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Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline Review, H2 2016

Published: Dec, 2016 | Pages: 71 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline Review, H2 2016

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline Review, H2 2016, provides an overview of the Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) (Metabolic Disorders) pipeline landscape.

Mucopolysaccharidosis type III (MPS III), also known as Sanfilippo syndrome, is an inherited metabolic disease caused by an absence or malfunctioning of certain enzymes needed to breakdown molecules called glycosaminoglycans (GAG). Symptoms include seizures, hyperactivity, liver and spleen enlargement, severe diarrhea or constipation and enlargement of tonsils and adenoids. Treatment includes enzyme replacement therapy (ERT).

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline Review, H2 2016, provides comprehensive information on the therapeutics under development for Mucopolysaccharidosis III (MPS III) (Sanfilippo 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 III (MPS III) (Sanfilippo Syndrome ) (Metabolic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase II, IND/CTA Filed, Preclinical and Discovery stages are 7, 1, 7 and 3 respectively. Similarly, the Universities portfolio in Preclinical stages comprises 1 molecules, respectively.

Mucopolysaccharidosis III (MPS III) (Sanfilippo 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 III (MPS III) (Sanfilippo Syndrome ) (Metabolic Disorders).
- The pipeline guide reviews pipeline therapeutics for Mucopolysaccharidosis III (MPS III) (Sanfilippo 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 III (MPS III) (Sanfilippo Syndrome ) (Metabolic Disorders) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Mucopolysaccharidosis III (MPS III) (Sanfilippo 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 III (MPS III) (Sanfilippo 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 III (MPS III) (Sanfilippo 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 III (MPS III) (Sanfilippo 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 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 Alexion Pharmaceuticals Inc 18 ArmaGen Inc 19 Axcentua Pharmaceuticals AB 20 BioMarin Pharmaceutical Inc 21 Dorphan SA 22 Laboratorios Del Dr Esteve SA 23 Lysogene SAS 24 Swedish Orphan Biovitrum AB 25 UniQure NV 26 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Therapeutics Assessment 27 Assessment by Monotherapy Products 27 Assessment by Target 28 Assessment by Mechanism of Action 30 Assessment by Route of Administration 32 Assessment by Molecule Type 34 Drug Profiles 36 ABO-101 - Drug Profile 36 Product Description 36 Mechanism Of Action 36 R&D Progress 36 ABO-102 - Drug Profile 38 Product Description 38 Mechanism Of Action 38 R&D Progress 38 AGT-184 - Drug Profile 41 Product Description 41 Mechanism Of Action 41 R&D Progress 41 AMT-110 - Drug Profile 42 Product Description 42 Mechanism Of Action 42 R&D Progress 42 AXP-10711 - Drug Profile 44 Product Description 44 Mechanism Of Action 44 R&D Progress 44 BMN-250 - Drug Profile 46 Product Description 46 Mechanism Of Action 46 R&D Progress 46 EGT-101 - Drug Profile 48 Product Description 48 Mechanism Of Action 48 R&D Progress 48 EGT-201 - Drug Profile 49 Product Description 49 Mechanism Of Action 49 R&D Progress 49 lesinidase alfa - Drug Profile 50 Product Description 50 Mechanism Of Action 50 R&D Progress 50 LYSSAF-301 - Drug Profile 53 Product Description 53 Mechanism Of Action 53 R&D Progress 53 LYSSAF-302 - Drug Profile 54 Product Description 54 Mechanism Of Action 54 R&D Progress 54 Proteins for Mucopolysaccharidosis Type IIIC - Drug Profile 55 Product Description 55 Mechanism Of Action 55 R&D Progress 55 Recombinant N-Acetylgalactosamine-6-Sulfatase Replacement for Mucopolysaccharidosis Type IIID - Drug Profile 56 Product Description 56 Mechanism Of Action 56 R&D Progress 56 Small Molecule for Sanfilippo Syndrome - Drug Profile 57 Product Description 57 Mechanism Of Action 57 R&D Progress 57 Small Molecule to Target Alpha-N-Acetylglucosaminidase for MPSIIIB - Drug Profile 58 Product Description 58 Mechanism Of Action 58 R&D Progress 58 SOBI-003 - Drug Profile 59 Product Description 59 Mechanism Of Action 59 R&D Progress 59 Stem Cell Therapy for Mucopolysaccharidosis Type IIIA - Drug Profile 60 Product Description 60 Mechanism Of Action 60 R&D Progress 60 Stem Cell Therapy for Sanfilippo Syndrome - Drug Profile 61 Product Description 61 Mechanism Of Action 61 R&D Progress 61 Stem Cell Therapy to Activate NAGLU for Mucopolysaccharidosis IIIB - Drug Profile 62 Product Description 62 Mechanism Of Action 62 R&D Progress 62 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Dormant Projects 63 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Discontinued Products 64 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Product Development Milestones 65 Featured News & Press Releases 65 Oct 19, 2016: European Commission Grants SOBI003 Orphan Designation for the Treatment of MPS IIIA 65 May 24, 2016: Abeona Therapeutics Announces FDA Allowance of Investigational New Drug for Phase 1/2 Clinical Study With ABO-101 Gene Therapy for Patients With Sanfilippo Syndrome Type B (MPS IIIB) 65 Feb 25, 2016: ESTEVE Provides Update On EGT-201 For Sanfilippo B Syndrome 66 Jan 11, 2016: Abeona Therapeutics Announces Initial European Regulatory Approval for Phase 1/2 Gene Therapy Clinical Study for Patients With Sanfilippo Syndrome 67 May 20, 2015: PlasmaTech Biopharmaceuticals Announces Orphan Drug and Rare Pediatric Disease Designations For ABX-101 From FDA 68 Apr 02, 2014: Orphan designation granted for MPSIII 68 Jul 25, 2013: New stem cell gene therapy gives hope to prevent inherited neurological disease 68 Apr 11, 2013: Axcentua granted patent on ten solid forms of genistein 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 III (MPS III) (Sanfilippo Syndrome ), H2 2016 9 Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo 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 Clinical Stage Development, H2 2016 13 Comparative Analysis by Early Stage Development, H2 2016 14 Products under Development by Companies, H2 2016 15 Products under Investigation by Universities/Institutes, H2 2016 16 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Abeona Therapeutics Inc, H2 2016 17 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Alexion Pharmaceuticals Inc, H2 2016 18 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by ArmaGen Inc, H2 2016 19 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Axcentua Pharmaceuticals AB, H2 2016 20 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by BioMarin Pharmaceutical Inc, H2 2016 21 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Dorphan SA, H2 2016 22 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Laboratorios Del Dr Esteve SA, H2 2016 23 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Lysogene SAS, H2 2016 24 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Swedish Orphan Biovitrum AB, H2 2016 25 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by UniQure NV, H2 2016 26 Assessment by Monotherapy Products, H2 2016 27 Number of Products by Stage and Target, H2 2016 29 Number of Products by Stage and Mechanism of Action, H2 2016 31 Number of Products by Stage and Route of Administration, H2 2016 33 Number of Products by Stage and Molecule Type, H2 2016 35 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Dormant Projects, H2 2016 63 Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Discontinued Products, H2 2016 64



                                

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