Lysosomal Storage Disorder - Pipeline Review, H1 2015

01:13 EDT 15 Apr 2015 | BioPortfolio Reports

Recently added to the BioPortfolio report store, Lysosomal Storage Disorder - Pipeline Review, H1 2015 is a new report from Global Markets Direct published on 2015-03-10. This 105-page report is available in PDF from $2000.

Lysosomal Storage Disorder - Pipeline Review, H1 2015


Global Markets Direct’s, ‘Lysosomal Storage Disorder - Pipeline Review, H1 2015’, 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.


- 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

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 For more information open Lysosomal Storage Disorder - Pipeline Review, H1 2015.


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