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

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


  • Products Id :- GMDHC7005IDB
  • |
  • Pages: 77
  • |
  • August 2015
  • |
  • |
  Request for Sample Report

Executive Summary

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

Table of Contents

Table of Contents 2

List of Tables 5

List of Figures 6

Introduction 7

Global Markets Direct Report Coverage 7

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) Overview 8

Therapeutics Development 9

Pipeline Products for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome )-Overview 9

Pipeline Products for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome )-Comparative Analysis 10

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome )-Therapeutics under Development by Companies 11

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome )-Therapeutics under Investigation by Universities/Institutes 12

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome )-Pipeline Products Glance 13

Clinical Stage Products 13

Early Stage Products 14

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome )-Products under Development by Companies 15

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome )-Products under Investigation by Universities/Institutes 16

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome )-Companies Involved in Therapeutics Development 17

Amicus Therapeutics, Inc. 17

AngioChem Inc. 18

ArmaGen Technologies, Inc. 19

Athersys, Inc. 20

biOasis Technologies Inc. 21

Fate Therapeutics, Inc. 22

OPKO Health, Inc. 23

PTC Therapeutics, Inc. 24

RegenxBio Inc. 25

Sangamo BioSciences, Inc. 26

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome )-Therapeutics Assessment 27

Assessment by Monotherapy Products 27

Assessment by Combination Products 28

Assessment by Target 29

Assessment by Mechanism of Action 31

Assessment by Route of Administration 33

Assessment by Molecule Type 35

Drug Profiles 37

AGT-181-Drug Profile 37

Product Description 37

Mechanism of Action 37

R&D Progress 37

ataluren-Drug Profile 38

Product Description 38

Mechanism of Action 38

R&D Progress 38

Cell Therapy for Mucopolysaccharidosis I-Drug Profile 41

Product Description 41

Mechanism of Action 41

R&D Progress 41

Cell Therapy to Activate Iduronidase for Hurler Syndrome-Drug Profile 42

Product Description 42

Mechanism of Action 42

R&D Progress 42

FT-1050-Drug Profile 43

Product Description 43

Mechanism of Action 43

R&D Progress 43

laronidase + chaperone-Drug Profile 46

Product Description 46

Mechanism of Action 46

R&D Progress 46

MultiStem-Drug Profile 47

Product Description 47

Mechanism of Action 47

R&D Progress 47

Oligonucleotide for Mucopolysaccharidosis I-Drug Profile 51

Product Description 51

Mechanism of Action 51

R&D Progress 51

Recombinant Enzyme to Replace Alpha-L-Iduronidase for Hurler Syndrome-Drug Profile 52

Product Description 52

Mechanism of Action 52

R&D Progress 52

Recombinant Enzyme to Target LRP1 for Mucopolysaccharidosis I-Drug Profile 53

Product Description 53

Mechanism of Action 53

R&D Progress 53

RGX-111-Drug Profile 54

Product Description 54

Mechanism of Action 54

R&D Progress 54

SB-318-Drug Profile 55

Product Description 55

Mechanism of Action 55

R&D Progress 55

Stem Cell Therapy for Type1 Mucopolysaccharidosis-Drug Profile 56

Product Description 56

Mechanism of Action 56

R&D Progress 56

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome )-Recent Pipeline Updates 57

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome )-Dormant Projects 72

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome )-Product Development Milestones 73

Featured News & Press Releases 73

Apr 08, 2015: ArmaGen Announces FDA Acceptance of IND Application for AGT-181 for the Treatment of Hurler Syndrome 73

Dec 19, 2014: Translarna Granted Orphan Drug Designation in the U.S. and Europe for the Treatment of Mucopolysaccharidosis I 73

Jul 10, 2012: Athersys Receives Orphan Drug Designation For MultiStem In Hurler's Syndrome 74

Apr 20, 2012: Athersys Announces Publication Of Results Of Preclinical Study Of MultiStem In Cell Transplant 75

Appendix 76

Methodology 76

Coverage 76

Secondary Research 76

Primary Research 76

Expert Panel Validation 76

Contact Us 76

Disclaimer 77

List of Tables

Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ), H2 2015 9

Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome )-Comparative Analysis, H2 2015 10

Number of Products under Development by Companies, H2 2015 11

Number of Products under Investigation by Universities/Institutes, H2 2015 12

Comparative Analysis by Clinical Stage Development, H2 2015 13

Comparative Analysis by Early Stage Development, H2 2015 14

Products under Development by Companies, H2 2015 15

Products under Investigation by Universities/Institutes, H2 2015 16

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome )-Pipeline by Amicus Therapeutics, Inc., H2 2015 17

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome )-Pipeline by AngioChem Inc., H2 2015 18

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome )-Pipeline by ArmaGen Technologies, Inc., H2 2015 19

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome )-Pipeline by Athersys, Inc., H2 2015 20

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome )-Pipeline by biOasis Technologies Inc., H2 2015 21

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome )-Pipeline by Fate Therapeutics, Inc., H2 2015 22

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome )-Pipeline by OPKO Health, Inc., H2 2015 23

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome )-Pipeline by PTC Therapeutics, Inc., H2 2015 24

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome )-Pipeline by RegenxBio Inc., H2 2015 25

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome )-Pipeline by Sangamo BioSciences, Inc., H2 2015 26

Assessment by Monotherapy Products, H2 2015 27

Assessment by Combination Products, H2 2015 28

Number of Products by Stage and Target, H2 2015 30

Number of Products by Stage and Mechanism of Action, H2 2015 32

Number of Products by Stage and Route of Administration, H2 2015 34

Number of Products by Stage and Molecule Type, H2 2015 36

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) Therapeutics-Recent Pipeline Updates, H2 2015 57

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome )-Dormant Projects, H2 2015 72

List of Figures

Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ), H2 2015 9

Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome )-Comparative Analysis, H2 2015 10

Number of Products under Development by Companies, H2 2015 11

Comparative Analysis by Clinical Stage Development, H2 2015 13

Comparative Analysis by Early Stage Products, H2 2015 14

Assessment by Monotherapy Products, H2 2015 27

Number of Products by Top 10 Targets, H2 2015 29

Number of Products by Stage and Top 10 Targets, H2 2015 29

Number of Products by Top 10 Mechanism of Actions, H2 2015 31

Number of Products by Stage and Top 10 Mechanism of Actions, H2 2015 31

Number of Products by Top 10 Routes of Administration, H2 2015 33

Number of Products by Stage and Top 10 Routes of Administration, H2 2015 33

Number of Products by Top 10 Molecule Types, H2 2015 35

Number of Products by Stage and Top 10 Molecule Types, H2 2015 35

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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 ) Therapeutic Products under Development, Key Players in Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) Therapeutics, Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) Pipeline Overview, Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) Pipeline, Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) Pipeline Assessment


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