Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline Review, H1 2014

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline Review, H1 2014

Summary

Global Markets Direct’s, ‘Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline Review, H1 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
Table of Contents 2
List of Tables 4
List of Figures 4
Introduction 5
Global Markets Direct Report Coverage 5
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) Overview 6
Therapeutics Development 7
Pipeline Products for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Overview 7
Pipeline Products for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Comparative Analysis 8
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Therapeutics under Development by Companies 9
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline Products Glance 10
Early Stage Products 10
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Products under Development by Companies 11
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Companies Involved in Therapeutics Development 12
Athersys, Inc. 12
OPKO Health, Inc. 13
Amicus Therapeutics, Inc. 14
ReGenX Biosciences, LLC 15
Fate Therapeutics, Inc. 16
ArmaGen Technologies, Inc. 17
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Therapeutics Assessment 18
Assessment by Monotherapy Products 18
Assessment by Combination Products 19
Assessment by Target 20
Assessment by Mechanism of Action 21
Assessment by Route of Administration 22
Assessment by Molecule Type 23
Drug Profiles 25
MultiStem - Drug Profile 25
Product Description 25
Mechanism of Action 25
R&D Progress 25
AGT-181 - Drug Profile 28
Product Description 28
Mechanism of Action 28
R&D Progress 28
FT-1050 - Drug Profile 29
Product Description 29
Mechanism of Action 29
R&D Progress 29
Gene Therapy To Activate Iduronidase For Hurler Syndrome - Drug Profile 31
Product Description 31
Mechanism of Action 31
R&D Progress 31
Oligonucleotide For MPS-1 - Drug Profile 32
Product Description 32
Mechanism of Action 32
R&D Progress 32
laronidase + Chaperone - Drug Profile 33
Product Description 33
Mechanism of Action 33
R&D Progress 33
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Recent Pipeline Updates 34
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Dormant Projects 44
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Product Development Milestones 45
Featured News & Press Releases 45
Jun 25, 2013: Amicus Therapeutics Announces Chaperone-Advanced Replacement Therapy In Development For Mucopolysaccharidosis Type I 45
Jul 10, 2012: Athersys Receives Orphan Drug Designation For MultiStem In Hurler’s Syndrome 46
Apr 20, 2012: Athersys Announces Publication Of Results Of Preclinical Study Of MultiStem In Cell Transplant 46
Appendix 48
Methodology 48
Coverage 48
Secondary Research 48
Primary Research 48
Expert Panel Validation 48
Contact Us 49
Disclaimer 49

List of Tables
Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ), H1 2014 7
Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Comparative Analysis, H1 2014 8
Number of Products under Development by Companies, H1 2014 9
Comparative Analysis by Early Stage Development, H1 2014 10
Products under Development by Companies, H1 2014 11
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Athersys, Inc., H1 2014 12
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by OPKO Health, Inc., H1 2014 13
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Amicus Therapeutics, Inc., H1 2014 14
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by ReGenX Biosciences, LLC, H1 2014 15
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Fate Therapeutics, Inc., H1 2014 16
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by ArmaGen Technologies, Inc., H1 2014 17
Assessment by Monotherapy Products, H1 2014 18
Assessment by Combination Products, H1 2014 19
Number of Products by Stage and Target, H1 2014 20
Number of Products by Stage and Mechanism of Action, H1 2014 21
Number of Products by Stage and Route of Administration, H1 2014 22
Number of Products by Stage and Molecule Type, H1 2014 24
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) Therapeutics - Recent Pipeline Updates, H1 2014 34
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Dormant Projects, H1 2014 44

List of Figures
Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Comparative Analysis, H1 2014 8
Number of Products by Top 10 Molecule Type, H1 2014 23