Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline Review, H2 2016

 


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Abstract

 
Mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, is a condition that affects many different parts of the body and occurs almost exclusively in males. Signs and symptoms include claw-like hands, protruding tongue, changing facial features, including thickening of the lips, tongue and nostrils and delayed development. Treatment includes bone marrow transplantation, enzyme therapy and gene therapy.  

Description

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline Review, H2 2016, provides an overview of the Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) (Metabolic Disorders) pipeline landscape.

Mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, is a condition that affects many different parts of the body and occurs almost exclusively in males. Signs and symptoms include claw-like hands, protruding tongue, changing facial features, including thickening of the lips, tongue and nostrils and delayed development. Treatment includes bone marrow transplantation, enzyme therapy and gene therapy.
 

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Report Source Information

Supplied by: Research and Experts 
Date Published: 2016-12-21 

Report: Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline Review, H2 2016

 

Contents

Table of Contents
Table of Contents 2
List of Tables 5
List of Figures 6
Introduction 7
Global Markets Direct Report Coverage 7
Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) Overview 8
Therapeutics Development 9
Pipeline Products for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Overview 9
Pipeline Products for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Comparative Analysis 10
Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Therapeutics under Development by Companies 11
Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Therapeutics under Investigation by Universities/Institutes 12
Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline Products Glance 13
Late Stage Products 13
Clinical Stage Products 14
Early Stage Products 15
Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Products under Development by Companies 16
Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Products under Investigation by Universities/Institutes 17
Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Companies Involved in Therapeutics Development 18
Alexion Pharmaceuticals Inc 18
AngioChem Inc 19
ArmaGen Inc 20
Bioasis Technologies Inc 21
Green Cross Corp 22
Inventiva 23
JCR Pharmaceuticals Co Ltd 24
Laboratorios Del Dr Esteve SA 25
RegenxBio Inc 26
Sangamo BioSciences Inc 27
Shire Plc 28
Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Therapeutics Assessment 29
Assessment by Monotherapy Products 29
Assessment by Target 30
Assessment by Mechanism of Action 32
Assessment by Route of Administration 34
Assessment by Molecule Type 36
Drug Profiles 38
AGT-182 - Drug Profile 38
Product Description 38
Mechanism Of Action 38
R&D Progress 38
DUOC-01 - Drug Profile 40
Product Description 40
Mechanism Of Action 40
R&D Progress 40
EGT-301 - Drug Profile 41
Product Description 41
Mechanism Of Action 41
R&D Progress 41
Gene Therapy to Activate Iduronate 2 Sulfatase for Mucopolysaccharidosis II - Drug Profile 42
Product Description 42
Mechanism Of Action 42
R&D Progress 42
idursulfase - Drug Profile 43
Product Description 43
Mechanism Of Action 43
R&D Progress 43
idursulfase beta - Drug Profile 45
Product Description 45
Mechanism Of Action 45
R&D Progress 45
JR-032 - Drug Profile 47
Product Description 47
Mechanism Of Action 47
R&D Progress 47
JR-141 - Drug Profile 48
Product Description 48
Mechanism Of Action 48
R&D Progress 48
MTf-I2S - Drug Profile 49
Product Description 49
Mechanism Of Action 49
R&D Progress 49
MTfp-I2S - Drug Profile 50
Product Description 50
Mechanism Of Action 50
R&D Progress 50
odiparcil - Drug Profile 51
Product Description 51
Mechanism Of Action 51
R&D Progress 51
pentosan polysulfate sodium - Drug Profile 52
Product Description 52
Mechanism Of Action 52
R&D Progress 52
Recombinant Enzymes for Pompe, Fabry and Hunter Diseases - Drug Profile 53
Product Description 53
Mechanism Of Action 53
R&D Progress 53
Recombinant Iduronate 2-Sulfatase Replacement for Mucopolysaccharidosis II - Drug Profile 54
Product Description 54
Mechanism Of Action 54
R&D Progress 54
RGX-121 - Drug Profile 55
Product Description 55
Mechanism Of Action 55
R&D Progress 55
SB-913 - Drug Profile 57
Product Description 57
Mechanism Of Action 57
R&D Progress 57
Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Dormant Projects 59
Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Discontinued Products 60
Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Product Development Milestones 61
Featured News & Press Releases 61
Oct 26, 2016: National Center for Child Health and Development Initiates Phase 1/2 Trial of Green Cross' idursulfase-beta ICV for the Treatment of Hunter Syndrome with Neurocognitive Decline 61
Sep 13, 2016: REGENXBIO Publishes Data from Ongoing Preclinical Studies of NAV Gene Therapy RGX-121 61
Jul 05, 2016: REGENXBIO Provides Update On Gene Therapy Development Program RGX-121 62
Jun 20, 2016: Sangamo BioSciences Announces FDA Clearance of Investigational New Drug Application for ZFN-Mediated Genome Editing Treatment of MPS II 62
Apr 19, 2016: Preclinical Data from REGENXBIO's RGX-121 Gene Therapy Program to be Presented at the American Society of Gene & Cell Therapy 19th Annual Meeting 63
Feb 25, 2016: ESTEVE Provides Update On EGT-301 For Hunter Syndrome 64
Jul 31, 2015: biOasis Announces the Successful Delivery to the CNS of an Enzyme used to Treat Hunter Syndrome 65
Sep 18, 2014: biOasis Initiates MPS II Study with Renowned Lysosomal Storage Disease Expert Dr. Maurizio Scarpa and The Brains for Brain Foundation 65
Oct 02, 2013: Green Cross Exports Orphan Disease Drug Hunterase to Algeria 66
Apr 08, 2013: Green Cross Announces Publication Of Clinical Trial Paper On Hunter Syndrome Drug Hunterase In International Scientific Press 67
Feb 18, 2013: Green Cross Receives FDA Orphan Drug Designation For Hunterase For Treatment Of Hunter Syndrome 68
Jan 10, 2012: Green Cross Receives Korean Approval For Hunterase 68
Oct 12, 2011: Green Cross Files Application For Korean Approval Of Hunterase For Treatment Of Hunter Syndrome 69
Appendix 70
Methodology 70
Coverage 70
Secondary Research 70
Primary Research 70
Expert Panel Validation 70
Contact Us 70
Disclaimer 71
 

List of Tables

List of Tables
Number of Products under Development for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ), H2 2016 9
Number of Products under Development for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Comparative Analysis, H2 2016 10
Number of Products under Development by Companies, H2 2016 11
Number of Products under Investigation by Universities/Institutes, H2 2016 12
Comparative Analysis by Late Stage Development, H2 2016 13
Comparative Analysis by Clinical Stage Development, H2 2016 14
Comparative Analysis by Early Stage Development, H2 2016 15
Products under Development by Companies, H2 2016 16
Products under Investigation by Universities/Institutes, H2 2016 17
Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by Alexion Pharmaceuticals Inc, H2 2016 18
Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by AngioChem Inc, H2 2016 19
Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by ArmaGen Inc, H2 2016 20
Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by Bioasis Technologies Inc, H2 2016 21
Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by Green Cross Corp, H2 2016 22
Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by Inventiva, H2 2016 23
Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by JCR Pharmaceuticals Co Ltd, H2 2016 24
Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by Laboratorios Del Dr Esteve SA, H2 2016 25
Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by RegenxBio Inc, H2 2016 26
Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by Sangamo BioSciences Inc, H2 2016 27
Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by Shire Plc, H2 2016 28
Assessment by Monotherapy Products, H2 2016 29
Number of Products by Stage and Target, H2 2016 31
Number of Products by Stage and Mechanism of Action, H2 2016 33
Number of Products by Stage and Route of Administration, H2 2016 35
Number of Products by Stage and Molecule Type, H2 2016 37
Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Dormant Projects, H2 2016 59
Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Discontinued Products, H2 2016 60
 

List of Figures

List of Figures
Number of Products under Development for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ), H2 2016 9
Number of Products under Development for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Comparative Analysis, H2 2016 10
Number of Products under Development by Companies, H2 2016 11
Number of Products under Investigation by Universities/Institutes, H2 2016 12
Comparative Analysis by Clinical Stage Development, H2 2016 14
Comparative Analysis by Early Stage Products, H2 2016 15
Assessment by Monotherapy Products, H2 2016 29
Number of Products by Targets, H2 2016 30
Number of Products by Stage and Targets, H2 2016 30
Number of Products by Mechanism of Actions, H2 2016 32
Number of Products by Stage and Mechanism of Actions, H2 2016 32
Number of Products by Routes of Administration, H2 2016 34
Number of Products by Stage and Routes of Administration, H2 2016 34
Number of Products by Molecule Types, H2 2016 36
Number of Products by Stage and Molecule Types, H2 2016 36
 

Scope


- The pipeline guide provides a snapshot of the global therapeutic landscape of Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) (Metabolic Disorders).
- The pipeline guide reviews pipeline therapeutics for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) (Metabolic Disorders) 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 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) (Metabolic Disorders) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) (Metabolic Disorders) 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 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) (Metabolic Disorders)

 

Highlights

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline Review, H2 2016, provides comprehensive information on the therapeutics under development for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) (Metabolic Disorders), 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 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) (Metabolic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) 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, IND/CTA Filed, Preclinical and Discovery stages are 2, 2, 1, 8 and 1 respectively. Similarly, the Universities portfolio in Phase I and Preclinical stages comprises 1 and 1 molecules, respectively.

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) (Metabolic Disorders) 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: Genetic Disorders 
Pages: 71 

Companies Featured

Alexion Pharmaceuticals Inc AngioChem Inc ArmaGen Inc Bioasis Technologies Inc Green Cross Corp Inventiva JCR Pharmaceuticals Co Ltd Laboratorios Del Dr Esteve SA RegenxBio Inc Sangamo BioSciences 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 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) (Metabolic Disorders).
- 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 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) (Metabolic Disorders) 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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Branch ID: 1591

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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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Branch ID: 7190

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Genetic Disorders

Genetic Disorders

 

Healthcare Disorders

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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.

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