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Lysosomal Storage Disorder - Pipeline Review, H1 2014

Published By :

Global Markets Direct

Published Date : Apr 2014

Category :

Metabolic Disorders

No. of Pages : 105 Pages

Lysosomal Storage Disorder Pipeline Review, H1 2014, provides an overview of the Lysosomal Storage Disorders therapeutic pipeline.

This report provides comprehensive information on the therapeutic development for Lysosomal Storage Disorder, complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type, along with latest updates, and featured news and press releases. It also reviews key players involved in the therapeutic development for Lysosomal Storage Disorder and special features on late-stage and discontinued projects.

Global Markets Directs report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report is built using data and information sourced from Global Markets Directs proprietary databases, Company/University websites, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together by Global Markets Directs team. Drug profiles/records featured in the report undergoes periodic updation following a stringent set of processes that ensures that all the profiles are updated with the latest set of information. Additionally, processes including live news & deals tracking, browser based alert-box and clinical trials registries tracking ensure that the most recent developments are captured on a real time basis.

The report enhances decision making capabilities and help to create effective counter strategies to gain competitive advantage. It strengthens R&D pipelines by identifying new targets and MOAs to produce first-in-class and best-in-class products.

Scope

  • The report provides a snapshot of the global therapeutic landscape of Lysosomal Storage Disorder
  • The report reviews key pipeline products under drug profile section which includes, product description, MoA and R&D brief, licensing and collaboration details & other developmental activities 
  • The report reviews key players involved in the therapeutics development for Lysosomal Storage Disorder and enlists all their major and minor projects
  • The report summarizes all the dormant and discontinued pipeline projects 
  • A review of the Lysosomal Storage Disorder products under development by companies and universities/research institutes based on information derived from company and industry-specific sources
  • Pipeline products coverage based on various stages of development ranging from pre-registration till discovery and undisclosed stages
  • A detailed assessment of monotherapy and combination therapy pipeline projects
  • Coverage of the Lysosomal Storage Disorder pipeline on the basis of target, MoA, route of administration and molecule type
  • Latest news and deals relating related to pipeline products

Reasons to buy

  • Provides strategically significant competitor information, analysis, and insights to formulate effective R&D development strategies
  • Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
  • Develop strategic initiatives by understanding the focus areas of leading companies
  • Identify and understand important and diverse types of therapeutics under development for Lysosomal Storage Disorder
  • Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline
  • Devise corrective measures for pipeline projects by understanding Lysosomal Storage Disorder pipeline depth and focus of Indication therapeutics
  • Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope
  • Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline
Table of Content

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 15
Lysosomal Storage Disorder - Pipeline Products Glance 16
Late Stage Products 16
Clinical Stage Products 17
Early Stage Products 18
Lysosomal Storage Disorder - Products under Development by Companies 19
Lysosomal Storage Disorder - Products under Investigation by Universities/Institutes 20
Lysosomal Storage Disorder - Companies Involved in Therapeutics Development 21
Sangamo BioSciences, Inc. 21
Neuralstem, Inc. 22
Amicus Therapeutics, Inc. 23
Cytomedix, Inc. 24
AngioChem Inc. 25
Zymenex A/S 26
Synageva BioPharma Corp. 27
Fate Therapeutics, Inc. 28
to-BBB technologies BV 29
REGiMMUNE Corporation 30
biOasis Technologies Inc. 31
ArmaGen Technologies, Inc. 32
Orphazyme ApS 33
Oxyrane Belgium NV 34
Ultragenyx Pharmaceutical Inc. 35
Minoryx Therapeutics s.l. 36
Lysosomal Storage Disorder - Therapeutics Assessment 37
Assessment by Monotherapy Products 37
Assessment by Combination Products 38
Assessment by Target 39
Assessment by Mechanism of Action 41
Assessment by Route of Administration 43
Assessment by Molecule Type 45
Drug Profiles 47
sebelipase alfa - Drug Profile 47
Product Description 47
Mechanism of Action 47
R&D Progress 47
ALD-601 - Drug Profile 49
Product Description 49
Mechanism of Action 49
R&D Progress 49
NSI-566 - Drug Profile 50
Product Description 50
Mechanism of Action 50
R&D Progress 50
Orph-001 - Drug Profile 52
Product Description 52
Mechanism of Action 52
R&D Progress 52
FT-1050 - Drug Profile 53
Product Description 53
Mechanism of Action 53
R&D Progress 53
UX-002 - Drug Profile 55
Product Description 55
Mechanism of Action 55
R&D Progress 55
UX-004 - Drug Profile 56
Product Description 56
Mechanism of Action 56
R&D Progress 56
2B3-201 - Drug Profile 57
Product Description 57
Mechanism of Action 57
R&D Progress 57
Recombinant Enzyme For Lysosomal Storage Disorder - Drug Profile 58
Product Description 58
Mechanism of Action 58
R&D Progress 58
ZFP Transcription Factors for Lysosomal Storage Diseases - Drug Profile 59
Product Description 59
Mechanism of Action 59
R&D Progress 59
RGI-5000 - Drug Profile 60
Product Description 60
Mechanism of Action 60
R&D Progress 60
Chaperone-ERT Combinations - Drug Profile 61
Product Description 61
Mechanism of Action 61
R&D Progress 61
Recombinant Human Acid Ceramidase - Drug Profile 62
Product Description 62
Mechanism of Action 62
R&D Progress 62
Second Generation HSC - Drug Profile 63
Product Description 63
Mechanism of Action 63
R&D Progress 63
ZA-011 - Drug Profile 64
Product Description 64
Mechanism of Action 64
R&D Progress 64
Drug For LSD-2 Disease - Drug Profile 65
Product Description 65
Mechanism of Action 65
R&D Progress 65
Drug For LSD-3 Disease - Drug Profile 66
Product Description 66
Mechanism of Action 66
R&D Progress 66
Drug For LSD-4 Disease - Drug Profile 67
Product Description 67
Mechanism of Action 67
R&D Progress 67
Drug For LSD-5 Disease - Drug Profile 68
Product Description 68
Mechanism of Action 68
R&D Progress 68
ML-SA1 - Drug Profile 69
Product Description 69
Mechanism of Action 69
R&D Progress 69
Enzyme Peptide Conjugates - Drug Profile 70
Product Description 70
Mechanism of Action 70
R&D Progress 70
Cyclodextrins + Delta-Tocopherol - Drug Profile 71
Product Description 71
Mechanism of Action 71
R&D Progress 71
AGT-184 - Drug Profile 72
Product Description 72
Mechanism of Action 72
R&D Progress 72
Enzyme For Lysosomal Storage Disorder - Drug Profile 73
Product Description 73
Mechanism of Action 73
R&D Progress 73
Therapy For GM1-gangliosidosis And Morquio B - Drug Profile 74
Product Description 74
Mechanism of Action 74
R&D Progress 74
Lysosomal Storage Disorder - Recent Pipeline Updates 75
Lysosomal Storage Disorder - Dormant Projects 91
Lysosomal Storage Disorder - Product Development Milestones 92
Featured News & Press Releases 92
Mar 04, 2014: Synageva BioPharma Issued U.S. Patent for Treatment of LAL Deficiency 92
Feb 26, 2014: Fate Therapeutics Announces Observed Effects of Pharmacologic Modulation on T Cell Compartment From Its Phase 1b Study of PROHEMA 92
Feb 13, 2014: Synageva BioPharma Highlights Sebelipase Alfa and LAL Deficiency Data at the Lysosomal Disease Network WORLD Symposium 94
Jan 29, 2014: Synageva BioPharma Announces Data Presentations at the 10th Annual Lysosomal Disease Network World Symposium 95
Jan 06, 2014: Synageva Biopharma Provides Clinical Trial Updates with Sebelipase ALFA in LAL Deficiency 95
Dec 02, 2013: Synageva BioPharma Completes Target Enrollment in Phase 3 ARISE Trial with Sebelipase Alfa in Children and Adults with LAL Deficiency 97
Oct 21, 2013: For first time, drug developed based on zebrafish studies passes Phase I clinical trial 98
Oct 11, 2013: Synageva BioPharma Highlights Data at the NASPGHAN Meeting 99
Sep 04, 2013: Synageva BioPharma Highlights Data at the International Congress of Inborn Errors of Metabolism 101
May 20, 2013: Synageva\'s Sebelipase Alfa Receives Breakthrough Therapy Designation For Early Onset LAL Deficiency From FDA 103

Appendix 104
Methodology 104
Coverage 104
Secondary Research 104
Primary Research 104
Expert Panel Validation 104
Contact Us 105
Disclaimer 105

List of Table


Number of Products under Development for Lysosomal Storage Disorder, H1 2014 10
Number of Products under Development for Lysosomal Storage Disorder - Comparative Analysis, H1 2014 11
Number of Products under Development by Companies, H1 2014 13
Number of Products under Development by Companies, H1 2014 (Contd..1) 14
Number of Products under Investigation by Universities/Institutes, H1 2014 15
Comparative Analysis by Late Stage Development, H1 2014 16
Comparative Analysis by Clinical Stage Development, H1 2014 17
Comparative Analysis by Early Stage Development, H1 2014 18
Products under Development by Companies, H1 2014 19
Products under Investigation by Universities/Institutes, H1 2014 20
Lysosomal Storage Disorder - Pipeline by Sangamo BioSciences, Inc., H1 2014 21
Lysosomal Storage Disorder - Pipeline by Neuralstem, Inc., H1 2014 22
Lysosomal Storage Disorder - Pipeline by Amicus Therapeutics, Inc., H1 2014 23
Lysosomal Storage Disorder - Pipeline by Cytomedix, Inc., H1 2014 24
Lysosomal Storage Disorder - Pipeline by AngioChem Inc., H1 2014 25
Lysosomal Storage Disorder - Pipeline by Zymenex A/S, H1 2014 26
Lysosomal Storage Disorder - Pipeline by Synageva BioPharma Corp., H1 2014 27
Lysosomal Storage Disorder - Pipeline by Fate Therapeutics, Inc., H1 2014 28
Lysosomal Storage Disorder - Pipeline by to-BBB technologies BV, H1 2014 29
Lysosomal Storage Disorder - Pipeline by REGiMMUNE Corporation, H1 2014 30
Lysosomal Storage Disorder - Pipeline by biOasis Technologies Inc., H1 2014 31
Lysosomal Storage Disorder - Pipeline by ArmaGen Technologies, Inc., H1 2014 32
Lysosomal Storage Disorder - Pipeline by Orphazyme ApS, H1 2014 33
Lysosomal Storage Disorder - Pipeline by Oxyrane Belgium NV, H1 2014 34
Lysosomal Storage Disorder - Pipeline by Ultragenyx Pharmaceutical Inc., H1 2014 35
Lysosomal Storage Disorder - Pipeline by Minoryx Therapeutics s.l., H1 2014 36
Assessment by Monotherapy Products, H1 2014 37
Assessment by Combination Products, H1 2014 38
Number of Products by Stage and Target, H1 2014 40
Number of Products by Stage and Mechanism of Action, H1 2014 42
Number of Products by Stage and Route of Administration, H1 2014 44
Number of Products by Stage and Molecule Type, H1 2014 46
Lysosomal Storage Disorder Therapeutics - Recent Pipeline Updates, H1 2014 75
Lysosomal Storage Disorder - Dormant Projects, H1 2014 91

List of Chart


Number of Products under Development for Lysosomal Storage Disorder, H1 2014 10
Number of Products under Development for Lysosomal Storage Disorder - Comparative Analysis, H1 2014 11
Number of Products under Development by Companies, H1 2014 12
Comparative Analysis by Early Stage Products, H1 2014 18
Assessment by Monotherapy Products, H1 2014 37
Number of Products by Top 10 Target, H1 2014 39
Number of Products by Stage and Top 10 Target, H1 2014 40
Number of Products by Top 10 Mechanism of Action, H1 2014 41
Number of Products by Stage and Top 10 Mechanism of Action, H1 2014 42
Number of Products by Top 10 Route of Administration, H1 2014 43
Number of Products by Stage and Top 10 Route of Administration, H1 2014 44
Number of Products by Top 10 Molecule Type, H1 2014 45
Number of Products by Stage and Top 10 Molecule Type, H1 2014 46

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