Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) – Pipeline Review, H2 2017

Global Markets Direct’s, ‘Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) – Pipeline Review, H2 2017’, provides an overview of the Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ), 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 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) 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 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome )

The report reviews pipeline therapeutics for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) 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 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) therapeutics and enlists all their major and minor projects

The report assesses Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) 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 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome )

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 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome )

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 Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) 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

AngioChem Inc

ArmaGen Inc

Denali Therapeutics Inc

Green Cross Corp

JCR Pharmaceuticals Co Ltd

Laboratorios Del Dr Esteve SA

RegenxBio Inc

Sangamo Therapeutics Inc

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) Overview

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) Therapeutics Development

Pipeline Overview

Pipeline by Companies

Pipeline by Universities/Institutes

Products under Development by Companies

Products under Development by Universities/Institutes

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) Therapeutics Assessment

Assessment by Target

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) Companies Involved in Therapeutics Development

AngioChem Inc

ArmaGen Inc

Denali Therapeutics Inc

Green Cross Corp

JCR Pharmaceuticals Co Ltd

Laboratorios Del Dr Esteve SA

RegenxBio Inc

Sangamo Therapeutics Inc

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) Drug Profiles

AGT-182 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

DUOC-01 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

EGT-301 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy to Activate Iduronate 2 Sulfatase for Mucopolysaccharidosis II Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Iduronate 2 Sulfatase Replacement for Mucopolysaccharidosis II Drug Profile

Product Description

Mechanism Of Action

R&D Progress

idursulfase beta Drug Profile

Product Description

Mechanism Of Action

R&D Progress

JR-032 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

JR-141 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

pentosan polysulfate sodium Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Recombinant Iduronate 2-Sulfatase Replacement for Mucopolysaccharidosis II Drug Profile

Product Description

Mechanism Of Action

R&D Progress

RGX-121 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

SB-913 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) Dormant Projects

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) Discontinued Products

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) Product Development Milestones

Featured News & Press Releases

Dec 19, 2017: REGENXBIO Announces IND Active for Phase I/II Trial of RGX-121 to Treat Mucopolysaccharidosis Type II

Dec 07, 2017: Sangamo Announces EMA Recommendation of Orphan Medicinal Product Designation for Investigational Genome Editing Treatment For MPS II

Nov 15, 2017: Sangamo Announces Treatment of First Patient in Landmark Phase 1/2 Clinical Trial Evaluating In Vivo Genome Editing for MPS II

Jul 13, 2017: Sangamo Receives Fast Track Designation From The FDA For SB-913

May 04, 2017: Sangamo Therapeutics Announces Rare Pediatric Disease Designation for SB-913 In Vivo Genome Editing Treatment for MPS II

Mar 01, 2017: Sangamo Therapeutics Receives Orphan Drug Designation from the FDA for SB-913 Genome Editing Treatment for MPS II

Feb 08, 2017: Sangamo Therapeutics Announces Data on SB-913 at The 13th Annual WORLDSymposium Meeting

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

Sep 13, 2016: REGENXBIO Publishes Data from Ongoing Preclinical Studies of NAV Gene Therapy RGX-121

Jul 05, 2016: REGENXBIO Provides Update On Gene Therapy Development Program RGX-121

Jun 20, 2016: Sangamo BioSciences Announces FDA Clearance of Investigational New Drug Application for ZFN-Mediated Genome Editing Treatment of MPS II

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

Feb 25, 2016: ESTEVE Provides Update On EGT-301 For Hunter Syndrome

Oct 02, 2013: Green Cross Exports Orphan Disease Drug Hunterase to Algeria

Apr 08, 2013: Green Cross Announces Publication Of Clinical Trial Paper On Hunter Syndrome Drug Hunterase In International Scientific Press

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

List of Tables

List of Tables

Number of Products under Development for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ), H2 2017

Number of Products under Development by Companies, H2 2017

Number of Products under Development by Universities/Institutes, H2 2017

Products under Development by Companies, H2 2017

Products under Development by Universities/Institutes, H2 2017

Number of Products by Stage and Target, H2 2017

Number of Products by Stage and Mechanism of Action, H2 2017

Number of Products by Stage and Route of Administration, H2 2017

Number of Products by Stage and Molecule Type, H2 2017

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) Pipeline by AngioChem Inc, H2 2017

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) Pipeline by ArmaGen Inc, H2 2017

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) Pipeline by Denali Therapeutics Inc, H2 2017

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) Pipeline by Green Cross Corp, H2 2017

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) Pipeline by JCR Pharmaceuticals Co Ltd, H2 2017

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) Pipeline by Laboratorios Del Dr Esteve SA, H2 2017

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) Pipeline by RegenxBio Inc, H2 2017

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) Pipeline by Sangamo Therapeutics Inc, H2 2017

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) Dormant Projects, H2 2017

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) Discontinued Products, H2 2017

List of Figures

List of Figures

Number of Products under Development for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ), H2 2017

Number of Products under Development by Companies, H2 2017

Number of Products under Development by Universities/Institutes, H2 2017

Number of Products by Stage and Targets, H2 2017

Number of Products by Mechanism of Actions, H2 2017

Number of Products by Stage and Mechanism of Actions, H2 2017

Number of Products by Routes of Administration, H2 2017

Number of Products by Stage and Routes of Administration, H2 2017

Number of Products by Molecule Types, H2 2017

Number of Products by Stage and Molecule Types, H2 2017

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