Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline Review, H1 2018

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Date: 12-Jun-2018
No. of pages: 59
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Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline Review, H1 2018

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline Review, H1 2018, provides an overview of the Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) (Genetic 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 (MPS II) (Hunter Syndrome ) - Pipeline Review, H1 2018, provides comprehensive information on the therapeutics under development for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) (Genetic 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 ) (Genetic 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 1, 4, 1, 3 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 ) (Genetic 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.

Scope

- The pipeline guide provides a snapshot of the global therapeutic landscape of Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) (Genetic Disorders).

- The pipeline guide reviews pipeline therapeutics for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) (Genetic 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 ) (Genetic Disorders) therapeutics and enlists all their major and minor projects.

- The pipeline guide evaluates Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) (Genetic 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 ) (Genetic 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 (MPS II) (Hunter Syndrome ) (Genetic 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 ) (Genetic 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.

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline Review, H1 2018

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
GC Pharma
JCR Pharmaceuticals Co Ltd
Laboratorios Del Dr Esteve SA
RegenxBio Inc
Sangamo Therapeutics Inc
Shire Plc
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 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
idursulfase beta - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
idursulfase biosimilar - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
JR-141 - 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
Jun 01, 2018: JCR Announces Completion of PMDA Review of Phase 3 Clinical Trial of JR-141 for Hunter Syndrome
May 28, 2018: JCR Receives Approval in Brazil to Initiate Phase 2 Clinical Trial of JR-141 for Hunter Syndrome
May 02, 2018: REGENXBIO Receives FDA Fast Track Designation for RGX-121 Gene Therapy for the Treatment of Mucopolysaccharidosis Type II
Apr 04, 2018: Sangamo Announces Publication In Molecular Therapy Of Preclinical Study Data From MPS II In Vivo Genome Editing Program
Mar 27, 2018: JCR Announces Designation of JR-141 for Hunter Treatment under "SAKIGAKE Designation System" by Japan's Ministry of Health, Labour and Welfare
Mar 21, 2018: UNC Pediatrics Delivers Investigational Genome Editing Therapy in Clinical Trial for the Rare Hunter Syndrome
Feb 06, 2018: Sangamo Therapeutics Presents Initial Safety Data from CHAMPIONS Genome Editing Study for MPS II at WORLDSymposium
Jan 29, 2018: JCR to Present Data On JR-141 At The 14th Annual WORLD Symposium 2018
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 31, 2017: JCR Announces First Patient Dosed in Phase 1/2 Clinical Trial of JR-141 in Hunter Syndrome
Mar 01, 2017: Sangamo Therapeutics Receives Orphan Drug Designation from the FDA for SB-913 Genome Editing Treatment for MPS II
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer

List of Tables
Number of Products under Development for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ), H1 2018
Number of Products under Development by Companies, H1 2018
Number of Products under Development by Universities/Institutes, H1 2018
Products under Development by Companies, H1 2018
Products under Development by Universities/Institutes, H1 2018
Number of Products by Stage and Target, H1 2018
Number of Products by Stage and Mechanism of Action, H1 2018
Number of Products by Stage and Route of Administration, H1 2018
Number of Products by Stage and Molecule Type, H1 2018
Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by AngioChem Inc, H1 2018
Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by ArmaGen Inc, H1 2018
Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by Denali Therapeutics Inc, H1 2018
Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by GC Pharma, H1 2018
Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by JCR Pharmaceuticals Co Ltd, H1 2018
Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by Laboratorios Del Dr Esteve SA, H1 2018
Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by RegenxBio Inc, H1 2018
Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by Sangamo Therapeutics Inc, H1 2018
Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by Shire Plc, H1 2018
Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Dormant Projects, H1 2018
Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Discontinued Products, H1 2018

List of Figures
Number of Products under Development for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ), H1 2018
Number of Products under Development by Companies, H1 2018
Number of Products under Development by Universities/Institutes, H1 2018
Number of Products by Stage and Targets, H1 2018
Number of Products by Mechanism of Actions, H1 2018
Number of Products by Stage and Mechanism of Actions, H1 2018
Number of Products by Routes of Administration, H1 2018
Number of Products by Stage and Routes of Administration, H1 2018
Number of Products by Molecule Types, H1 2018
Number of Products by Stage and Molecule Types, H1 2018
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