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Metachromatic Leukodystrophy (MLD) - Pipeline Review, H2 2017

Published By :

Global Markets Direct

Published Date : Dec 2017

Category :

Pharmaceutical

No. of Pages : 33 Pages

Metachromatic Leukodystrophy (MLD) - Pipeline Review, H2 2017

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Metachromatic Leukodystrophy (MLD) - Pipeline Review, H2 2017, 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 2017, 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 III, Phase II and Preclinical stages are 1, 2 and 2 respectively. Similarly, the Universities portfolio in Phase II and Phase I stages comprises 1 and 1 molecules, respectively.

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 Directs 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 its 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.
Table of Contents
Table of Contents 2
List of Tables 3
List of Figures 3
Introduction 4
Global Markets Direct Report Coverage 4
Metachromatic Leukodystrophy (MLD) - Overview 5
Metachromatic Leukodystrophy (MLD) - Therapeutics Development 6
Pipeline Overview 6
Pipeline by Companies 7
Pipeline by Universities/Institutes 8
Products under Development by Companies 9
Products under Development by Universities/Institutes 10
Metachromatic Leukodystrophy (MLD) - Therapeutics Assessment 11
Assessment by Target 11
Assessment by Mechanism of Action 12
Assessment by Route of Administration 14
Assessment by Molecule Type 16
Metachromatic Leukodystrophy (MLD) - Companies Involved in Therapeutics Development 18
ArmaGen Inc 18
GlaxoSmithKline Plc 18
Recursion Pharmaceuticals Inc 18
RegenxBio Inc 19
Shire Plc 19
Metachromatic Leukodystrophy (MLD) - Drug Profiles 21
AGT-183 - Drug Profile 21
Product Description 21
Mechanism Of Action 21
R&D Progress 21
DUOC-01 - Drug Profile 22
Product Description 22
Mechanism Of Action 22
R&D Progress 22
Gene Therapy to Activate Arylsulfatase A for Metachromatic Leukodystrophy - Drug Profile 23
Product Description 23
Mechanism Of Action 23
R&D Progress 23
GSK-2696274 - Drug Profile 25
Product Description 25
Mechanism Of Action 25
R&D Progress 25
SHP-611 - Drug Profile 26
Product Description 26
Mechanism Of Action 26
R&D Progress 26
Small Molecules for Metachromatic Leukodystrophy - Drug Profile 28
Product Description 28
Mechanism Of Action 28
R&D Progress 28
Stem Cell Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy - Drug Profile 29
Product Description 29
Mechanism Of Action 29
R&D Progress 29
Metachromatic Leukodystrophy (MLD) - Dormant Projects 30
Metachromatic Leukodystrophy (MLD) - Discontinued Products 31
Appendix 32
Methodology 32
Coverage 32
Secondary Research 32
Primary Research 32
Expert Panel Validation 32
Contact Us 32
Disclaimer 33

List of Tables
Number of Products under Development for Metachromatic Leukodystrophy (MLD), H2 2017 6
Number of Products under Development by Companies, H2 2017 7
Number of Products under Development by Universities/Institutes, H2 2017 8
Products under Development by Companies, H2 2017 9
Products under Development by Universities/Institutes, H2 2017 10
Number of Products by Stage and Target, H2 2017 11
Number of Products by Stage and Mechanism of Action, H2 2017 13
Number of Products by Stage and Route of Administration, H2 2017 15
Number of Products by Stage and Molecule Type, H2 2017 17
Metachromatic Leukodystrophy (MLD) - Pipeline by ArmaGen Inc, H2 2017 18
Metachromatic Leukodystrophy (MLD) - Pipeline by GlaxoSmithKline Plc, H2 2017 18
Metachromatic Leukodystrophy (MLD) - Pipeline by Recursion Pharmaceuticals Inc, H2 2017 19
Metachromatic Leukodystrophy (MLD) - Pipeline by RegenxBio Inc, H2 2017 19
Metachromatic Leukodystrophy (MLD) - Pipeline by Shire Plc, H2 2017 20
Metachromatic Leukodystrophy (MLD) - Dormant Projects, H2 2017 30
Metachromatic Leukodystrophy (MLD) - Discontinued Products, H2 2017 31

List of Figures
Number of Products under Development for Metachromatic Leukodystrophy (MLD), H2 2017 6
Number of Products under Development by Companies, H2 2017 7
Number of Products under Development by Universities/Institutes, H2 2017 8
Number of Products by Stage and Targets, H2 2017 11
Number of Products by Mechanism of Actions, H2 2017 12
Number of Products by Stage and Mechanism of Actions, H2 2017 12
Number of Products by Routes of Administration, H2 2017 14
Number of Products by Stage and Routes of Administration, H2 2017 14
Number of Products by Molecule Types, H2 2017 16
Number of Products by Stage and Molecule Types, H2 2017 16

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