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Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline Review, H1 2015

Summary

Global Markets Direct’s, ‘Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline Review, H1 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, H1 2015
Table of Contents
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
Amicus Therapeutics, Inc. 16
AngioChem Inc. 17
ArmaGen Technologies, Inc. 18
Athersys, Inc. 19
biOasis Technologies Inc. 20
Fate Therapeutics, Inc. 21
OPKO Health, Inc. 22
PTC Therapeutics, Inc. 23
ReGenX Biosciences, LLC 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 30
Assessment by Molecule Type 32
Drug Profiles 34
laronidase + Chaperone - Drug Profile 34
Product Description 34
Mechanism of Action 34
RandD Progress 34
AGT-181 - Drug Profile 35
Product Description 35
Mechanism of Action 35
RandD Progress 35
ataluren - Drug Profile 36
Product Description 36
Mechanism of Action 36
RandD Progress 36
Cell Therapy for Mucopolysaccharidosis I - Drug Profile 39
Product Description 39
Mechanism of Action 39
RandD Progress 39
Cell Therapy to Activate Iduronidase for Hurler Syndrome - Drug Profile 40
Product Description 40
Mechanism of Action 40
RandD Progress 40
FT-1050 - Drug Profile 41
Product Description 41
Mechanism of Action 41
RandD Progress 41
Gene Therapy to Activate Iduronidase for Hurler Syndrome - Drug Profile 44
Product Description 44
Mechanism of Action 44
RandD Progress 44
MultiStem - Drug Profile 45
Product Description 45
Mechanism of Action 45
RandD Progress 45
Oligonucleotide for Mucopolysaccharidosis I - Drug Profile 48
Product Description 48
Mechanism of Action 48
RandD Progress 48
Recombinant Enzyme to Replace Alpha-L-Iduronidase for Hurler Syndrome - Drug Profile 49
Product Description 49
Mechanism of Action 49
RandD Progress 49
Recombinant Enzyme to Replace Alpha-L-Iduronidase for Mucopolysaccharidosis I - Drug Profile 50
Product Description 50
Mechanism of Action 50
RandD Progress 50
Stem Cell Therapy for Type1 Mucopolysaccharidosis - Drug Profile 51
Product Description 51
Mechanism of Action 51
RandD Progress 51
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Recent Pipeline Updates 52
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Dormant Projects 65
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Product Development Milestones 66
Featured News and Press Releases 66
Dec 19, 2014: Translarna Granted Orphan Drug Designation in the U.S. and Europe for the Treatment of Mucopolysaccharidosis I 66
Jul 10, 2012: Athersys Receives Orphan Drug Designation For MultiStem In Hurler's Syndrome 67
Apr 20, 2012: Athersys Announces Publication Of Results Of Preclinical Study Of MultiStem In Cell Transplant 67
Appendix 69
Methodology 69
Coverage 69
Secondary Research 69
Primary Research 69
Expert Panel Validation 69
Contact Us 70
Disclaimer 70

List of Tables

Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome), H1 2015 8
Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Comparative Analysis, H1 2015 9
Number of Products under Development by Companies, H1 2015 10
Number of Products under Investigation by Universities/Institutes, H1 2015 11
Comparative Analysis by Clinical Stage Development, H1 2015 12
Comparative Analysis by Early Stage Development, H1 2015 13
Products under Development by Companies, H1 2015 14
Products under Investigation by Universities/Institutes, H1 2015 15
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by Amicus Therapeutics, Inc., H1 2015 16
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by AngioChem Inc., H1 2015 17
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by ArmaGen Technologies, Inc., H1 2015 18
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by Athersys, Inc., H1 2015 19
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by biOasis Technologies Inc., H1 2015 20
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by Fate Therapeutics, Inc., H1 2015 21
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by OPKO Health, Inc., H1 2015 22
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by PTC Therapeutics, Inc., H1 2015 23
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by ReGenX Biosciences, LLC, H1 2015 24
Assessment by Monotherapy Products, H1 2015 25
Assessment by Combination Products, H1 2015 26
Number of Products by Stage and Target, H1 2015 27
Number of Products by Stage and Mechanism of Action, H1 2015 29
Number of Products by Stage and Route of Administration, H1 2015 31
Number of Products by Stage and Molecule Type, H1 2015 33
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) Therapeutics - Recent Pipeline Updates, H1 2015 52
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Dormant Projects, H1 2015 65

List of Figures

Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome), H1 2015 8
Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Comparative Analysis, H1 2015 9
Number of Products under Development by Companies, H1 2015 10
Comparative Analysis by Early Stage Products, H1 2015 13
Assessment by Monotherapy Products, H1 2015 25
Number of Products by Stage and Top 10 Targets, H1 2015 27
Number of Products by Top 10 Mechanism of Actions, H1 2015 28
Number of Products by Stage and Top 10 Mechanism of Actions, H1 2015 29
Number of Products by Top 10 Routes of Administration, H1 2015 30
Number of Products by Stage and Top 10 Routes of Administration, H1 2015 31
Number of Products by Top 10 Molecule Types, H1 2015 32
Number of Products by Stage and Top 10 Molecule Types, H1 2015 33

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