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Published: Nov, 2014 | Pages:
74 | Publisher: Global Markets Direct
Industry: Healthcare | Report Format: Electronic (PDF)
Gaucher's Disease - Pipeline Review, H2 2014 Summary Global Markets Direct's, 'Gaucher's Disease - Pipeline Review, H2 2014', provides an overview of the Gaucher's Disease's therapeutic pipeline. This report provides comprehensive information on the therapeutic development for Gaucher's Disease, 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 Gaucher's Disease 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 Gaucher's Disease - 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 Gaucher's Disease and enlists all their major and minor projects - The report summarizes all the dormant and discontinued pipeline projects - A review of the Gaucher's Disease 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 Gaucher's Disease 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 Gaucher's Disease - Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline - Devise corrective measures for pipeline projects by understanding Gaucher's Disease 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 5 List of Figures 6 Introduction 7 Global Markets Direct Report Coverage 7 Gaucher's Disease Overview 8 Therapeutics Development 9 Pipeline Products for Gaucher's Disease - Overview 9 Pipeline Products for Gaucher's Disease - Comparative Analysis 10 Gaucher's Disease - Therapeutics under Development by Companies 11 Gaucher's Disease - Therapeutics under Investigation by Universities/Institutes 13 Gaucher's Disease - Pipeline Products Glance 14 Late Stage Products 14 Clinical Stage Products 15 Early Stage Products 16 Gaucher's Disease - Products under Development by Companies 17 Gaucher's Disease - Products under Investigation by Universities/Institutes 18 Gaucher's Disease - Companies Involved in Therapeutics Development 19 Amicus Therapeutics, Inc. 19 Biosidus S.A. 20 Dong-A Socio Group 21 ExSAR Corporation 22 Genzyme Corporation 23 greenovation Biotech GmbH 24 JCR Pharmaceuticals Co., Ltd. 25 Lixte Biotechnology Holdings, Inc. 26 Neuraltus Pharmaceuticals, Inc. 27 Pharming Group N.V. 28 Protalix BioTherapeutics, Inc. 29 UAB Profarma 30 Gaucher's Disease - Therapeutics Assessment 31 Assessment by Monotherapy Products 31 Assessment by Combination Products 32 Assessment by Target 33 Assessment by Mechanism of Action 35 Assessment by Route of Administration 37 Assessment by Molecule Type 39 Drug Profiles 41 afegostat tartrate + ERT - Drug Profile 41 Product Description 41 Mechanism of Action 41 R&D Progress 41 ambroxol - Drug Profile 42 Product Description 42 Mechanism of Action 42 R&D Progress 42 DOS-000011 - Drug Profile 44 Product Description 44 Mechanism of Action 44 R&D Progress 44 eliglustat tartrate - Drug Profile 45 Product Description 45 Mechanism of Action 45 R&D Progress 45 Gluco-Cerebosidase - Drug Profile 47 Product Description 47 Mechanism of Action 47 R&D Progress 47 imiglucerase biosimilar - Drug Profile 48 Product Description 48 Mechanism of Action 48 R&D Progress 48 imiglucerase biosimilar - Drug Profile 49 Product Description 49 Mechanism of Action 49 R&D Progress 49 imiglucerase biosimilar - Drug Profile 50 Product Description 50 Mechanism of Action 50 R&D Progress 50 JR-101 - Drug Profile 51 Product Description 51 Mechanism of Action 51 R&D Progress 51 LB-201 - Drug Profile 52 Product Description 52 Mechanism of Action 52 R&D Progress 52 LB-205 - Drug Profile 53 Product Description 53 Mechanism of Action 53 R&D Progress 53 NP-003 - Drug Profile 54 Product Description 54 Mechanism of Action 54 R&D Progress 54 PRX-112 - Drug Profile 55 Product Description 55 Mechanism of Action 55 R&D Progress 55 Recombinant Enzyme for Gaucher's Disease - Drug Profile 56 Product Description 56 Mechanism of Action 56 R&D Progress 56 Recombinant Enzyme to Replace Beta-Cerebrosidase for Gaucher's Disease - 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 Activate Glucocerebrosidase for Gaucher and Parkinson Disease - Drug Profile 59 Product Description 59 Mechanism of Action 59 R&D Progress 59 Gaucher's Disease - Recent Pipeline Updates 60 Gaucher's Disease - Dormant Projects 64 Gaucher's Disease - Product Development Milestones 65 Featured News & Press Releases 65 Sep 02, 2014: Shire's VPRIV400 units/vial launched in Japan for the improvement of symptoms of Gaucher disease 65 Jul 04, 2014: Shire's VPRIV approved in Japan for the improvement of symptoms of Gaucher disease 65 Jun 18, 2014: Protalix BioTherapeutics Initiates Phase II Study With PRX-112, an Orally-Administered Enzyme Replacement Therapy for the Treatment of Gaucher Disease 66 Feb 14, 2014: Shire Announces FDA Approval of Manufacturing Facility for VPRIV (Velaglucerase Alfa for Injection) Drug Substance 67 Feb 12, 2014: Protalix BioTherapeutics Announces Oral GCD Data to be Presented at WORLD Symposium 2014 68 Dec 11, 2013: FDA Grants Priority Review for Genzyme's Cerdelga, an Investigational Oral Therapy for Gaucher Disease 69 Oct 14, 2013: Protalix BioTherapeutics Announces Positive Phase I Clinical Trial Results for Oral GCD in Gaucher Disease Patients 69 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: Shire's Vpriv Shows Significant Improvement In Gaucher-Related Bone Disease 71 Appendix 73 Methodology 73 Coverage 73 Secondary Research 73 Primary Research 73 Expert Panel Validation 73 Contact Us 74 Disclaimer 74
List of Tables Number of Products under Development for Gaucher's Disease, H2 2014 9 Number of Products under Development for Gaucher's Disease - Comparative Analysis, H2 2014 10 Number of Products under Development by Companies, H2 2014 12 Number of Products under Investigation by Universities/Institutes, H2 2014 13 Comparative Analysis by Late Stage Development, H2 2014 14 Comparative Analysis by Clinical Stage Development, H2 2014 15 Comparative Analysis by Early Stage Development, H2 2014 16 Products under Development by Companies, H2 2014 17 Products under Investigation by Universities/Institutes, H2 2014 18 Gaucher's Disease - Pipeline by Amicus Therapeutics, Inc., H2 2014 19 Gaucher's Disease - Pipeline by Biosidus S.A., H2 2014 20 Gaucher's Disease - Pipeline by Dong-A Socio Group, H2 2014 21 Gaucher's Disease - Pipeline by ExSAR Corporation, H2 2014 22 Gaucher's Disease - Pipeline by Genzyme Corporation, H2 2014 23 Gaucher's Disease - Pipeline by greenovation Biotech GmbH, H2 2014 24 Gaucher's Disease - Pipeline by JCR Pharmaceuticals Co., Ltd., H2 2014 25 Gaucher's Disease - Pipeline by Lixte Biotechnology Holdings, Inc., H2 2014 26 Gaucher's Disease - Pipeline by Neuraltus Pharmaceuticals, Inc., H2 2014 27 Gaucher's Disease - Pipeline by Pharming Group N.V., H2 2014 28 Gaucher's Disease - Pipeline by Protalix BioTherapeutics, Inc., H2 2014 29 Gaucher's Disease - Pipeline by UAB Profarma, H2 2014 30 Assessment by Monotherapy Products, H2 2014 31 Assessment by Combination Products, H2 2014 32 Number of Products by Stage and Target, H2 2014 34 Number of Products by Stage and Mechanism of Action, H2 2014 36 Number of Products by Stage and Route of Administration, H2 2014 38 Number of Products by Stage and Molecule Type, H2 2014 40 Gaucher's Disease Therapeutics - Recent Pipeline Updates, H2 2014 60 Gaucher's Disease - Dormant Projects, H2 2014 64
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