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

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


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Executive Summary

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

Table of Contents 2

List of Tables 4

List of Figures 5

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

R&D Progress 34

AGT-181-Drug Profile 35

Product Description 35

Mechanism of Action 35

R&D Progress 35

ataluren-Drug Profile 36

Product Description 36

Mechanism of Action 36

R&D Progress 36

Cell Therapy for Mucopolysaccharidosis I-Drug Profile 39

Product Description 39

Mechanism of Action 39

R&D Progress 39

Cell Therapy to Activate Iduronidase for Hurler Syndrome-Drug Profile 40

Product Description 40

Mechanism of Action 40

R&D Progress 40

FT-1050-Drug Profile 41

Product Description 41

Mechanism of Action 41

R&D Progress 41

Gene Therapy to Activate Iduronidase for Hurler Syndrome-Drug Profile 44

Product Description 44

Mechanism of Action 44

R&D Progress 44

MultiStem-Drug Profile 45

Product Description 45

Mechanism of Action 45

R&D Progress 45

Oligonucleotide for Mucopolysaccharidosis I-Drug Profile 48

Product Description 48

Mechanism of Action 48

R&D Progress 48

Recombinant Enzyme to Replace Alpha-L-Iduronidase for Hurler Syndrome-Drug Profile 49

Product Description 49

Mechanism of Action 49

R&D Progress 49

Recombinant Enzyme to Replace Alpha-L-Iduronidase for Mucopolysaccharidosis I-Drug Profile 50

Product Description 50

Mechanism of Action 50

R&D Progress 50

Stem Cell Therapy for Type1 Mucopolysaccharidosis-Drug Profile 51

Product Description 51

Mechanism of Action 51

R&D 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 & 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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Amicus Therapeutics, Inc.

AngioChem Inc.

ArmaGen Technologies, Inc.

Athersys, Inc.

biOasis Technologies Inc.

Fate Therapeutics, Inc.

OPKO Health, Inc.

PTC Therapeutics, Inc.

ReGenX Biosciences, LLC

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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