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Lysosomal Storage Disorder-Pipeline Review, H1 2015

Lysosomal Storage Disorder-Pipeline Review, H1 2015


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Executive Summary

Lysosomal Storage Disorder-Pipeline Review, H1 2015

Summary

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.

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 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

List of Tables

Number of Products under Development for Lysosomal Storage Disorder, H1 2015 10

Number of Products under Development for Lysosomal Storage Disorder-Comparative Analysis, H1 2015 11

Number of Products under Development by Companies, H1 2015 13

Number of Products under Investigation by Universities/Institutes, H1 2015 14

Comparative Analysis by Late Stage Development, H1 2015 15

Comparative Analysis by Clinical Stage Development, H1 2015 16

Comparative Analysis by Early Stage Development, H1 2015 17

Products under Development by Companies, H1 2015 18

Products under Development by Companies, H1 2015 (Contd..1) 19

Products under Investigation by Universities/Institutes, H1 2015 20

Lysosomal Storage Disorder-Pipeline by Amicus Therapeutics, Inc., H1 2015 21

Lysosomal Storage Disorder-Pipeline by AngioChem Inc., H1 2015 22

Lysosomal Storage Disorder-Pipeline by BBB Therapeutics B.V., H1 2015 23

Lysosomal Storage Disorder-Pipeline by Chiesi Farmaceutici SpA, H1 2015 24

Lysosomal Storage Disorder-Pipeline by Fate Therapeutics, Inc., H1 2015 25

Lysosomal Storage Disorder-Pipeline by Minoryx Therapeutics s.l., H1 2015 26

Lysosomal Storage Disorder-Pipeline by Neuralstem, Inc., H1 2015 27

Lysosomal Storage Disorder-Pipeline by Nuo Therapeutics, Inc., H1 2015 28

Lysosomal Storage Disorder-Pipeline by Orphazyme ApS, H1 2015 29

Lysosomal Storage Disorder-Pipeline by Oxyrane Belgium NV, H1 2015 30

Lysosomal Storage Disorder-Pipeline by REGiMMUNE Corporation, H1 2015 31

Lysosomal Storage Disorder-Pipeline by Sagetis Biotech, S.L., H1 2015 32

Lysosomal Storage Disorder-Pipeline by Sangamo BioSciences, Inc., H1 2015 33

Lysosomal Storage Disorder-Pipeline by Synageva BioPharma Corp., H1 2015 34

Lysosomal Storage Disorder-Pipeline by Ultragenyx Pharmaceutical Inc., H1 2015 35

Assessment by Monotherapy Products, H1 2015 36

Number of Products by Stage and Target, H1 2015 38

Number of Products by Stage and Mechanism of Action, H1 2015 40

Number of Products by Stage and Route of Administration, H1 2015 42

Number of Products by Stage and Molecule Type, H1 2015 44

Lysosomal Storage Disorder Therapeutics-Recent Pipeline Updates, H1 2015 77

Lysosomal Storage Disorder-Dormant Projects, H1 2015 94

List of Figures

Number of Products under Development for Lysosomal Storage Disorder, H1 2015 10

Number of Products under Development for Lysosomal Storage Disorder-Comparative Analysis, H1 2015 11

Number of Products under Development by Companies, H1 2015 12

Number of Products under Investigation by Universities/Institutes, H1 2015 14

Comparative Analysis by Late Stage Development, H1 2015 15

Comparative Analysis by Early Stage Products, H1 2015 17

Assessment by Monotherapy Products, H1 2015 36

Number of Products by Top 10 Targets, H1 2015 37

Number of Products by Stage and Top 10 Targets, H1 2015 38

Number of Products by Top 10 Mechanism of Actions, H1 2015 39

Number of Products by Stage and Top 10 Mechanism of Actions, H1 2015 40

Number of Products by Top 10 Routes of Administration, H1 2015 41

Number of Products by Stage and Top 10 Routes of Administration, H1 2015 42

Number of Products by Top 10 Molecule Types, H1 2015 43

Number of Products by Stage and Top 10 Molecule Types, H1 2015 44

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Amicus Therapeutics, Inc.

AngioChem Inc.

BBB Therapeutics B.V.

Chiesi Farmaceutici SpA

Fate Therapeutics, Inc.

Minoryx Therapeutics s.l.

Neuralstem, Inc.

Nuo Therapeutics, Inc.

Orphazyme ApS

Oxyrane Belgium NV

REGiMMUNE Corporation

Sagetis Biotech, S.L.

Sangamo BioSciences, Inc.

Synageva BioPharma Corp.

Ultragenyx Pharmaceutical Inc.

Lysosomal Storage Disorder Therapeutic Products under Development, Key Players in Lysosomal Storage Disorder Therapeutics, Lysosomal Storage Disorder Pipeline Overview, Lysosomal Storage Disorder Pipeline, Lysosomal Storage Disorder Pipeline Assessment


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