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Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) - Drugs in Development, 2021

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    Report

  • 88 Pages
  • November 2021
  • Region: Global
  • Global Markets Direct
  • ID: 5509473
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The publisher's latest Pharmaceutical and Healthcare disease pipeline guide Mucopolysaccharidosis II - Drugs In Development, 2021, 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


The publisher's Pharmaceutical and Healthcare latest pipeline guide Mucopolysaccharidosis II - Drugs In Development, 2021, 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 Pre-Registration, Phase III, Phase II, Phase I, Preclinical and Discovery stages are 1, 1, 4, 2, 5 and 1 respectively. Similarly, the Universities portfolio in Phase II and Phase I 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 the publisher’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 (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’s most 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

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
  • AVROBIO Inc
  • Bioasis Technologies Inc
  • Denali Therapeutics Inc
  • Esteve Pharmaceuticals SA
  • GC Pharma
  • Generium
  • Homology Medicines Inc
  • Immusoft Corp
  • JCR Pharmaceuticals Co Ltd
  • RegenxBio Inc
  • ReqMed Co Ltd
  • Sigilon Therapeutics Inc
  • Takeda Pharmaceutical Co Ltd
  • Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Drug Profiles
  • AVRRD-05 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • DNL-310 - 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 for Mucopolysaccharidosis II - 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
  • GNR-055 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • HMI-203 - 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
  • pabinafusp alfa - 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
  • SIG-018 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • xB-3008 - 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
  • Nov 03, 2021: AVROBIO receives Rare Pediatric Disease Designation from the U.S. FDA for AVR-RD-05, a Gene Therapy for Mucopolysaccharidosis Type II (MPSII) or Hunter Syndrome
  • Nov 02, 2021: GC Green Cross designated as a European orphan drug for the treatment of severe Hunter syndrome
  • Oct 20, 2021: Homology Medicines announces presentation of data supporting clinical programs in MPS II and PKU, including nonclinical and patient-focused research, at American Society of Human Genetics Meeting
  • Oct 18, 2021: Homology medicines initiates clinical trial for HMI-203, a one-time investigational gene therapy candidate for adults with MPS II (Hunter Syndrome)
  • Oct 18, 2021: JCR Pharmaceuticals Co : EMA grants PRIME designation for JR-141 for the treatment of Mucopolysaccharidosis type II (Hunter Syndrome)
  • Oct 15, 2021: JR-141 (Pabinafusp Alfa) for Hunter syndrome notice on the publication of a nonclinical and clinical evidence in International Journal of Molecular Sciences
  • Jul 29, 2021: JCR Pharmaceuticals announces presentation at 16th International Symposium on MPS and Related Diseases.
  • Jul 29, 2021: JR-141 (Pabinafusp Alfa) for Hunter Syndrome notice on the publication of a case report in Japan in JIMD reports
  • May 19, 2021: JCR Pharmaceuticals: Launch of IZCARGO I.V. infusion 10mg (JR-141: Pabinafusp Alfa) for treatment of MPS II (Hunter Syndrome) in Japan
  • Apr 19, 2021: JR-141 (Pabinafusp Alfa) for Hunter Syndrome notice on the publication of the results of non-clinical trials in molecular genetics and metabolism report
  • Mar 23, 2021: JCR Pharmaceuticals announces approval of IZCARGO (pabinafusp alfa) for treatment of MPS II (Hunter syndrome) in Japan
  • Mar 03, 2021: GC Green Cross to ship'Hunterase ICV' in Japan
  • Feb 10, 2021: JCR Pharmaceuticals receives FDA IND clearance to initiate global phase 3 clinical trial of JR-141 for Mucopolysaccharidosis Type II (Hunter Syndrome)
  • Feb 10, 2021: FDA grants fast track designation for JR-141 for the treatment of mucopolysaccharidosis type II (Hunter syndrome)
  • Feb 08, 2021: Homology Medicines announces first presentation of data with HMI-203 in vivo gene therapy development candidate for Hunter syndrome

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 ), 2021
  • Number of Products under Development by Companies, 2021
  • Number of Products under Development by Universities/Institutes, 2021
  • Products under Development by Companies, 2021
  • Products under Development by Companies, 2021 (Contd..1)
  • Products under Development by Universities/Institutes, 2021
  • Number of Products by Stage and Target, 2021
  • Number of Products by Stage and Mechanism of Action, 2021
  • Number of Products by Stage and Route of Administration, 2021
  • Number of Products by Stage and Molecule Type, 2021
  • Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by AngioChem Inc, 2021
  • Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by AVROBIO Inc, 2021
  • Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by Bioasis Technologies Inc, 2021
  • Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by Denali Therapeutics Inc, 2021
  • Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by Esteve Pharmaceuticals SA, 2021
  • Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by GC Pharma, 2021
  • Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by Generium, 2021
  • Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by Homology Medicines Inc, 2021
  • Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by Immusoft Corp, 2021
  • Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by JCR Pharmaceuticals Co Ltd, 2021
  • Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by RegenxBio Inc, 2021
  • Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by ReqMed Co Ltd, 2021
  • Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by Sigilon Therapeutics Inc, 2021
  • Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline by Takeda Pharmaceutical Co Ltd, 2021
  • Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Dormant Projects, 2021
  • Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Discontinued Products, 2021

List of Figures
  • Number of Products under Development for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ), 2021
  • Number of Products under Development by Companies, 2021
  • Number of Products under Development by Universities/Institutes, 2021
  • Number of Products by Stage and Targets, 2021
  • Number of Products by Mechanism of Actions, 2021
  • Number of Products by Stage and Mechanism of Actions, 2021
  • Number of Products by Routes of Administration, 2021
  • Number of Products by Stage and Routes of Administration, 2021
  • Number of Products by Molecule Types, 2021
  • Number of Products by Stage and Molecule Types, 2021

Companies Mentioned

A selection of companies mentioned in this report includes:

  • AngioChem Inc
  • AVROBIO Inc
  • Bioasis Technologies Inc
  • Denali Therapeutics Inc
  • Esteve Pharmaceuticals SA
  • GC Pharma
  • Generium
  • Homology Medicines Inc
  • Immusoft Corp
  • JCR Pharmaceuticals Co Ltd
  • RegenxBio Inc
  • ReqMed Co Ltd
  • Sigilon Therapeutics Inc
  • Takeda Pharmaceutical Co Ltd