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

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


Introduction
Global Markets Direct Report Coverage
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) Overview
Therapeutics Development
Pipeline Products for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Overview
Pipeline Products for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Comparative Analysis
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Therapeutics under Development by Companies
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Therapeutics under Investigation by Universities/Institutes
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline Products Glance
Clinical Stage Products
Early Stage Products
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Products under Development by Companies
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Products under Investigation by Universities/Institutes
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Companies Involved in Therapeutics Development
Amicus Therapeutics, Inc.
AngioChem Inc.
ArmaGen Technologies, Inc.
Athersys, Inc.
biOasis Technologies Inc.
Fate Therapeutics, Inc.
OPKO Health, Inc.
PTC Therapeutics, Inc.
RegenxBio Inc.
Sangamo BioSciences, Inc.
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Therapeutics Assessment
Assessment by Monotherapy Products
Assessment by Combination Products
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Drug Profiles
AGT-181 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
ataluren - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Cell Therapy for Mucopolysaccharidosis I - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Cell Therapy to Activate Iduronidase for Hurler Syndrome - Drug Profile
Product Description
Mechanism of Action
R&D Progress
FT-1050 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
laronidase + chaperone - Drug Profile
Product Description
Mechanism of Action
R&D Progress
MultiStem - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Oligonucleotide for Mucopolysaccharidosis I - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Recombinant Enzyme to Replace Alpha-L-Iduronidase for Hurler Syndrome - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Recombinant Enzyme to Target LRP1 for Mucopolysaccharidosis I - Drug Profile
Product Description
Mechanism of Action
R&D Progress
RGX-111 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
SB-318 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Stem Cell Therapy for Type1 Mucopolysaccharidosis - Drug Profile
Product Description
Mechanism of Action
R&D Progress
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Recent Pipeline Updates
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Dormant Projects
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Product Development Milestones
Featured News & Press Releases
Apr 08, 2015: ArmaGen Announces FDA Acceptance of IND Application for AGT-181 for the Treatment of Hurler Syndrome
Dec 19, 2014: Translarna Granted Orphan Drug Designation in the U.S. and Europe for the Treatment of Mucopolysaccharidosis I
Jul 10, 2012: Athersys Receives Orphan Drug Designation For MultiStem In Hurler’s Syndrome
Apr 20, 2012: Athersys Announces Publication Of Results Of Preclinical Study Of MultiStem In Cell Transplant
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
List of Tables
Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ), H2 2015
Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Comparative Analysis, H2 2015
Number of Products under Development by Companies, H2 2015
Number of Products under Investigation by Universities/Institutes, H2 2015
Comparative Analysis by Clinical Stage Development, H2 2015
Comparative Analysis by Early Stage Development, H2 2015
Products under Development by Companies, H2 2015
Products under Investigation by Universities/Institutes, H2 2015
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Amicus Therapeutics, Inc., H2 2015
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by AngioChem Inc., H2 2015
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by ArmaGen Technologies, Inc., H2 2015
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Athersys, Inc., H2 2015
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by biOasis Technologies Inc., H2 2015
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Fate Therapeutics, Inc., H2 2015
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by OPKO Health, Inc., H2 2015
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by PTC Therapeutics, Inc., H2 2015
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by RegenxBio Inc., H2 2015
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Sangamo BioSciences, Inc., H2 2015
Assessment by Monotherapy Products, H2 2015
Assessment by Combination Products, H2 2015
Number of Products by Stage and Target, H2 2015
Number of Products by Stage and Mechanism of Action, H2 2015
Number of Products by Stage and Route of Administration, H2 2015
Number of Products by Stage and Molecule Type, H2 2015
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) Therapeutics - Recent Pipeline Updates, H2 2015
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Dormant Projects, H2 2015
List of Figures
Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ), H2 2015
Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Comparative Analysis, H2 2015
Number of Products under Development by Companies, H2 2015
Comparative Analysis by Clinical Stage Development, H2 2015
Comparative Analysis by Early Stage Products, H2 2015
Assessment by Monotherapy Products, H2 2015
Number of Products by Top 10 Targets, H2 2015
Number of Products by Stage and Top 10 Targets, H2 2015
Number of Products by Top 10 Mechanism of Actions, H2 2015
Number of Products by Stage and Top 10 Mechanism of Actions, H2 2015
Number of Products by Top 10 Routes of Administration, H2 2015
Number of Products by Stage and Top 10 Routes of Administration, H2 2015
Number of Products by Top 10 Molecule Types, H2 2015
Number of Products by Stage and Top 10 Molecule Types, H2 2015

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