Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline Review, H2 2015 Is Released Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline Review, H2 2015 Summary Global Markets Direct s, Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline Review, H2 2015 , provides an overview of the Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) s therapeutic pipeline. This report provides comprehensive information on the therapeutic development for Mucopolysaccharidosis III (MPS III) (Sanfilippo 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 III (MPS III) (Sanfilippo Syndrome) and special features on late-stage and discontinued projects. Global Markets Directs 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 Directs 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 III (MPS III) (Sanfilippo 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 III (MPS III) (Sanfilippo Syndrome) and enlists all their major and minor projects - The report summarizes all the dormant and discontinued pipeline projects - A review of the Mucopolysaccharidosis III (MPS III) (Sanfilippo 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 III (MPS III) (Sanfilippo 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 III (MPS III) (Sanfilippo Syndrome) Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline 1
Devise corrective measures for pipeline projects by understanding Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) pipeline depth and focus of Indication therapeutics - Develop and design inlicensing 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. with TOC @ http://www.idatainsights.com/reports-landingpage.php?id=173386/mucopolysaccharidosis-iii-mps-iii-sanfilippo-syndrome-pipeline-review-h2-2015
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Introduction 7 Global Markets Direct Report Coverage 7 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Overview 8 Therapeutics Development 9 Pipeline Products for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Overview 9 Pipeline Products for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Comparative Analysis 10 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Therapeutics under Development by Companies 11 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Therapeutics under Investigation by Universities/Institutes 12 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline Products Glance 13 Clinical Stage Products 13 Early Stage Products 14 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Products under Development by Companies 15 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Products under Investigation by Universities/Institutes 16 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Companies Involved in Therapeutics Development 17 Abeona Therapeutics, Inc. 17
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Alexion Pharmaceuticals, Inc. 18 ArmaGen,Inc. 19 Axcentua Pharmaceuticals AB 20 BioMarin Pharmaceutical Inc. 21 Dorphan S.A. 22 Laboratorios Del Dr. Esteve S.A. 23 Lysogene 24 Phoenix Nest, Inc. 25 Shire Plc 26 Swedish Orphan Biovitrum AB 27 uniQure N.V. 28 Mucopolysaccharidosis III (MPS III) (Sanfilippo 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 ABO-101 - Drug Profile 38 Product Description 38 Mechanism of Action 38 R&D Progress 38 ABO-102 - Drug Profile 39 Product Description 39 Mechanism of Action 39 R&D Progress 39 AGT-184 - Drug Profile 40 3
Product Description 40 Mechanism of Action 40 R&D Progress 40 AMT-110 - Drug Profile 41 Product Description 41 Mechanism of Action 41 R&D Progress 41 AXP-10711 - Drug Profile 43 Product Description 43 Mechanism of Action 43 R&D Progress 43 BMN-250 - Drug Profile 45 Product Description 45 Mechanism of Action 45 R&D Progress 45 Gene Therapy to Activate Sulphamidase for Metabolic Disorders - Drug Profile 46 Product Description 46 Mechanism of Action 46 R&D Progress 46 LYS-SAF302 - Drug Profile 48 Product Description 48 Mechanism of Action 48 R&D Progress 48 Recombinant Enzyme to Replace N-Acetylgalactosamine-6-Sulfatase for Mucopolysaccharidosis Type IIID - Drug Profile 50 Product Description 50 Mechanism of Action 50 4
R&D Progress 50 SBC-103 - Drug Profile 51 Product Description 51 Mechanism of Action 51 R&D Progress 51 SHP-610 - Drug Profile 52 Product Description 52 Mechanism of Action 52 R&D Progress 52 Small Molecule for Sanfilippo Syndrome - Drug Profile 53 Product Description 53 Mechanism of Action 53 R&D Progress 53 Small Molecule for Sanfilippo Syndrome - Drug Profile 54 Product Description 54 Mechanism of Action 54 R&D Progress 54 Small Molecule to Target Alpha-N-Acetylglucosaminidase for MPSIIIB - Drug Profile 55 Product Description 55 Mechanism of Action 55 R&D Progress 55 SOBI-003 - Drug Profile 56 Product Description 56 Mechanism of Action 56 R&D Progress 56 Stem Cell Therapy for Sanfilippo Syndrome - Drug Profile 57
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Product Description 57 Mechanism of Action 57 R&D Progress 57 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Recent Pipeline Updates 58 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Dormant Projects 63 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Product Development Milestones 64 Featured News & Press Releases 64 May 20, 2015: PlasmaTech Biopharmaceuticals Announces Orphan Drug and Rare Pediatric Disease Designations For ABX-101 From FDA 64 May 20, 2015: PlasmaTech Biopharmaceuticals Announces Orphan Drug and Rare Pediatric Disease Designations for ABX-102 from FDA 64 Dec 10, 2014: FDA Grants BioMarin Orphan Drug Designation for NAGLU Fusion Protein, BMN 250, for the Treatment of MPS IIIB (Sanfilippo Syndrome Type B) 64 Apr 02, 2014: Orphan designation granted for MPSIII 65 Feb 11, 2014: BioMarin Announces Selection of NAGLU Fusion Protein Drug Development Candidate BMN 250 for the Treatment of Sanfilippo B 65 Jul 25, 2013: New stem cell gene therapy gives hope to prevent inherited neurological disease 66 Apr 11, 2013: Axcentua granted patent on ten solid forms of genistein 67 Appendix 68 Methodology 68 Coverage 68 Secondary Research 68 Primary Research 68 Expert Panel Validation 68 Contact Us 68 Disclaimer 69
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