Metachromatic Leukodystrophy (MLD) - Pipeline Review, H2 2016

 


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Abstract

 
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.  

Description

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.
 

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Report Source Information

Supplied by: Research and Experts 
Date Published: 2016-12-14 

Report: Metachromatic Leukodystrophy (MLD) - Pipeline Review, H2 2016

 

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
 

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)

 

Highlights

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.

 

Classification

Language Code: EN 
Region: Global 
Report Type: Market Report 
Industry: Life Sciences 
Sector: Central Nervous System 
Pages: 41 

Companies Featured

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

 

Reasons to buy this report


- 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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Metachromatic Leukodystrophy (MLD) - Pipeline Review, H2 2016

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Regional Information

Taxonomy

Branch ID: 1591

Taxonomy Location

The taxonomy on the left shows the ancestors of the Global node. This provides an insight into parent categories that may provide reports or articles that may compliment this report. The number in brackets after the node name indicates the number of reports that Research and Experts currently have in the parent category.

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Global

The global economy is the economy of all the world's countries. The total world population now stands at over 7 billion and the global unemployment rate is 9% with approximately half the world's population paid less than $2 a day. 

The global economy is picking up again after five years of uncertaintity and slow economic growth. The IMF is predicting global growth of 3.7% in 2014 rising to 3.9% in 2015. However, much uncertainity remains. Low inflation in developed economies means that these economies take longer to deal with debt burdens and are at increases risk of deflation. 

The second half of 2013 saw growth in the US and European economies (particularly UK, Germany and France).  

Growth in the US has been helped by cheaper energy from fracking , renewables and more oil and gas exploration. This cheaper energy is bring manufacturing jobs back to the US and helping to boost domestic demand. Recent winter weather conditions have hampered growth in the construction sector and have slowed demand in the construction equipment sector which saw exports drop by 25% in 2013. Growth should pick up in this sector with continued federal investment in infrastructure, new residential house builds and refurbishment and the energy boom.

In Europe growth is expected to be uneven with growth being slow in stressed economies such as Portugal and Greece but confidence picking up in UK, Ireland and even Spain showing some improvement.

Developing economies such as China and India will continue to have strong growth in 2014 and 2015. China has benefitted from increased investment with the Chinese govenment now focused on quality growth of 7.5% in 2014. India which is less reliant on exports expects growth of 5.4% in 2014 and 6.4% in 2015.

Much uncertainity still remains in the global economy with greater optimism needed to stabilise growth.

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IMF (International Monetary Fund)

 

 

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Country

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Industry Sector Information

Taxonomy

Branch ID: 7188

Taxonomy Location

The taxonomy on the left shows the ancestors of the Central Nervous System node. This provides an insight into parent categories that may provide reports or articles that may compliment this report. The number in brackets after the node name indicates the number of reports that Research and Experts currently have in the parent category.

Clicking on a parent category will present the list of reports in the parent node in a separate window.

Central Nervous System

Central Nervous System

 

Healthcare

The healthcare industry is changing rapidly due to changes in legislation (US Affordable Healthcare Act),  advances in IT and more pressures on governments and healthcare providers due to rising costs. 

According to a recent report by the OECD healthcare spending has fallen in one third of OECD member states due to the credit crunch and members are facing up to the challenge of providing greater care with fewer resources. Spending on healthcare has increased in Japan, Israel and Korea while it has dropped dramatically in Greece and Ireland.

Useful Links

World Health Organisation 

American Healthcare Association

The British Healthcare Business Intelligence Association

OECD, Health Policies and Data

Healthcare

Life Sciences

The Life Sciences industry includes biotechnology, healthcare, pharmaceuticals, medical devices and diagnostics. The industry is dominated by US companies requiring considerable investment in research and development to stay competitive.  While US companies dominate this space, competition is emerging from smaller companies in Europe and Asia. 

Companies trying to stay competitive or enter new markets in life sciences consult our reports. For example, Plimsoll, in their recent review of global antibiotic manufacturers have reviewed 150 of the largest manufacturers including analysis of  ANTIBIOTICE SA, BAYER HEALTHCARE MANUFACTURING SRL and CIPLA LTD. 

Our reports are a requirement for companies looking for the best competitive intelligence in the life sciences sector providing information on market size, rankings, best trading partners and financial analysis. 

Useful Links

Analytical Life Sciences and Diagnostics Association

German Life Sciences Association

 

Life Sciences

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