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Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline Review, H2 2013

Published By :

Global Markets Direct

Published Date : Dec 2013

Category :

Metabolic Disorders

No. of Pages : 47 Pages


Global Markets Directs, \'Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) Pipeline Review, H2 2013\', provides an overview of the indications therapeutic pipeline. This report provides information on the therapeutic development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome), complete with latest updates, and special features on late-stage and discontinued projects. It also reviews key players involved in the therapeutic development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome). Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) Pipeline Review, Half Year is built using data and information sourced from Global Markets Directs 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 Directs team.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease.

Scope

  • A snapshot of the global therapeutic scenario for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome).
  • 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.
  • Coverage of products based on various stages of development ranging from discovery till registration stages.
  • A feature on pipeline projects on the basis of monotherapy and combined therapeutics.
  • Coverage of the Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) pipeline on the basis of route of administration and molecule type.
  • Key discontinued pipeline projects.
  • Latest news and deals relating to the products.

Reasons to buy

  • Identify and understand important and diverse types of therapeutics under development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome).
  • Identify emerging players with potentially strong product portfolio and design effective counter-strategies to gain competitive advantage.
  • Plan mergers and acquisitions effectively by identifying 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
ArmaGen Technologies, Inc. 16
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Therapeutics Assessment 17
Assessment by Monotherapy Products 17
Assessment by Combination Products 18
Assessment by Target 19
Assessment by Mechanism of Action 20
Assessment by Route of Administration 21
Assessment by Molecule Type 23
Assessment by Therapeutic Class 25
Drug Profiles 27
PF-05285401 - Drug Profile 27
Product Description 27
Mechanism of Action 27
R&D Progress 27
AGT-181 - Drug Profile 30
Product Description 30
Mechanism of Action 30
R&D Progress 30
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 42
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Product Development Milestones 43
Featured News & Press Releases 43
Jun 25, 2013: Amicus Therapeutics Announces Chaperone-Advanced Replacement Therapy In Development For Mucopolysaccharidosis Type I 43
Jul 10, 2012: Athersys Receives Orphan Drug Designation For MultiStem In Hurler’s Syndrome 44
Apr 20, 2012: Athersys Announces Publication Of Results Of Preclinical Study Of MultiStem In Cell Transplant 44

Appendix 46
Methodology 46
Coverage 46
Secondary Research 46
Primary Research 46
Expert Panel Validation 46
Contact Us 47
Disclaimer 47

List of Table


Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome), H2 2013 7
Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Comparative Analysis, H2 2013 8
Number of Products under Development by Companies, H2 2013 9
Comparative Analysis by Early Stage Development, H2 2013 10
Products under Development by Companies, H2 2013 11
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by Athersys, Inc., H2 2013 12
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by OPKO Health, Inc., H2 2013 13
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by Amicus Therapeutics, Inc., H2 2013 14
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by ReGenX Biosciences, LLC, H2 2013 15
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Pipeline by ArmaGen Technologies, Inc., H2 2013 16
Assessment by Monotherapy Products, H2 2013 17
Assessment by Combination Products, H2 2013 18
Number of Products by Stage and Target, H2 2013 19
Number of Products by Stage and Mechanism of Action, H2 2013 20
Number of Products by Stage and Route of Administration, H2 2013 22
Number of Products by Stage and Molecule Type, H2 2013 24
Number of Products by Stage and Therapeutic Class, H2 2013 26
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) Therapeutics - Recent Pipeline Updates, H2 2013 34
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) - Dormant Projects, H2 2013 42

List of Chart


Number of Products by Top 10 Route of Administration, H2 2013 21
Number of Products by Stage and Top 10 Route of Administration, H2 2013 22
Number of Products by Top 10 Molecule Type, H2 2013 23
Number of Products by Stage and Top 10 Molecule Type, H2 2013 24
Number of Products by Top 10 Therapeutic Class, H2 2013 25
Number of Products by Stage and Top 10 Therapeutic Class, H2 2013 26

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