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Leukodystrophies-Pipeline Review, H1 2015

Leukodystrophies-Pipeline Review, H1 2015

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Executive Summary

Leukodystrophies-Pipeline Review, H1 2015


Global Markets Direct's, 'Leukodystrophies-Pipeline Review, H1 2015', provides an overview of the Leukodystrophies's therapeutic pipeline.

This report provides comprehensive information on the therapeutic development for Leukodystrophies, 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 Leukodystrophies 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.


The report provides a snapshot of the global therapeutic landscape of Leukodystrophies

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 Leukodystrophies and enlists all their major and minor projects

The report summarizes all the dormant and discontinued pipeline projects

A review of the Leukodystrophies 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 Leukodystrophies 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 Leukodystrophies

Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline

Devise corrective measures for pipeline projects by understanding Leukodystrophies 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

Leukodystrophies Overview 6

Therapeutics Development 7

Pipeline Products for Leukodystrophies-Overview 7

Pipeline Products for Leukodystrophies-Comparative Analysis 8

Leukodystrophies-Therapeutics under Development by Companies 9

Leukodystrophies-Pipeline Products Glance 10

Clinical Stage Products 10

Early Stage Products 11

Leukodystrophies-Products under Development by Companies 12

Leukodystrophies-Companies Involved in Therapeutics Development 13

ArmaGen Technologies, Inc. 13

Fate Therapeutics, Inc. 14

GlaxoSmithKline plc 15

Phenomenome Discoveries, Inc. 16

ReGenX Biosciences, LLC 17

Shire Plc 18

Leukodystrophies-Therapeutics Assessment 19

Assessment by Monotherapy Products 19

Assessment by Target 20

Assessment by Mechanism of Action 21

Assessment by Route of Administration 22

Assessment by Molecule Type 24

Drug Profiles 26

AGT-183-Drug Profile 26

Product Description 26

Mechanism of Action 26

R&D Progress 26

FT-1050-Drug Profile 28

Product Description 28

Mechanism of Action 28

R&D Progress 28

Gene Therapy to Activate Arylsulfatase A for Metachromatic Leukodystrophy-Drug Profile 31

Product Description 31

Mechanism of Action 31

R&D Progress 31

GSK-2696274-Drug Profile 32

Product Description 32

Mechanism of Action 32

R&D Progress 32

PPI-1011-Drug Profile 33

Product Description 33

Mechanism of Action 33

R&D Progress 33

SHP-611-Drug Profile 34

Product Description 34

Mechanism of Action 34

R&D Progress 34

Leukodystrophies-Recent Pipeline Updates 35

Leukodystrophies-Dormant Projects 38

Leukodystrophies-Discontinued Products 39

Appendix 40

Methodology 40

Coverage 40

Secondary Research 40

Primary Research 40

Expert Panel Validation 40

Contact Us 40

Disclaimer 41

List of Tables

Number of Products under Development for Leukodystrophies, H1 2015 7

Number of Products under Development for Leukodystrophies-Comparative Analysis, H1 2015 8

Number of Products under Development by Companies, H1 2015 9

Comparative Analysis by Clinical Stage Development, H1 2015 10

Comparative Analysis by Early Stage Development, H1 2015 11

Products under Development by Companies, H1 2015 12

Leukodystrophies-Pipeline by ArmaGen Technologies, Inc., H1 2015 13

Leukodystrophies-Pipeline by Fate Therapeutics, Inc., H1 2015 14

Leukodystrophies-Pipeline by GlaxoSmithKline plc, H1 2015 15

Leukodystrophies-Pipeline by Phenomenome Discoveries, Inc., H1 2015 16

Leukodystrophies-Pipeline by ReGenX Biosciences, LLC, H1 2015 17

Leukodystrophies-Pipeline by Shire Plc, H1 2015 18

Assessment by Monotherapy Products, H1 2015 19

Number of Products by Stage and Target, H1 2015 20

Number of Products by Stage and Mechanism of Action, H1 2015 21

Number of Products by Stage and Route of Administration, H1 2015 23

Number of Products by Stage and Molecule Type, H1 2015 25

Leukodystrophies Therapeutics-Recent Pipeline Updates, H1 2015 35

Leukodystrophies-Dormant Projects, H1 2015 38

Leukodystrophies-Discontinued Products, H1 2015 39

List of Figures

Number of Products under Development for Leukodystrophies, H1 2015 7

Number of Products under Development for Leukodystrophies-Comparative Analysis, H1 2015 8

Number of Products under Development by Companies, H1 2015 9

Comparative Analysis by Clinical Stage Development, H1 2015 10

Assessment by Monotherapy Products, H1 2015 19

Number of Products by Stage and Top 10 Targets, H1 2015 20

Number of Products by Stage and Top 10 Mechanism of Actions, H1 2015 21

Number of Products by Top 10 Routes of Administration, H1 2015 22

Number of Products by Stage and Top 10 Routes of Administration, H1 2015 23

Number of Products by Top 10 Molecule Types, H1 2015 24

Number of Products by Stage and Top 10 Molecule Types, H1 2015 25

Single User License:
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Corporate User License:
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ArmaGen Technologies, Inc.

Fate Therapeutics, Inc.

GlaxoSmithKline plc

Phenomenome Discoveries, Inc.

ReGenX Biosciences, LLC

Shire Plc

Leukodystrophies Therapeutic Products under Development, Key Players in Leukodystrophies Therapeutics, Leukodystrophies Pipeline Overview, Leukodystrophies Pipeline, Leukodystrophies Pipeline Assessment

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