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Published: Dec, 2016 | Pages:
71 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)
Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline Review, H2 2016 Summary Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline Review, H2 2016, provides an overview of the Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) (Metabolic Disorders) pipeline landscape. Mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, is a condition that affects many different parts of the body and occurs almost exclusively in males. Signs and symptoms include claw-like hands, protruding tongue, changing facial features, including thickening of the lips, tongue and nostrils and delayed development. Treatment includes bone marrow transplantation, enzyme therapy and gene therapy. Report Highlights Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline Review, H2 2016, provides comprehensive information on the therapeutics under development for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) (Metabolic 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 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) (Metabolic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, Phase II, IND/CTA Filed, Preclinical and Discovery stages are 2, 2, 1, 8 and 1 respectively. Similarly, the Universities portfolio in Phase I and Preclinical stages comprises 1 and 1 molecules, respectively. Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) (Metabolic 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 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) (Metabolic Disorders). - The pipeline guide reviews pipeline therapeutics for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) (Metabolic 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 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) (Metabolic Disorders) therapeutics and enlists all their major and minor projects. - The pipeline guide evaluates Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) (Metabolic 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 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) (Metabolic 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 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) (Metabolic 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 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) (Metabolic 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 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) Overview 8 Therapeutics Development 9 Pipeline Products for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Overview 9 Pipeline Products for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Comparative Analysis 10 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Therapeutics under Development by Companies 11 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Therapeutics under Investigation by Universities/Institutes 12 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline Products Glance 13 Late Stage Products 13 Clinical Stage Products 14 Early Stage Products 15 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Products under Development by Companies 16 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Products under Investigation by Universities/Institutes 17 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Companies Involved in Therapeutics Development 18 Alexion Pharmaceuticals Inc 18 AngioChem Inc 19 ArmaGen Inc 20 Bioasis Technologies Inc 21 Green Cross Corp 22 Inventiva 23 JCR Pharmaceuticals Co Ltd 24 Laboratorios Del Dr Esteve SA 25 RegenxBio Inc 26 Sangamo BioSciences Inc 27 Shire Plc 28 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - 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 AGT-182 - Drug Profile 38 Product Description 38 Mechanism Of Action 38 R&D Progress 38 DUOC-01 - Drug Profile 40 Product Description 40 Mechanism Of Action 40 R&D Progress 40 EGT-301 - Drug Profile 41 Product Description 41 Mechanism Of Action 41 R&D Progress 41 Gene Therapy to Activate Iduronate 2 Sulfatase for Mucopolysaccharidosis II - Drug Profile 42 Product Description 42 Mechanism Of Action 42 R&D Progress 42 idursulfase - Drug Profile 43 Product Description 43 Mechanism Of Action 43 R&D Progress 43 idursulfase beta - Drug Profile 45 Product Description 45 Mechanism Of Action 45 R&D Progress 45 JR-032 - Drug Profile 47 Product Description 47 Mechanism Of Action 47 R&D Progress 47 JR-141 - Drug Profile 48 Product Description 48 Mechanism Of Action 48 R&D Progress 48 MTf-I2S - Drug Profile 49 Product Description 49 Mechanism Of Action 49 R&D Progress 49 MTfp-I2S - Drug Profile 50 Product Description 50 Mechanism Of Action 50 R&D Progress 50 odiparcil - Drug Profile 51 Product Description 51 Mechanism Of Action 51 R&D Progress 51 pentosan polysulfate sodium - Drug Profile 52 Product Description 52 Mechanism Of Action 52 R&D Progress 52 Recombinant Enzymes for Pompe, Fabry and Hunter Diseases - Drug Profile 53 Product Description 53 Mechanism Of Action 53 R&D Progress 53 Recombinant Iduronate 2-Sulfatase Replacement for Mucopolysaccharidosis II - Drug Profile 54 Product Description 54 Mechanism Of Action 54 R&D Progress 54 RGX-121 - Drug Profile 55 Product Description 55 Mechanism Of Action 55 R&D Progress 55 SB-913 - Drug Profile 57 Product Description 57 Mechanism Of Action 57 R&D Progress 57 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Dormant Projects 59 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Discontinued Products 60 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Product Development Milestones 61 Featured News & Press Releases 61 Oct 26, 2016: National Center for Child Health and Development Initiates Phase 1/2 Trial of Green Cross' idursulfase-beta ICV for the Treatment of Hunter Syndrome with Neurocognitive Decline 61 Sep 13, 2016: REGENXBIO Publishes Data from Ongoing Preclinical Studies of NAV Gene Therapy RGX-121 61 Jul 05, 2016: REGENXBIO Provides Update On Gene Therapy Development Program RGX-121 62 Jun 20, 2016: Sangamo BioSciences Announces FDA Clearance of Investigational New Drug Application for ZFN-Mediated Genome Editing Treatment of MPS II 62 Apr 19, 2016: Preclinical Data from REGENXBIO's RGX-121 Gene Therapy Program to be Presented at the American Society of Gene & Cell Therapy 19th Annual Meeting 63 Feb 25, 2016: ESTEVE Provides Update On EGT-301 For Hunter Syndrome 64 Jul 31, 2015: biOasis Announces the Successful Delivery to the CNS of an Enzyme used to Treat Hunter Syndrome 65 Sep 18, 2014: biOasis Initiates MPS II Study with Renowned Lysosomal Storage Disease Expert Dr. Maurizio Scarpa and The Brains for Brain Foundation 65 Oct 02, 2013: Green Cross Exports Orphan Disease Drug Hunterase to Algeria 66 Apr 08, 2013: Green Cross Announces Publication Of Clinical Trial Paper On Hunter Syndrome Drug Hunterase In International Scientific Press 67 Feb 18, 2013: Green Cross Receives FDA Orphan Drug Designation For Hunterase For Treatment Of Hunter Syndrome 68 Jan 10, 2012: Green Cross Receives Korean Approval For Hunterase 68 Oct 12, 2011: Green Cross Files Application For Korean Approval Of Hunterase For Treatment Of Hunter Syndrome 69 Appendix 70 Methodology 70 Coverage 70 Secondary Research 70 Primary Research 70 Expert Panel Validation 70 Contact Us 70 Disclaimer 71
List of Tables
Number of Products under Development for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ), H2 2016 9 Number of Products under Development for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - 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 Late Stage Development, H2 2016 13 Comparative Analysis by Clinical Stage Development, H2 2016 14 Comparative Analysis by Early Stage Development, H2 2016 15 Products under Development by Companies, H2 2016 16 Products under Investigation by Universities/Institutes, H2 2016 17 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by Alexion Pharmaceuticals Inc, H2 2016 18 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by AngioChem Inc, H2 2016 19 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by ArmaGen Inc, H2 2016 20 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by Bioasis Technologies Inc, H2 2016 21 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by Green Cross Corp, H2 2016 22 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by Inventiva, H2 2016 23 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by JCR Pharmaceuticals Co Ltd, H2 2016 24 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by Laboratorios Del Dr Esteve SA, H2 2016 25 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by RegenxBio Inc, H2 2016 26 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by Sangamo BioSciences Inc, H2 2016 27 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by Shire Plc, 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 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Dormant Projects, H2 2016 59 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Discontinued Products, H2 2016 60
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