Metachromatic Leukodystrophy (MLD)-Pipeline Review, H2 2016

Metachromatic Leukodystrophy (MLD)-Pipeline Review, H2 2016


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Executive Summary

Metachromatic Leukodystrophy (MLD)-Pipeline Review, H2 2016

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Metachromatic Leukodystrophy (MLD)-Pipeline Review, H2 2016, provides an overview of the Metachromatic Leukodystrophy (MLD) (Central Nervous System) pipeline landscape.

Metachromatic leukodystrophy is an inherited disorder characterized by the accumulation of fats called sulfatides in cells. Symptoms include vision problems leading to blindness, personality changes, and motor disturbances such as clumsiness, muscle weakness (hypotonia), rigidity, inability to coordinate movement (ataxia), and/or muscle spasms especially of the neck, spine, arms, and legs. Treatment for metachromatic leukodystrophy is symptomatic and supportive.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Metachromatic Leukodystrophy (MLD)-Pipeline Review, H2 2016, provides comprehensive information on the therapeutics under development for Metachromatic Leukodystrophy (MLD) (Central Nervous System), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Metachromatic Leukodystrophy (MLD) (Central Nervous System) pipeline guide also reviews of key players involved in therapeutic development for Metachromatic Leukodystrophy (MLD) and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase II, Phase I and Preclinical stages are 3, 2 and 1 respectively. Similarly, the Universities portfolio in Phase II and Phase I stages comprises 1 and 1 molecules, respectively.Metachromatic Leukodystrophy (MLD).

Metachromatic Leukodystrophy (MLD) (Central Nervous System) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Global Markets Direct's proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope

The pipeline guide provides a snapshot of the global therapeutic landscape of Metachromatic Leukodystrophy (MLD) (Central Nervous System).

The pipeline guide reviews pipeline therapeutics for Metachromatic Leukodystrophy (MLD) (Central Nervous System) by companies and universities/research institutes based on information derived from company and industry-specific sources.

The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.

The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.

The pipeline guide reviews key companies involved in Metachromatic Leukodystrophy (MLD) (Central Nervous System) therapeutics and enlists all their major and minor projects.

The pipeline guide evaluates Metachromatic Leukodystrophy (MLD) (Central Nervous System) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.

The pipeline guide encapsulates all the dormant and discontinued pipeline projects.

The pipeline guide reviews latest news related to pipeline therapeutics for Metachromatic Leukodystrophy (MLD) (Central Nervous System)

Reasons To Buy

Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.

Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.

Find and recognize significant and varied types of therapeutics under development for Metachromatic Leukodystrophy (MLD) (Central Nervous System).

Classify potential new clients or partners in the target demographic.

Develop tactical initiatives by understanding the focus areas of leading companies.

Plan mergers and acquisitions meritoriously by identifying key players and it's most promising pipeline therapeutics.

Formulate corrective measures for pipeline projects by understanding Metachromatic Leukodystrophy (MLD) (Central Nervous System) 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.

Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.



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Table of Contents

Table of Contents 2

List of Tables 4

List of Figures 4

Introduction 5

Global Markets Direct Report Coverage 5

Metachromatic Leukodystrophy (MLD) Overview 6

Therapeutics Development 7

Pipeline Products for Metachromatic Leukodystrophy (MLD)-Overview 7

Pipeline Products for Metachromatic Leukodystrophy (MLD)-Comparative Analysis 8

Metachromatic Leukodystrophy (MLD)-Therapeutics under Development by Companies 9

Metachromatic Leukodystrophy (MLD)-Therapeutics under Investigation by Universities/Institutes 10

Metachromatic Leukodystrophy (MLD)-Pipeline Products Glance 11

Clinical Stage Products 11

Early Stage Products 12

Metachromatic Leukodystrophy (MLD)-Products under Development by Companies 13

Metachromatic Leukodystrophy (MLD)-Products under Investigation by Universities/Institutes 14

Metachromatic Leukodystrophy (MLD)-Companies Involved in Therapeutics Development 15

ArmaGen Inc 15

GlaxoSmithKline Plc 16

Novartis AG 17

Nuo Therapeutics Inc 18

RegenxBio Inc 19

Shire Plc 20

Metachromatic Leukodystrophy (MLD)-Therapeutics Assessment 21

Assessment by Monotherapy Products 21

Assessment by Target 22

Assessment by Mechanism of Action 23

Assessment by Route of Administration 25

Assessment by Molecule Type 27

Drug Profiles 29

AGT-183-Drug Profile 29

Product Description 29

Mechanism Of Action 29

R&D Progress 29

ALD-601-Drug Profile 30

Product Description 30

Mechanism Of Action 30

R&D Progress 30

DUOC-01-Drug Profile 31

Product Description 31

Mechanism Of Action 31

R&D Progress 31

Gene Therapy to Activate Arylsulfatase A for Metachromatic Leukodystrophy-Drug Profile 32

Product Description 32

Mechanism Of Action 32

R&D Progress 32

GSK-2696274-Drug Profile 34

Product Description 34

Mechanism Of Action 34

R&D Progress 34

HSC-835-Drug Profile 35

Product Description 35

Mechanism Of Action 35

R&D Progress 35

SHP-611-Drug Profile 36

Product Description 36

Mechanism Of Action 36

R&D Progress 36

Stem Cell Therapy to Activate Arylsulfatase A and ABCD1 for Metachromatic Leukodystrophy and Adrenoleukodystrophy-Drug Profile 37

Product Description 37

Mechanism Of Action 37

R&D Progress 37

Metachromatic Leukodystrophy (MLD)-Dormant Projects 38

Metachromatic Leukodystrophy (MLD)-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 Figures

Number of Products under Development for Metachromatic Leukodystrophy (MLD), H2 2016 7

Number of Products under Development for Metachromatic Leukodystrophy (MLD)-Comparative Analysis, H2 2016 8

Number of Products under Development by Companies, H2 2016 9

Number of Products under Investigation by Universities/Institutes, H2 2016 10

Comparative Analysis by Clinical Stage Development, H2 2016 11

Assessment by Monotherapy Products, H2 2016 21

Number of Products by Stage and Targets, H2 2016 22

Number of Products by Mechanism of Actions, H2 2016 23

Number of Products by Stage and Mechanism of Actions, H2 2016 23

Number of Products by Routes of Administration, H2 2016 25

Number of Products by Stage and Top 10 Routes of Administration, H2 2016 25

Number of Products by Molecule Types, H2 2016 27

Number of Products by Stage and Molecule Types, H2 2016 27

List of Tables

Number of Products under Development for Metachromatic Leukodystrophy (MLD), H2 2016 7

Number of Products under Development for Metachromatic Leukodystrophy (MLD)-Comparative Analysis, H2 2016 8

Number of Products under Development by Companies, H2 2016 9

Number of Products under Investigation by Universities/Institutes, H2 2016 10

Comparative Analysis by Clinical Stage Development, H2 2016 11

Comparative Analysis by Early Stage Development, H2 2016 12

Products under Development by Companies, H2 2016 13

Products under Investigation by Universities/Institutes, H2 2016 14

Metachromatic Leukodystrophy (MLD)-Pipeline by ArmaGen Inc, H2 2016 15

Metachromatic Leukodystrophy (MLD)-Pipeline by GlaxoSmithKline Plc, H2 2016 16

Metachromatic Leukodystrophy (MLD)-Pipeline by Novartis AG, H2 2016 17

Metachromatic Leukodystrophy (MLD)-Pipeline by Nuo Therapeutics Inc, H2 2016 18

Metachromatic Leukodystrophy (MLD)-Pipeline by RegenxBio Inc, H2 2016 19

Metachromatic Leukodystrophy (MLD)-Pipeline by Shire Plc, H2 2016 20

Assessment by Monotherapy Products, H2 2016 21

Number of Products by Stage and Target, H2 2016 22

Number of Products by Stage and Mechanism of Action, H2 2016 24

Number of Products by Stage and Route of Administration, H2 2016 26

Number of Products by Stage and Molecule Type, H2 2016 28

Metachromatic Leukodystrophy (MLD)-Dormant Projects, H2 2016 38

Metachromatic Leukodystrophy (MLD)-Discontinued Products, H2 2016 39

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ArmaGen Inc, GlaxoSmithKline Plc, Novartis AG, Nuo Therapeutics Inc, RegenxBio Inc, Shire Plc

Metachromatic Leukodystrophy (MLD) Therapeutic Products under Development, Key Players in Metachromatic Leukodystrophy (MLD) Therapeutics, Metachromatic Leukodystrophy (MLD) Pipeline Overview, Metachromatic Leukodystrophy (MLD) Pipeline, Metachromatic Leukodystrophy (MLD) Pipeline Assessment

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