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Published: Jan, 2015 | Pages:
70 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline Review, H1 2015 Summary Global Markets Direct's, 'Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline Review, H1 2015', provides an overview of the Mucopolysaccharidosis I (MPS I) (Hurler Syndrome)'s therapeutic pipeline. This report provides comprehensive information on the therapeutic development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome), 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 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 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 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - 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 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) and enlists all their major and minor projects - The report summarizes all the dormant and discontinued pipeline projects - A review of the Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 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 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 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 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline - Devise corrective measures for pipeline projects by understanding Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) 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 4 List of Figures 5 Introduction 6 Global Markets Direct Report Coverage 6 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) Overview 7 Therapeutics Development 8 Pipeline Products for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Overview 8 Pipeline Products for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Comparative Analysis 9 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Therapeutics under Development by Companies 10 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Therapeutics under Investigation by Universities/Institutes 11 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline Products Glance 12 Clinical Stage Products 12 Early Stage Products 13 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Products under Development by Companies 14 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Products under Investigation by Universities/Institutes 15 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Companies Involved in Therapeutics Development 16 Amicus Therapeutics, Inc. 16 AngioChem Inc. 17 ArmaGen Technologies, Inc. 18 Athersys, Inc. 19 biOasis Technologies Inc. 20 Fate Therapeutics, Inc. 21 OPKO Health, Inc. 22 PTC Therapeutics, Inc. 23 ReGenX Biosciences, LLC 24 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Therapeutics Assessment 25 Assessment by Monotherapy Products 25 Assessment by Combination Products 26 Assessment by Target 27 Assessment by Mechanism of Action 28 Assessment by Route of Administration 30 Assessment by Molecule Type 32 Drug Profiles 34 laronidase + Chaperone - Drug Profile 34 Product Description 34 Mechanism of Action 34 R&D Progress 34 AGT-181 - Drug Profile 35 Product Description 35 Mechanism of Action 35 R&D Progress 35 ataluren - Drug Profile 36 Product Description 36 Mechanism of Action 36 R&D Progress 36 Cell Therapy for Mucopolysaccharidosis I - Drug Profile 39 Product Description 39 Mechanism of Action 39 R&D Progress 39 Cell Therapy to Activate Iduronidase for Hurler Syndrome - Drug Profile 40 Product Description 40 Mechanism of Action 40 R&D Progress 40 FT-1050 - Drug Profile 41 Product Description 41 Mechanism of Action 41 R&D Progress 41 Gene Therapy to Activate Iduronidase for Hurler Syndrome - Drug Profile 44 Product Description 44 Mechanism of Action 44 R&D Progress 44 MultiStem - Drug Profile 45 Product Description 45 Mechanism of Action 45 R&D Progress 45 Oligonucleotide for Mucopolysaccharidosis I - Drug Profile 48 Product Description 48 Mechanism of Action 48 R&D Progress 48 Recombinant Enzyme to Replace Alpha-L-Iduronidase for Hurler Syndrome - Drug Profile 49 Product Description 49 Mechanism of Action 49 R&D Progress 49 Recombinant Enzyme to Replace Alpha-L-Iduronidase for Mucopolysaccharidosis I - Drug Profile 50 Product Description 50 Mechanism of Action 50 R&D Progress 50 Stem Cell Therapy for Type1 Mucopolysaccharidosis - Drug Profile 51 Product Description 51 Mechanism of Action 51 R&D Progress 51 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Recent Pipeline Updates 52 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Dormant Projects 65 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Product Development Milestones 66 Featured News & Press Releases 66 Dec 19, 2014: Translarna Granted Orphan Drug Designation in the U.S. and Europe for the Treatment of Mucopolysaccharidosis I 66 Jul 10, 2012: Athersys Receives Orphan Drug Designation For MultiStem In Hurler's Syndrome 67 Apr 20, 2012: Athersys Announces Publication Of Results Of Preclinical Study Of MultiStem In Cell Transplant 67 Appendix 69 Methodology 69 Coverage 69 Secondary Research 69 Primary Research 69 Expert Panel Validation 69 Contact Us 70 Disclaimer 70
List of Tables Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome), H1 2015 8 Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Comparative Analysis, H1 2015 9 Number of Products under Development by Companies, H1 2015 10 Number of Products under Investigation by Universities/Institutes, H1 2015 11 Comparative Analysis by Clinical Stage Development, H1 2015 12 Comparative Analysis by Early Stage Development, H1 2015 13 Products under Development by Companies, H1 2015 14 Products under Investigation by Universities/Institutes, H1 2015 15 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by Amicus Therapeutics, Inc., H1 2015 16 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by AngioChem Inc., H1 2015 17 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by ArmaGen Technologies, Inc., H1 2015 18 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by Athersys, Inc., H1 2015 19 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by biOasis Technologies Inc., H1 2015 20 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by Fate Therapeutics, Inc., H1 2015 21 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by OPKO Health, Inc., H1 2015 22 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by PTC Therapeutics, Inc., H1 2015 23 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by ReGenX Biosciences, LLC, H1 2015 24 Assessment by Monotherapy Products, H1 2015 25 Assessment by Combination Products, H1 2015 26 Number of Products by Stage and Target, H1 2015 27 Number of Products by Stage and Mechanism of Action, H1 2015 29 Number of Products by Stage and Route of Administration, H1 2015 31 Number of Products by Stage and Molecule Type, H1 2015 33 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) Therapeutics - Recent Pipeline Updates, H1 2015 52 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Dormant Projects, H1 2015 65
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