. It is a part of AI. It is a computerized technique in which human communication is analyzed and interpreted based on a set of theories and technologies by the computer. It is both based on text and speech provided to the computer. Currently, NLP approaches are based on machine learning that uses pat …
