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  4. > Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline Review, H2 2014

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline Review, H2 2014

Summary

Global Markets Direct’s, ‘Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline Review, H2 2014’, 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 2014
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
Athersys, Inc. 16
OPKO Health, Inc. 17
Amicus Therapeutics, Inc. 18
ReGenX Biosciences, LLC 19
AngioChem Inc. 20
PTC Therapeutics, Inc. 21
Fate Therapeutics, Inc. 22
biOasis Technologies Inc. 23
ArmaGen Technologies, Inc. 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 29
Assessment by Molecule Type 31
Drug Profiles 33
ataluren - Drug Profile 33
Product Description 33
Mechanism of Action 33
RandD Progress 33
FT-1050 - Drug Profile 36
Product Description 36
Mechanism of Action 36
RandD Progress 36
MultiStem - Drug Profile 38
Product Description 38
Mechanism of Action 38
RandD Progress 38
AGT-181 - Drug Profile 41
Product Description 41
Mechanism of Action 41
RandD Progress 41
Gene Therapy to Activate Iduronidase for Hurler Syndrome - Drug Profile 42
Product Description 42
Mechanism of Action 42
RandD Progress 42
Oligonucleotide for Mucopolysaccharidosis I - Drug Profile 43
Product Description 43
Mechanism of Action 43
RandD Progress 43
(laronidase + Chaperone) - Drug Profile 44
Product Description 44
Mechanism of Action 44
RandD Progress 44
Recombinant Enzyme for Hurler Syndrome - Drug Profile 45
Product Description 45
Mechanism of Action 45
RandD Progress 45
Stem Cell Therapy for Type1 Mucopolysaccharidosis - Drug Profile 46
Product Description 46
Mechanism of Action 46
RandD Progress 46
Cell Therapy for Mucopolysaccharidosis I - Drug Profile 47
Product Description 47
Mechanism of Action 47
RandD Progress 47
Cell Therapy to Activate Iduronidase for Hurler Syndrome - Drug Profile 48
Product Description 48
Mechanism of Action 48
RandD Progress 48
Recombinant Enzyme to Activate Alpha-L-Iduronidase for Mucopolysaccharidosis I - Drug Profile 49
Product Description 49
Mechanism of Action 49
RandD Progress 49
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Recent Pipeline Updates 50
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Dormant Projects 62
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Product Development Milestones 63
Featured News and Press Releases 63
Jun 25, 2013: Amicus Therapeutics Announces Chaperone-Advanced Replacement Therapy In Development For Mucopolysaccharidosis Type I 63
Jul 10, 2012: Athersys Receives Orphan Drug Designation For MultiStem In Hurler's Syndrome 64
Apr 20, 2012: Athersys Announces Publication Of Results Of Preclinical Study Of MultiStem In Cell Transplant 64
Appendix 66
Methodology 66
Coverage 66
Secondary Research 66
Primary Research 66
Expert Panel Validation 66
Contact Us 67
Disclaimer 67

List of Tables

Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ), H2 2014 8
Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler 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 I (MPS I) (Hurler Syndrome) - Pipeline by Athersys, Inc., H2 2014 16
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by OPKO Health, Inc., H2 2014 17
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by Amicus Therapeutics, Inc., H2 2014 18
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by ReGenX Biosciences, LLC, H2 2014 19
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by AngioChem Inc., H2 2014 20
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by PTC Therapeutics, Inc., H2 2014 21
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by Fate Therapeutics, Inc., H2 2014 22
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by biOasis Technologies Inc., H2 2014 23
Mucopolysaccharidosis I (MPS I) (Hurler Syndrom) - Pipeline by ArmaGen Technologies, Inc., H2 2014 24
Assessment by Monotherapy Products, H2 2014 25
Assessment by Combination Products, H2 2014 26
Number of Products by Stage and Target, H2 2014 27
Number of Products by Stage and Mechanism of Action, H2 2014 28
Number of Products by Stage and Route of Administration, H2 2014 30
Number of Products by Stage and Molecule Type, H2 2014 32
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) Therapeutics - Recent Pipeline Updates, H2 2014 50
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Dormant Projects, H2 2014 62

List of Figures

Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome), H2 2014 8
Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Comparative Analysis, H2 2014 9
Number of Products under Development by Companies, H2 2014 10
Comparative Analysis by Early Stage Products, H2 2014 13
Assessment by Monotherapy Products, H2 2014 25
Number of Products by Stage and Top 10 Target, H2 2014 27
Number of Products by Stage and Top 10 Mechanism of Action, H2 2014 28
Number of Products by Top 10 Route of Administration, H2 2014 29
Number of Products by Stage and Top 10 Route of Administration, H2 2014 30
Number of Products by Top 10 Molecule Type, H2 2014 31
Number of Products by Stage and Top 10 Molecule Type, H2 2014 32

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