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Gaucher Disease - Pipeline Review, H2 2016

Published By :

Global Markets Direct

Published Date : Dec 2016

Category :

Pharmaceutical

No. of Pages : 77 Pages

Gaucher Disease - Pipeline Review, H2 2016

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Gaucher Disease Pipeline Review, H2 2016, provides an overview of the Gaucher Disease (Genetic Disorders) pipeline landscape.

Gaucher disease is a rare genetic disorder occurs due to lacks an enzyme called glucocerebrosidase. Symptoms include cognitive impairment, enlarged spleen, severe swelling, lung disease, fatigue and seizures. Treatment includes enzyme replacement therapy.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Gaucher Disease Pipeline Review, H2 2016, provides comprehensive information on the therapeutics under development for Gaucher Disease (Genetic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Gaucher Disease (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Gaucher Disease and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase II, Phase I, Preclinical and Discovery stages are 4, 2, 9 and 1 respectively. Similarly, the Universities portfolio in Preclinical and Discovery stages comprises 1 and 5 molecules, respectively.

Gaucher Disease (Genetic Disorders) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Global Markets Directs proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope

- The pipeline guide provides a snapshot of the global therapeutic landscape of Gaucher Disease (Genetic Disorders).
- The pipeline guide reviews pipeline therapeutics for Gaucher Disease (Genetic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
- The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
- The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
- The pipeline guide reviews key companies involved in Gaucher Disease (Genetic Disorders) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Gaucher Disease (Genetic Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
- The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
- The pipeline guide reviews latest news related to pipeline therapeutics for Gaucher Disease (Genetic Disorders)

Reasons to buy

- Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
- Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
- Find and recognize significant and varied types of therapeutics under development for Gaucher Disease (Genetic Disorders).
- Classify potential new clients or partners in the target demographic.
- Develop tactical initiatives by understanding the focus areas of leading companies.
- Plan mergers and acquisitions meritoriously by identifying key players and its most promising pipeline therapeutics.
- Formulate corrective measures for pipeline projects by understanding Gaucher Disease (Genetic Disorders) 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.
- Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.
Table of Contents
Table of Contents 2
List of Tables 5
List of Figures 6
Introduction 7
Global Markets Direct Report Coverage 7
Gaucher Disease Overview 8
Therapeutics Development 9
Pipeline Products for Gaucher Disease - Overview 9
Pipeline Products for Gaucher Disease - Comparative Analysis 10
Gaucher Disease - Therapeutics under Development by Companies 11
Gaucher Disease - Therapeutics under Investigation by Universities/Institutes 12
Gaucher Disease - Pipeline Products Glance 13
Clinical Stage Products 13
Early Stage Products 14
Gaucher Disease - Products under Development by Companies 15
Gaucher Disease - Products under Investigation by Universities/Institutes 16
Gaucher Disease - Companies Involved in Therapeutics Development 17
Actelion Ltd 17
Genzyme Corp 18
greenovation Biotech GmbH 19
JCR Pharmaceuticals Co Ltd 20
Lixte Biotechnology Holdings Inc 21
Lysosomal Therapeutics Inc 22
Neuraltus Pharmaceuticals Inc 23
Pharming Group NV 24
Protalix BioTherapeutics Inc 25
Sangamo BioSciences Inc 26
Shire Plc 27
The International Biotechnology Center (IBC) Generium 28
Gaucher Disease - Therapeutics Assessment 29
Assessment by Monotherapy Products 29
Assessment by Target 30
Assessment by Mechanism of Action 32
Assessment by Route of Administration 34
Assessment by Molecule Type 36
Drug Profiles 38
ADN-LYS - Drug Profile 38
Product Description 38
Mechanism Of Action 38
R&D Progress 38
ambroxol - Drug Profile 39
Product Description 39
Mechanism Of Action 39
R&D Progress 39
Gene Therapy for Gaucher Disease - Drug Profile 40
Product Description 40
Mechanism Of Action 40
R&D Progress 40
ibiglustat - Drug Profile 41
Product Description 41
Mechanism Of Action 41
R&D Progress 41
imiglucerase biosimilar - Drug Profile 43
Product Description 43
Mechanism Of Action 43
R&D Progress 43
LB-201 - Drug Profile 44
Product Description 44
Mechanism Of Action 44
R&D Progress 44
LB-205 - Drug Profile 45
Product Description 45
Mechanism Of Action 45
R&D Progress 45
miglustat - Drug Profile 46
Product Description 46
Mechanism Of Action 46
R&D Progress 46
MOSS-GBA - Drug Profile 48
Product Description 48
Mechanism Of Action 48
R&D Progress 48
NCGC-607 - Drug Profile 49
Product Description 49
Mechanism Of Action 49
R&D Progress 49
NP-003 - Drug Profile 50
Product Description 50
Mechanism Of Action 50
R&D Progress 50
PRX-112 - Drug Profile 51
Product Description 51
Mechanism Of Action 51
R&D Progress 51
Recombinant Beta Glucosidase 2 Replacement for Gaucher's Disease and Male Infertility - Drug Profile 53
Product Description 53
Mechanism Of Action 53
R&D Progress 53
Recombinant Beta-Cerebrosidase Replacement for Gaucher's Disease - Drug Profile 54
Product Description 54
Mechanism Of Action 54
R&D Progress 54
Recombinant Protein for Type I and Type III Gaucher's Disease - Drug Profile 55
Product Description 55
Mechanism Of Action 55
R&D Progress 55
SBLSD-3 - Drug Profile 56
Product Description 56
Mechanism Of Action 56
R&D Progress 56
Small Molecule to Activate Beta Galactosidase for Gaucher Disease and GM1 Gangliosidosis - Drug Profile 57
Product Description 57
Mechanism Of Action 57
R&D Progress 57
Small Molecule to Activate Glucocerebrosidase for Parkinson's, Gaucher's and Neurodegenerative Diseases - Drug Profile 58
Product Description 58
Mechanism Of Action 58
R&D Progress 58
Small Molecules to Inhibit Glucocerebrosidase for Gaucher Disease - Drug Profile 59
Product Description 59
Mechanism Of Action 59
R&D Progress 59
Small Molecules to Inhibit Glucosidase for Diabetes and Gaucher's Disease - Drug Profile 60
Product Description 60
Mechanism Of Action 60
R&D Progress 60
taliglucerase alfa - Drug Profile 61
Product Description 61
Mechanism Of Action 61
R&D Progress 61
velaglucerase alfa - Drug Profile 62
Product Description 62
Mechanism Of Action 62
R&D Progress 62
Gaucher Disease - Dormant Projects 63
Gaucher Disease - Discontinued Products 65
Gaucher Disease - Product Development Milestones 66
Featured News & Press Releases 66
Sep 02, 2014: Shires VPRIV400 units/vial launched in Japan for the improvement of symptoms of Gaucher disease 66
Jul 04, 2014: Shires VPRIV approved in Japan for the improvement of symptoms of Gaucher disease 66
Jun 18, 2014: Protalix BioTherapeutics Initiates Phase II Study With PRX-112, an Orally-Administered Enzyme Replacement Therapy for the Treatment of Gaucher Disease 67
Mar 14, 2014: Shire Pharmaceuticals Initiates Voluntary Nationwide Recall Of One Batch, Packaged into Three Lots, of VPRIV (velaglucerase alfa for injection) 67
Feb 14, 2014: Shire Announces FDA Approval of Manufacturing Facility for VPRIV (Velaglucerase Alfa for Injection) Drug Substance 68
Feb 12, 2014: Protalix BioTherapeutics Announces Oral GCD Data to be Presented at WORLD Symposium 2014 69
Oct 14, 2013: Protalix BioTherapeutics Announces Positive Phase I Clinical Trial Results for Oral GCD in Gaucher Disease Patients 70
Apr 02, 2013: Protalix BioTherapeutics Treats First Gaucher Patient In Phase I Study With PRX-112 70
Mar 07, 2013: Protalix BioTherapeutics Receives Approval From Israeli Ministry Of Health To Initiate Phase I Clinical Trial Of PRX-112 For Treatment Of Gaucher Disease 71
Jun 28, 2012: Shires Vpriv Shows Significant Improvement In Gaucher-Related Bone Disease 72
Jun 28, 2012: Shires Vpriv Shows Significant Improvement In Gaucher-Related Bone Disease 72
Feb 22, 2012: Shire Announces European Approval Of Manufacturing Facility For VPRIV 73
Dec 19, 2011: Lixte's LB-205 Shows Therapeutic Potential For Gaucher Disease 74
Nov 22, 2011: Shire Announces Regulatory Filings In US And EU For New Manufacturing Facility 74
Mar 21, 2011: Shire Presents Data For VPRIV In Gaucher Disease At 2011 ACMG Annual Meeting 75
Appendix 76
Methodology 76
Coverage 76
Secondary Research 76
Primary Research 76
Expert Panel Validation 76
Contact Us 76
Disclaimer 77

List of Tables
Number of Products under Development for Gaucher Disease, H2 2016 9
Number of Products under Development for Gaucher Disease - Comparative Analysis, H2 2016 10
Number of Products under Development by Companies, H2 2016 11
Number of Products under Investigation by Universities/Institutes, H2 2016 12
Comparative Analysis by Clinical Stage Development, H2 2016 13
Comparative Analysis by Early Stage Development, H2 2016 14
Products under Development by Companies, H2 2016 15
Products under Investigation by Universities/Institutes, H2 2016 16
Gaucher Disease - Pipeline by Actelion Ltd, H2 2016 17
Gaucher Disease - Pipeline by Genzyme Corp, H2 2016 18
Gaucher Disease - Pipeline by greenovation Biotech GmbH, H2 2016 19
Gaucher Disease - Pipeline by JCR Pharmaceuticals Co Ltd, H2 2016 20
Gaucher Disease - Pipeline by Lixte Biotechnology Holdings Inc, H2 2016 21
Gaucher Disease - Pipeline by Lysosomal Therapeutics Inc, H2 2016 22
Gaucher Disease - Pipeline by Neuraltus Pharmaceuticals Inc, H2 2016 23
Gaucher Disease - Pipeline by Pharming Group NV, H2 2016 24
Gaucher Disease - Pipeline by Protalix BioTherapeutics Inc, H2 2016 25
Gaucher Disease - Pipeline by Sangamo BioSciences Inc, H2 2016 26
Gaucher Disease - Pipeline by Shire Plc, H2 2016 27
Gaucher Disease - Pipeline by The International Biotechnology Center (IBC) Generium, H2 2016 28
Assessment by Monotherapy Products, H2 2016 29
Number of Products by Stage and Target, H2 2016 31
Number of Products by Stage and Mechanism of Action, H2 2016 33
Number of Products by Stage and Route of Administration, H2 2016 35
Number of Products by Stage and Molecule Type, H2 2016 37
Gaucher Disease - Dormant Projects, H2 2016 63
Gaucher Disease - Dormant Projects (Contd..1), H2 2016 64
Gaucher Disease - Discontinued Products, H2 2016 65

List of Figures
Number of Products under Development for Gaucher Disease, H2 2016 9
Number of Products under Development for Gaucher Disease - Comparative Analysis, H2 2016 10
Number of Products under Development by Companies, H2 2016 11
Number of Products under Investigation by Universities/Institutes, H2 2016 12
Comparative Analysis by Clinical Stage Development, H2 2016 13
Comparative Analysis by Early Stage Products, H2 2016 14
Assessment by Monotherapy Products, H2 2016 29
Number of Products by Targets, H2 2016 30
Number of Products by Stage and Targets, H2 2016 30
Number of Products by Top 10 Mechanism of Actions, H2 2016 32
Number of Products by Stage and Top 10 Mechanism of Actions, H2 2016 32
Number of Products by Routes of Administration, H2 2016 34
Number of Products by Stage and Routes of Administration, H2 2016 34
Number of Products by Molecule Types, H2 2016 36
Number of Products by Stage and Molecule Types, H2 2016 36

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