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

  • ID: 4576052
  • Drug Pipelines
  • 59 pages
  • Global Markets Direct
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  • AngioChem Inc
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  • GC Pharma
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Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline Review, H1 2018

Summary:

This 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:

This 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 the researcher'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 for Purchase:
  • 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.
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FEATURED COMPANIES

  • AngioChem Inc
  • ArmaGen Inc
  • Denali Therapeutics Inc
  • GC Pharma
  • RegenxBio Inc
  • Sangamo Therapeutics Inc
  • MORE
Introduction

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
Note: Product cover images may vary from those shown
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  • 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
Note: Product cover images may vary from those shown
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