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Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline Review, H2 2015

Summary

Global Markets Direct’s, ‘Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline Review, H2 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

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline Review, H2 2015
Table of Contents
Table of Contents 2
List of Tables 5
List of Figures 6
Introduction 7
Global Markets Direct Report Coverage 7
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) Overview 8
Therapeutics Development 9
Pipeline Products for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Overview 9
Pipeline Products for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Comparative Analysis 10
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Therapeutics under Development by Companies 11
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Therapeutics under Investigation by Universities/Institutes 12
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline Products Glance 13
Clinical Stage Products 13
Early Stage Products 14
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Products under Development by Companies 15
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Products under Investigation by Universities/Institutes 16
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Companies Involved in Therapeutics Development 17
Amicus Therapeutics, Inc. 17
AngioChem Inc. 18
ArmaGen Technologies, Inc. 19
Athersys, Inc. 20
biOasis Technologies Inc. 21
Fate Therapeutics, Inc. 22
OPKO Health, Inc. 23
PTC Therapeutics, Inc. 24
RegenxBio Inc. 25
Sangamo BioSciences, Inc. 26
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Therapeutics Assessment 27
Assessment by Monotherapy Products 27
Assessment by Combination Products 28
Assessment by Target 29
Assessment by Mechanism of Action 31
Assessment by Route of Administration 33
Assessment by Molecule Type 35
Drug Profiles 37
AGT-181 - Drug Profile 37
Product Description 37
Mechanism of Action 37
RandD Progress 37
ataluren - Drug Profile 38
Product Description 38
Mechanism of Action 38
RandD Progress 38
Cell Therapy for Mucopolysaccharidosis I - Drug Profile 41
Product Description 41
Mechanism of Action 41
RandD Progress 41
Cell Therapy to Activate Iduronidase for Hurler Syndrome - Drug Profile 42
Product Description 42
Mechanism of Action 42
RandD Progress 42
FT-1050 - Drug Profile 43
Product Description 43
Mechanism of Action 43
RandD Progress 43
laronidase + chaperone - Drug Profile 46
Product Description 46
Mechanism of Action 46
RandD Progress 46
MultiStem - Drug Profile 47
Product Description 47
Mechanism of Action 47
RandD Progress 47
Oligonucleotide for Mucopolysaccharidosis I - Drug Profile 51
Product Description 51
Mechanism of Action 51
RandD Progress 51
Recombinant Enzyme to Replace Alpha-L-Iduronidase for Hurler Syndrome - Drug Profile 52
Product Description 52
Mechanism of Action 52
RandD Progress 52
Recombinant Enzyme to Target LRP1 for Mucopolysaccharidosis I - Drug Profile 53
Product Description 53
Mechanism of Action 53
RandD Progress 53
RGX-111 - Drug Profile 54
Product Description 54
Mechanism of Action 54
RandD Progress 54
SB-318 - Drug Profile 55
Product Description 55
Mechanism of Action 55
RandD Progress 55
Stem Cell Therapy for Type1 Mucopolysaccharidosis - Drug Profile 56
Product Description 56
Mechanism of Action 56
RandD Progress 56
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Recent Pipeline Updates 57
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Dormant Projects 72
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Product Development Milestones 73
Featured News and Press Releases 73
Apr 08, 2015: ArmaGen Announces FDA Acceptance of IND Application for AGT-181 for the Treatment of Hurler Syndrome 73
Dec 19, 2014: Translarna Granted Orphan Drug Designation in the U.S. and Europe for the Treatment of Mucopolysaccharidosis I 73
Jul 10, 2012: Athersys Receives Orphan Drug Designation For MultiStem In Hurler's Syndrome 74
Apr 20, 2012: Athersys Announces Publication Of Results Of Preclinical Study Of MultiStem In Cell Transplant 75
Appendix 76
Methodology 76
Coverage 76
Secondary Research 76
Primary Research 76
Expert Panel Validation 76
Contact Us 76
Disclaimer 77

List of Tables
Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ), H2 2015 9
Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Comparative Analysis, H2 2015 10
Number of Products under Development by Companies, H2 2015 11
Number of Products under Investigation by Universities/Institutes, H2 2015 12
Comparative Analysis by Clinical Stage Development, H2 2015 13
Comparative Analysis by Early Stage Development, H2 2015 14
Products under Development by Companies, H2 2015 15
Products under Investigation by Universities/Institutes, H2 2015 16
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Amicus Therapeutics, Inc., H2 2015 17
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by AngioChem Inc., H2 2015 18
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by ArmaGen Technologies, Inc., H2 2015 19
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Athersys, Inc., H2 2015 20
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by biOasis Technologies Inc., H2 2015 21
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Fate Therapeutics, Inc., H2 2015 22
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by OPKO Health, Inc., H2 2015 23
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by PTC Therapeutics, Inc., H2 2015 24
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by RegenxBio Inc., H2 2015 25
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Sangamo BioSciences, Inc., H2 2015 26
Assessment by Monotherapy Products, H2 2015 27
Assessment by Combination Products, H2 2015 28
Number of Products by Stage and Target, H2 2015 30
Number of Products by Stage and Mechanism of Action, H2 2015 32
Number of Products by Stage and Route of Administration, H2 2015 34
Number of Products by Stage and Molecule Type, H2 2015 36
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) Therapeutics - Recent Pipeline Updates, H2 2015 57
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Dormant Projects, H2 2015 72

List of Figures
Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ), H2 2015 9
Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Comparative Analysis, H2 2015 10
Number of Products under Development by Companies, H2 2015 11
Comparative Analysis by Clinical Stage Development, H2 2015 13
Comparative Analysis by Early Stage Products, H2 2015 14
Assessment by Monotherapy Products, H2 2015 27
Number of Products by Top 10 Targets, H2 2015 29
Number of Products by Stage and Top 10 Targets, H2 2015 29
Number of Products by Top 10 Mechanism of Actions, H2 2015 31
Number of Products by Stage and Top 10 Mechanism of Actions, H2 2015 31
Number of Products by Top 10 Routes of Administration, H2 2015 33
Number of Products by Stage and Top 10 Routes of Administration, H2 2015 33
Number of Products by Top 10 Molecule Types, H2 2015 35
Number of Products by Stage and Top 10 Molecule Types, H2 2015 35

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