Lysosomal Storage Disorder - Pipeline Review, H2 2014

Published: Oct, 2014 | Pages: 105 | Publisher: Global Markets Direct
Industry: Healthcare | Report Format: Electronic (PDF)

Lysosomal Storage Disorder - Pipeline Review, H2 2014

Summary

Global Markets Direct's, 'Lysosomal Storage Disorder - Pipeline Review, H2 2014', provides an overview of the Lysosomal Storage Disorder's 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 Direct's 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 Direct's 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 Direct's 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.

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

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 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 Combination Products 37
Assessment by Target 38
Assessment by Mechanism of Action 40
Assessment by Route of Administration 42
Assessment by Molecule Type 44
Drug Profiles 46
2B3-201 - Drug Profile 46
Product Description 46
Mechanism of Action 46
R&D Progress 46
ALD-601 - Drug Profile 48
Product Description 48
Mechanism of Action 48
R&D Progress 48
Chaperone-ERT Combinations - Drug Profile 49
Product Description 49
Mechanism of Action 49
R&D Progress 49
cyclodextrins + delta-tocopherol - Drug Profile 50
Product Description 50
Mechanism of Action 50
R&D Progress 50
FT-1050 - Drug Profile 51
Product Description 51
Mechanism of Action 51
R&D Progress 51
Gene Therapy for Lysosomal Storage Disease - Drug Profile 53
Product Description 53
Mechanism of Action 53
R&D Progress 53
LSD-1 - Drug Profile 54
Product Description 54
Mechanism of Action 54
R&D Progress 54
LSD-2 - Drug Profile 55
Product Description 55
Mechanism of Action 55
R&D Progress 55
ML-SA1 - Drug Profile 56
Product Description 56
Mechanism of Action 56
R&D Progress 56
NSI-566 - Drug Profile 57
Product Description 57
Mechanism of Action 57
R&D Progress 57
Orph-001 - Drug Profile 59
Product Description 59
Mechanism of Action 59
R&D Progress 59
Recombinant Enzyme for Farber Disease and Cystic Fibrosis - Drug Profile 60
Product Description 60
Mechanism of Action 60
R&D Progress 60
Recombinant Enzyme for LSD-2 Disease - Drug Profile 61
Product Description 61
Mechanism of Action 61
R&D Progress 61
Recombinant Enzyme for LSD-3 Disease - Drug Profile 62
Product Description 62
Mechanism of Action 62
R&D Progress 62
Recombinant Enzyme for LSD-4 Disease - Drug Profile 63
Product Description 63
Mechanism of Action 63
R&D Progress 63
Recombinant Enzyme for LSD-5 Disease - Drug Profile 64
Product Description 64
Mechanism of Action 64
R&D Progress 64
Recombinant Enzyme for Lysosomal Storage Disorder - Drug Profile 65
Product Description 65
Mechanism of Action 65
R&D Progress 65
Recombinant Enzyme for Lysosomal Storage Disorder - Drug Profile 66
Product Description 66
Mechanism of Action 66
R&D Progress 66
RGI-5000 - 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 Molecules for GM1-gangliosidosis And Morquio B - Drug Profile 70
Product Description 70
Mechanism of Action 70
R&D Progress 70
Small Molecules for Lysosomal Storage Disorders - Drug Profile 71
Product Description 71
Mechanism of Action 71
R&D Progress 71
Small Molecules to Activate Beta-Galactosidase for GM1 Gangliosidosis - 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 93
Lysosomal Storage Disorder - Product Development Milestones 94
Featured News & Press Releases 94
Oct 23, 2014: Synageva BioPharma Presents Sebelipase Alfa Data At The NASPGHAN Meeting 94
Oct 21, 2014: Synageva BioPharma Starts Rolling Submission Of A Biologics License Application To The FDA For Sebelipase Alfa 95
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 96
Jul 29, 2014: Fate Therapeutics Announces FDA Clearance of IND for Clinical Development of PROHEMA in Inherited Metabolic Disorders 96
Jun 30, 2014: Synageva BioPharma Reports Positive Top-Line Results From Phase 3 Study Of Sebelipase Alfa In Children And Adults With LAL Deficiency 97
Mar 04, 2014: Synageva BioPharma Issued U.S. Patent for Treatment of LAL Deficiency 98
Feb 26, 2014: Fate Therapeutics Announces Observed Effects of Pharmacologic Modulation on T Cell Compartment From Its Phase 1b Study of PROHEMA 99
Feb 13, 2014: Synageva BioPharma Highlights Sebelipase Alfa and LAL Deficiency Data at the Lysosomal Disease Network WORLD Symposium 100
Jan 29, 2014: Synageva BioPharma Announces Data Presentations at the 10th Annual Lysosomal Disease Network World Symposium 101
Jan 06, 2014: Synageva Biopharma Provides Clinical Trial Updates with Sebelipase ALFA in LAL Deficiency 101
Appendix 104
Methodology 104
Coverage 104
Secondary Research 104
Primary Research 104
Expert Panel Validation 104
Contact Us 105
Disclaimer 105

List of Tables
Number of Products under Development for Lysosomal Storage Disorder, H2 2014 10
Number of Products under Development for Lysosomal Storage Disorder - Comparative Analysis, H2 2014 11
Number of Products under Development by Companies, H2 2014 13
Number of Products under Investigation by Universities/Institutes, H2 2014 14
Comparative Analysis by Late Stage Development, H2 2014 15
Comparative Analysis by Clinical Stage Development, H2 2014 16
Comparative Analysis by Early Stage Development, H2 2014 17
Products under Development by Companies, H2 2014 18
Products under Development by Companies, H2 2014 (Contd..1) 19
Products under Investigation by Universities/Institutes, H2 2014 20
Lysosomal Storage Disorder - Pipeline by Amicus Therapeutics, Inc., H2 2014 21
Lysosomal Storage Disorder - Pipeline by AngioChem Inc., H2 2014 22
Lysosomal Storage Disorder - Pipeline by BBB Therapeutics B.V., H2 2014 23
Lysosomal Storage Disorder - Pipeline by Chiesi Farmaceutici SpA, H2 2014 24
Lysosomal Storage Disorder - Pipeline by Fate Therapeutics, Inc., H2 2014 25
Lysosomal Storage Disorder - Pipeline by Minoryx Therapeutics s.l., H2 2014 26
Lysosomal Storage Disorder - Pipeline by Neuralstem, Inc., H2 2014 27
Lysosomal Storage Disorder - Pipeline by Nuo Therapeutics, Inc., H2 2014 28
Lysosomal Storage Disorder - Pipeline by Orphazyme ApS, H2 2014 29
Lysosomal Storage Disorder - Pipeline by Oxyrane Belgium NV, H2 2014 30
Lysosomal Storage Disorder - Pipeline by REGiMMUNE Corporation, H2 2014 31
Lysosomal Storage Disorder - Pipeline by Sagetis Biotech, S.L., H2 2014 32
Lysosomal Storage Disorder - Pipeline by Sangamo BioSciences, Inc., H2 2014 33
Lysosomal Storage Disorder - Pipeline by Synageva BioPharma Corp., H2 2014 34
Lysosomal Storage Disorder - Pipeline by Ultragenyx Pharmaceutical Inc., H2 2014 35
Assessment by Monotherapy Products, H2 2014 36
Assessment by Combination Products, H2 2014 37
Number of Products by Stage and Target, H2 2014 39
Number of Products by Stage and Mechanism of Action, H2 2014 41
Number of Products by Stage and Route of Administration, H2 2014 43
Number of Products by Stage and Molecule Type, H2 2014 45
Lysosomal Storage Disorder Therapeutics - Recent Pipeline Updates, H2 2014 77
Lysosomal Storage Disorder - Dormant Projects, H2 2014 93