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

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



Executive Summary

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

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Mucopolysaccharidosis II-Pipeline Review, H2 2017, provides an overview of the Mucopolysaccharidosis II (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.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Mucopolysaccharidosis II-Pipeline Review, H2 2017, provides comprehensive information on the therapeutics under development for Mucopolysaccharidosis II (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 (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 II, IND/CTA Filed, Preclinical, Discovery and Unknown stages are 4, 1, 3, 1 and 1 respectively. Similarly, the Universities portfolio in Phase I and Preclinical stages comprises 1 and 1 molecules, respectively.

Mucopolysaccharidosis II (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'sproprietary 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 Mucopolysaccharidosis II (Metabolic Disorders).

The pipeline guide reviews pipeline therapeutics for Mucopolysaccharidosis II (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 (Metabolic Disorders) therapeutics and enlists all their major and minor projects.

The pipeline guide evaluates Mucopolysaccharidosis II (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 (Metabolic Disorders)

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 Mucopolysaccharidosis II (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'smost promising pipeline therapeutics.

Formulate corrective measures for pipeline projects by understanding Mucopolysaccharidosis II (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.

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'sRGX-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

To know more information on Purchase by Section, please send a mail to sales@kenresearch.com

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

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

Single User License:
Report can be used by individual purchaser only

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Report can be shared by unlimited users within one corporate location, e.g. a regional office

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AngioChem Inc

ArmaGen Inc

Denali Therapeutics Inc

Green Cross Corp

JCR Pharmaceuticals Co Ltd

Laboratorios Del Dr Esteve SA

RegenxBio Inc

Sangamo Therapeutics Inc


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