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Lysosomal Storage Disorder Pipeline Review, H1 2013

Published By :

Global Markets Direct

Published Date : Apr 2013

Category :

Metabolic Disorders

No. of Pages : 76 Pages


Global Markets Directs, \'Lysosomal Storage Disorder Pipeline Review, H1 2013\', provides an overview of the indications therapeutic pipeline. This report provides information on the therapeutic development for Lysosomal Storage Disorder, complete with latest updates, and special features on late-stage and discontinued projects. It also reviews key players involved in the therapeutic development for Lysosomal Storage Disorder. 

Lysosomal Storage Disorder Pipeline Review, Half Year 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.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease.

Scope

  • A snapshot of the global therapeutic scenario for Lysosomal Storage Disorder.
  • 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.
  • Coverage of products based on various stages of development ranging from discovery till registration stages.
  • A feature on pipeline projects on the basis of monotherapy and combined therapeutics.
  • Coverage of the Lysosomal Storage Disorder pipeline on the basis of route of administration and molecule type.
  • Key discontinued pipeline projects.
  • Latest news and deals relating to the products.

Reasons to buy

  • Identify and understand important and diverse types of therapeutics under development for Lysosomal Storage Disorder.
  • Identify emerging players with potentially strong product portfolio and design effective counter-strategies to gain competitive advantage.
  • Plan mergers and acquisitions effectively by identifying 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 Contents

Table of Contents 2

List of Tables 6
List of Figures 6
Introduction 7
Global Markets Direct Report Coverage 7
Lysosomal Storage Disorder Overview 8
Therapeutics Development 9
An Overview of Pipeline Products for Lysosomal Storage Disorder 9
Lysosomal Storage Disorder Therapeutics under Development by Companies 11
Lysosomal Storage Disorder Therapeutics under Investigation by Universities/Institutes 13
Late Stage Products 14
Comparative Analysis 14
Mid Clinical Stage Products 15
Comparative Analysis 15
Early Clinical Stage Products 16
Comparative Analysis 16
Discovery and Pre-Clinical Stage Products 17
Comparative Analysis 17
Lysosomal Storage Disorder Therapeutics Products under Development by Companies 18
Lysosomal Storage Disorder Therapeutics Products under Investigation by Universities/Institutes 20
Companies Involved in Lysosomal Storage Disorder Therapeutics Development 21
Sangamo BioSciences, Inc. 21
BioMarin Pharmaceutical Inc. 22
Neuralstem, Inc. 23
Amicus Therapeutics, Inc. 24
Cytomedix, Inc. 25
AngioChem Inc. 26
Zymenex A/S 27
Synageva BioPharma Corp. 28
REGiMMUNE Corporation 29
Lysosomal Storage Disorder Therapeutics Assessment 30
Assessment by Monotherapy Products 30
Assessment by Route of Administration 31
Assessment by Molecule Type 33
Drug Profiles 35
sebelipase alfa - Drug Profile 35
Product Description 35
Mechanism of Action 35
R&D Progress 35
ALD-601 - Drug Profile 37
Product Description 37
Mechanism of Action 37
R&D Progress 37
NSI-566RSC - Drug Profile 38
Product Description 38
Mechanism of Action 38
R&D Progress 38
Orph-001 - Drug Profile 40
Product Description 40
Mechanism of Action 40
R&D Progress 40
UX-003 - Drug Profile 41
Product Description 41
Mechanism of Action 41
R&D Progress 41
UX-002 - Drug Profile 42
Product Description 42
Mechanism of Action 42
R&D Progress 42
UX-004 - Drug Profile 43
Product Description 43
Mechanism of Action 43
R&D Progress 43
EpiC-Enzymes - Drug Profile 44
Product Description 44
Mechanism of Action 44
R&D Progress 44
Drug For Lysosomal Storage Disorder - Drug Profile 46
Product Description 46
Mechanism of Action 46
R&D Progress 46
ZA-011 - Drug Profile 47
Product Description 47
Mechanism of Action 47
R&D Progress 47
Drug for Lysosomal Storage Diseases - Drug Profile 48
Product Description 48
Mechanism of Action 48
R&D Progress 48
Drug For Other Glycan Based-Lysosomal Storage Diseases - Drug Profile 50
Product Description 50
Mechanism of Action 50
R&D Progress 50
Drug For LSD2 Disease - Drug Profile 51
Product Description 51
Mechanism of Action 51
R&D Progress 51
Drug For LSD3 Disease - Drug Profile 52
Product Description 52
Mechanism of Action 52
R&D Progress 52
Drug For LSD4 Disease - Drug Profile 53
Product Description 53
Mechanism of Action 53
R&D Progress 53
Drug For LSD5 Disease - Drug Profile 54
Product Description 54
Mechanism of Action 54
R&D Progress 54
bimoclomol - Drug Profile 55
Product Description 55
Mechanism of Action 55
R&D Progress 55
RGI-5000 - Drug Profile 56
Product Description 56
Mechanism of Action 56
R&D Progress 56
ML-SA1 - Drug Profile 57
Product Description 57
Mechanism of Action 57
R&D Progress 57
Enzyme Peptide Conjugates - Drug Profile 58
Product Description 58
Mechanism of Action 58
R&D Progress 58
Chaperone-ERT Combinations - Drug Profile 59
Product Description 59
Mechanism of Action 59
R&D Progress 59
Lysosomal Storage Disorder Therapeutics Drug Profile Updates 60
Lysosomal Storage Disorder Therapeutics Discontinued Products 68
Lysosomal Storage Disorder Product Development Milestones 69
Featured News & Press Releases 69
Nov 10, 2012: Synageva BioPharma Announces Data From Extension Study Of Sebelipase Alfa In Late Onset Lysosomal Acid Lipase Deficiency At AASLD Annual Meeting 69
Aug 15, 2012: Orphazyme Receives European Patent Covering Use Of HSP70 For Treatment Of Lysosomal Storage Diseases 70
Jun 21, 2012: Synageva BioPharma To Present SBC-102 Data At Upcoming SSIEM Meeting 70
Feb 09, 2012: Synageva BioPharma Announces Encouraging Interim Data From Phase I/II Trial Of SBC-102 At 8th Annual Lysosomal Disease Network World Symposium 71
Jan 30, 2012: Synageva To Present Phase I/II Clinical Data Of SBC-102 At Lysosomal Disease Network World Symposium 72
Dec 22, 2011: Synageva BioPharma Completes Enrollment In Phase I/II Clinical Trial Of SBC-102 For Late Onset LAL Deficiency 72
Jun 23, 2011: Synageva Receives FDA Fast Track Designation For SBC-102 For Lysosomal Acid Lipase Deficiency 73
Feb 01, 2011: BioMarin Initiates Pivotal Phase III Trial For GALNS For Treatment Of MPS IVA 73
Feb 01, 2011: Synageva Biopharma Announced To Present Preclinical Data On Lysosomal Acid Lipase (LAL) Deficiency At The Lysosomal Disease Networks 2011 World Symposium 74
Jan 19, 2011: BioMarin Initiates Phase I/II Trial For BMN 701 For Treatment Of Pompe Disease 74

Appendix 75
Methodology 75
Coverage 75
Secondary Research 75
Primary Research 75
Expert Panel Validation 75
Contact Us 76
Disclaimer 76

List of Table


Number of Products Under Development for Lysosomal Storage Disorder, H1 2013 9
Products under Development for Lysosomal Storage Disorder Comparative Analysis, H1 2013 10
Number of Products under Development by Companies, H1 2013 12
Number of Products under Investigation by Universities/Institutes, H1 2013 13
Comparative Analysis by Late Stage Development, H1 2013 14
Comparative Analysis by Mid Clinical Stage Development, H1 2013 15
Comparative Analysis by Early Clinical Stage Development, H1 2013 16
Comparative Analysis by Discovery and Pre-Clinical Stage Development, H1 2013 17
Products under Development by Companies, H1 2013 18
Products under Development by Companies, H1 2013 (Contd..1) 19
Products under Investigation by Universities/Institutes, H1 2013 20
Sangamo BioSciences, Inc., H1 2013 21
BioMarin Pharmaceutical Inc., H1 2013 22
Neuralstem, Inc., H1 2013 23
Amicus Therapeutics, Inc., H1 2013 24
Cytomedix, Inc., H1 2013 25
AngioChem Inc., H1 2013 26
Zymenex A/S, H1 2013 27
Synageva BioPharma Corp., H1 2013 28
REGiMMUNE Corporation, H1 2013 29
Assessment by Monotherapy Products, H1 2013 30
Assessment by Stage and Route of Administration, H1 2013 32
Assessment by Stage and Molecule Type, H1 2013 34
Lysosomal Storage Disorder Therapeutics Drug Profile Updates 60
Lysosomal Storage Disorder Therapeutics Discontinued Products 68

List of Chart


Number of Products under Development for Lysosomal Storage Disorder, H1 2013 9
Products under Development for Lysosomal Storage Disorder Comparative Analysis, H1 2013 10
Products under Development by Companies, H1 2013 11
Products under Investigation by Universities/Institutes, H1 2013 13
Late Stage Products, H1 2013 14
Mid Clinical Stage Products, H1 2013 15
Early Clinical Stage Products, H1 2013 16
Discovery and Pre-Clinical Stage Products, H1 2013 17
Assessment by Monotherapy Products, H1 2013 30
Assessment by Route of Administration, H1 2013 31
Assessment by Stage and Route of Administration, H1 2013 32
Assessment by Molecule Type, H1 2013 33
Assessment by Stage and Molecule Type, H1 2013 34

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