Metachromatic Leukodystrophy (MLD) – Pipeline Review, H2 2016

Global Markets Direct’s, ‘Metachromatic Leukodystrophy (MLD) – Pipeline Review, H2 2016’, provides an overview of the Metachromatic Leukodystrophy (MLD) pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Metachromatic Leukodystrophy (MLD), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Metachromatic Leukodystrophy (MLD) and features dormant 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, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report 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.

Note: Certain sections in the report may be removed or altered based on the availability and relevance of data.

Scope

The report provides a snapshot of the global therapeutic landscape of Metachromatic Leukodystrophy (MLD)

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

The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages

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

The report reviews key players involved Metachromatic Leukodystrophy (MLD) therapeutics and enlists all their major and minor projects

The report assesses Metachromatic Leukodystrophy (MLD) therapeutics based on drug target, mechanism of action (MoA), route of administration (RoA) and molecule type

The report summarizes all the dormant and discontinued pipeline projects

The report reviews latest news related to pipeline therapeutics for Metachromatic Leukodystrophy (MLD)

Reasons to buy

Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies

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

Identify and understand important and diverse types of therapeutics under development for Metachromatic Leukodystrophy (MLD)

Identify potential new clients or partners in the target demographic

Develop strategic initiatives by understanding the focus areas of leading companies

Plan mergers and acquisitions effectively by identifying key players and it’s most promising pipeline therapeutics

Devise corrective measures for pipeline projects by understanding Metachromatic Leukodystrophy (MLD) 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

Companies mentioned

ArmaGen Inc

GlaxoSmithKline Plc

Novartis AG

Nuo Therapeutics Inc

RegenxBio Inc

Shire Plc

Table of Contents

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 Tables

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

List of Figures

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

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