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Published: Jun, 2017 | Pages:
74 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)
Gaucher Disease - Pipeline Review, H1 2017 Summary Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Gaucher Disease - Pipeline Review, H1 2017, 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, H1 2017, 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, 11 and 2 respectively. Similarly, the Universities portfolio in Phase I, Preclinical and Discovery stages comprises 1, 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 Direct's 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 it's 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
List of Tables List of Figures Introduction Global Markets Direct Report Coverage Gaucher Disease - Overview Gaucher Disease - Therapeutics Development Pipeline Overview Pipeline by Companies Pipeline by Universities/Institutes Products under Development by Companies Products under Development by Universities/Institutes Gaucher Disease - Therapeutics Assessment Assessment by Target Assessment by Mechanism of Action Assessment by Route of Administration Assessment by Molecule Type Gaucher Disease - Companies Involved in Therapeutics Development Actelion Ltd Genzyme Corp greenovation Biotech GmbH JCR Pharmaceuticals Co Ltd Lixte Biotechnology Holdings Inc Neuraltus Pharmaceuticals Inc Orphazyme ApS Pharming Group NV Protalix BioTherapeutics Inc Shire Plc Gaucher Disease - Drug Profiles ADN-LYS - Drug Profile Product Description Mechanism Of Action R&D Progress ambroxol - Drug Profile Product Description Mechanism Of Action R&D Progress arimoclomol - Drug Profile Product Description Mechanism Of Action R&D Progress AVRRD-02 - Drug Profile Product Description Mechanism Of Action R&D Progress Gene Therapy for Gaucher Disease - Drug Profile Product Description Mechanism Of Action R&D Progress Gene Therapy for Gaucher Disease - Drug Profile Product Description Mechanism Of Action R&D Progress ibiglustat - Drug Profile Product Description Mechanism Of Action R&D Progress imiglucerase biosimilar - Drug Profile Product Description Mechanism Of Action R&D Progress LB-201 - Drug Profile Product Description Mechanism Of Action R&D Progress LB-205 - Drug Profile Product Description Mechanism Of Action R&D Progress LTI-291 - Drug Profile Product Description Mechanism Of Action R&D Progress miglustat - Drug Profile Product Description Mechanism Of Action R&D Progress MOSS-GBA - Drug Profile Product Description Mechanism Of Action R&D Progress NCGC-607 - Drug Profile Product Description Mechanism Of Action R&D Progress NP-003 - Drug Profile Product Description Mechanism Of Action R&D Progress Pcgin - Drug Profile Product Description Mechanism Of Action R&D Progress PRX-112 - Drug Profile Product Description Mechanism Of Action R&D Progress Recombinant Beta Glucosidase 2 Replacement for Gaucher's Disease and Male Infertility - Drug Profile Product Description Mechanism Of Action R&D Progress Recombinant Beta-Cerebrosidase Replacement for Gaucher's Disease - Drug Profile Product Description Mechanism Of Action R&D Progress Recombinant Protein for Type I and Type III Gaucher's Disease - Drug Profile Product Description Mechanism Of Action R&D Progress RND-001 - Drug Profile Product Description Mechanism Of Action R&D Progress Small Molecule to Activate Beta Galactosidase for Gaucher Disease and GM1 Gangliosidosis - Drug Profile Product Description Mechanism Of Action R&D Progress Small Molecules to Inhibit Glucocerebrosidase for Gaucher Disease - Drug Profile Product Description Mechanism Of Action R&D Progress Small Molecules to Inhibit Glucosidase for Diabetes and Gaucher's Disease - Drug Profile Product Description Mechanism Of Action R&D Progress taliglucerase alfa - Drug Profile Product Description Mechanism Of Action R&D Progress velaglucerase alfa - Drug Profile Product Description Mechanism Of Action R&D Progress Gaucher Disease - Dormant Projects Gaucher Disease - Discontinued Products Gaucher Disease - Product Development Milestones Featured News & Press Releases Mar 22, 2017: AVROBIO Expands Rare Disease Pipeline with Gene Therapy to Treat Gaucher Disease Oct 25, 2016: Asthma research unexpectedly yields new treatment approach for inherited enzyme disease Sep 02, 2014: Shire's VPRIV400 units/vial launched in Japan for the improvement of symptoms of Gaucher disease Jul 04, 2014: Shire's VPRIV approved in Japan for the improvement of symptoms of Gaucher disease Jun 18, 2014: Protalix BioTherapeutics Initiates Phase II Study With PRX-112, an Orally-Administered Enzyme Replacement Therapy for the Treatment of Gaucher Disease Mar 14, 2014: Shire Pharmaceuticals Initiates Voluntary Nationwide Recall Of One Batch, Packaged into Three Lots, of VPRIV (velaglucerase alfa for injection) Feb 14, 2014: Shire Announces FDA Approval of Manufacturing Facility for VPRIV (Velaglucerase Alfa for Injection) Drug Substance Feb 12, 2014: Protalix BioTherapeutics Announces Oral GCD Data to be Presented at WORLD Symposium 2014 Oct 14, 2013: Protalix BioTherapeutics Announces Positive Phase I Clinical Trial Results for Oral GCD in Gaucher Disease Patients Apr 02, 2013: Protalix BioTherapeutics Treats First Gaucher Patient In Phase I Study With PRX-112 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 Jun 28, 2012: Shire's Vpriv Shows Significant Improvement In Gaucher-Related Bone Disease Jun 28, 2012: Shire's Vpriv Shows Significant Improvement In Gaucher-Related Bone Disease Feb 22, 2012: Shire Announces European Approval Of Manufacturing Facility For VPRIV Dec 19, 2011: Lixte's LB-205 Shows Therapeutic Potential For Gaucher Disease Appendix Methodology Coverage Secondary Research Primary Research Expert Panel Validation Contact Us Disclaimer
List of Tables
Number of Products under Development for Gaucher Disease, H1 2017 Number of Products under Development by Companies, H1 2017 Number of Products under Development by Universities/Institutes, H1 2017 Products under Development by Companies, H1 2017 Products under Development by Universities/Institutes, H1 2017 Number of Products by Stage and Target, H1 2017 Number of Products by Stage and Mechanism of Action, H1 2017 Number of Products by Stage and Route of Administration, H1 2017 Number of Products by Stage and Molecule Type, H1 2017 Gaucher Disease - Pipeline by Actelion Ltd, H1 2017 Gaucher Disease - Pipeline by Genzyme Corp, H1 2017 Gaucher Disease - Pipeline by greenovation Biotech GmbH, H1 2017 Gaucher Disease - Pipeline by JCR Pharmaceuticals Co Ltd, H1 2017 Gaucher Disease - Pipeline by Lixte Biotechnology Holdings Inc, H1 2017 Gaucher Disease - Pipeline by Neuraltus Pharmaceuticals Inc, H1 2017 Gaucher Disease - Pipeline by Orphazyme ApS, H1 2017 Gaucher Disease - Pipeline by Pharming Group NV, H1 2017 Gaucher Disease - Pipeline by Protalix BioTherapeutics Inc, H1 2017 Gaucher Disease - Pipeline by Shire Plc, H1 2017 Gaucher Disease - Dormant Projects, H1 2017 Gaucher Disease - Discontinued Products, H1 2017
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