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Published: Dec, 2014 | Pages:
59 | Publisher: Global Markets Direct
Industry: Healthcare | Report Format: Electronic (PDF)
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline Review, H2 2014 Summary Global Markets Direct's, 'Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline Review, H2 2014', 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 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 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 - Devise corrective measures for pipeline projects by understanding Mucopolysaccharidosis III (MPS III) (Sanfilippo 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 5 List of Figures 5 Introduction 6 Global Markets Direct Report Coverage 6 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) Overview 7 Therapeutics Development 8 Pipeline Products for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Overview 8 Pipeline Products for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Comparative Analysis 9 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Therapeutics under Development by Companies 10 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Therapeutics under Investigation by Universities/Institutes 11 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline Products Glance 12 Clinical Stage Products 12 Early Stage Products 13 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Products under Development by Companies 14 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Products under Investigation by Universities/Institutes 15 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Companies Involved in Therapeutics Development 16 ArmaGen Technologies, Inc. 16 Axcentua Pharmaceuticals AB 17 BioMarin Pharmaceutical Inc. 18 Dorphan S.A. 19 Laboratorios Del Dr. Esteve S.A. 20 Shire Plc 21 Synageva BioPharma Corp. 22 uniQure N.V. 23 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Therapeutics Assessment 24 Assessment by Monotherapy Products 24 Assessment by Target 25 Assessment by Mechanism of Action 26 Assessment by Route of Administration 28 Assessment by Molecule Type 30 Drug Profiles 32 ABX-A - Drug Profile 32 Product Description 32 Mechanism of Action 32 R&D Progress 32 ABX-B - Drug Profile 33 Product Description 33 Mechanism of Action 33 R&D Progress 33 AGT-184 - Drug Profile 34 Product Description 34 Mechanism of Action 34 R&D Progress 34 AMT-110 - Drug Profile 35 Product Description 35 Mechanism of Action 35 R&D Progress 35 AXP-10711 - Drug Profile 36 Product Description 36 Mechanism of Action 36 R&D Progress 36 BMN-250 - Drug Profile 38 Product Description 38 Mechanism of Action 38 R&D Progress 38 Gene Therapy for Sanfilippo A Syndrome - Drug Profile 39 Product Description 39 Mechanism of Action 39 R&D Progress 39 Gene Therapy to Activate Alpha-N-Acetylglucosaminidase for Sanfilippo Type B Syndrome - Drug Profile 40 Product Description 40 Mechanism of Action 40 R&D Progress 40 HGT-3010 - Drug Profile 41 Product Description 41 Mechanism of Action 41 R&D Progress 41 SAF-301 - Drug Profile 42 Product Description 42 Mechanism of Action 42 R&D Progress 42 SBC-103 - Drug Profile 43 Product Description 43 Mechanism of Action 43 R&D Progress 43 SHP-610 - Drug Profile 44 Product Description 44 Mechanism of Action 44 R&D Progress 44 Small Molecule for Sanfilippo Syndrome - Drug Profile 45 Product Description 45 Mechanism of Action 45 R&D Progress 45 Small Molecule to Target Alpha-N-Acetylglucosaminidase for MPSIIIB - Drug Profile 46 Product Description 46 Mechanism of Action 46 R&D Progress 46 Stem Cell Therapy for Sanfilippo Syndrome - Drug Profile 47 Product Description 47 Mechanism of Action 47 R&D Progress 47 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Recent Pipeline Updates 48 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Dormant Projects 51 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Product Development Milestones 52 Featured News & Press Releases 52 Apr 02, 2014: Orphan designation granted for MPSIII 52 Feb 12, 2014: Synageva BioPharma Highlights SBC-103 Data at the Lysosomal Disease Network WORLD Symposium 52 Feb 11, 2014: BioMarin Announces Selection of NAGLU Fusion Protein Drug Development Candidate BMN 250 for the Treatment of Sanfilippo B 54 Jan 29, 2014: Synageva BioPharma Announces Data Presentations On SBC-103 at the 10th Annual Lysosomal Disease Network World Symposium 55 Jul 25, 2013: New stem cell gene therapy gives hope to prevent inherited neurological disease 55 Apr 11, 2013: Axcentua granted patent on ten solid forms of genistein 57 Jan 30, 2013: Synageva BioPharma To Present Scientific Data On SBC-103 At 9th Annual LDN WORLD Symposium 57 Jan 30, 2012: Synageva To Present Preclinical Data Of SBC-103 At Lysosomal Disease Network World Symposium 57 Appendix 58 Methodology 58 Coverage 58 Secondary Research 58 Primary Research 58 Expert Panel Validation 58 Contact Us 59 Disclaimer 59
List of Tables Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome), H2 2014 8 Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Comparative Analysis, H2 2014 9 Number of Products under Development by Companies, H2 2014 10 Number of Products under Investigation by Universities/Institutes, H2 2014 11 Comparative Analysis by Clinical Stage Development, H2 2014 12 Comparative Analysis by Early Stage Development, H2 2014 13 Products under Development by Companies, H2 2014 14 Products under Investigation by Universities/Institutes, H2 2014 15 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline by ArmaGen Technologies, Inc., H2 2014 16 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline by Axcentua Pharmaceuticals AB, H2 2014 17 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline by BioMarin Pharmaceutical Inc., H2 2014 18 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline by Dorphan S.A., H2 2014 19 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline by Laboratorios Del Dr. Esteve S.A., H2 2014 20 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline by Shire Plc, H2 2014 21 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline by Synageva BioPharma Corp., H2 2014 22 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline by uniQure N.V., H2 2014 23 Assessment by Monotherapy Products, H2 2014 24 Number of Products by Stage and Target, H2 2014 25 Number of Products by Stage and Mechanism of Action, H2 2014 27 Number of Products by Stage and Route of Administration, H2 2014 29 Number of Products by Stage and Molecule Type, H2 2014 31 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) Therapeutics - Recent Pipeline Updates, H2 2014 48 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Dormant Projects, H2 2014 51
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