Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

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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.

The Mucopolysaccharidosis type II (MPS II) (Hunter Syndrome) pipeline market research report  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 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 also provides an overview of key players involved in therapeutic development for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) and features dormant and discontinued projects.

MPS II Pipeline Products Market by Targets

The key target in the MPS II  pipeline products market is Iduronate 2 Sulfatase.

MPS II Pipeline Products Market Segmentation by Mechanisms of Action

The key mechanisms of action in the MPS II  pipeline products market are Iduronate 2 Sulfatase Activator, and Iduronate 2 Sulfatase Replacement. Iduronate 2 Sulfatase Activator is the leading mechanism of action in the MPS II pipeline products market.

MPS II Pipeline Products Market Analysis by Mechanisms of Action

MPS II Pipeline Products Market Analysis by Mechanisms of Action

For more mechanisms of action insights into the MPS II pipeline products market, download a free report sample

MPS II Pipeline Products Market Segmentation by Routes of Administration

The key routes of administration in the MPS II pipeline products market are intravenous, parenteral, subcutaneous, intracerebral, intracisternal, and intrathecal. Most of the products are administered through the intravenous route.

MPS II Pipeline Products Market Analysis by Routes of Administration

MPS II Pipeline Products Market Analysis by Routes of AdministrationFor more routes of administration insights into the MPS II pipeline products market, download a free report sample

MPS II Pipeline Products Market Segmentation by Molecule Types

The key molecule types in the MPS II pipeline products market are gene therapy, recombinant enzyme, gene-modified cell therapy, biologic, cell therapy, fusion protein, oligonucleotide, and polysaccharide. Gene therapy and recombinant enzyme are the leading molecule types in the MPS II pipeline products market.

MPS II Pipeline Products Market Analysis by Molecule Types

MPS II Pipeline Products Market Analysis by Molecule TypesFor more molecule type insights into the MPS II pipeline products market, download a free report sample

MPS II Pipeline Products Market - Competitive Landscape

Some of the leading companies in the MPS II pipeline products market are GC Biopharma Corp, AVROBIO Inc, Homology Medicines Inc, JCR Pharmaceuticals Co Ltd, AngioChem Inc, Apteeus SAS, Bioasis Technologies Inc, Biorchestra Co Ltd, Capsida Biotherapeutics Inc, and Denali Therapeutics Inc. GC Biopharma Corp is the leading company in the MPS II pipeline products market.

MPS II Pipeline Products Market Analysis by Companies

MPS II Pipeline Products Market Analysis by CompaniesTo know more about the leading players in the MPS II pipeline products market, download a free report sample

MPS II Pipeline Products Market Report Overview

Key Target Iduronate 2 Sulfatase
Key Mechanisms of Action Iduronate 2 Sulfatase Activator, and Iduronate 2 Sulfatase Replacement
Key Routes of Administration Intravenous, Parenteral, Subcutaneous, Intracerebral, Intracisternal, and Intrathecal
Key Molecule Types Gene Therapy, Recombinant Enzyme, Gene-Modified Cell Therapy, Biologic, Cell Therapy, Fusion Protein, Oligonucleotide, and Polysaccharide
Leading Companies GC Biopharma Corp, AVROBIO Inc, Homology Medicines Inc, JCR Pharmaceuticals Co Ltd, AngioChem Inc, Apteeus SAS, Bioasis Technologies Inc, Biorchestra Co Ltd, Capsida Biotherapeutics Inc, and Denali Therapeutics Inc

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.

AngioChem Inc
Apteeus SAS
AVROBIO Inc
Bioasis Technologies Inc
Biorchestra Co Ltd
Capsida Biotherapeutics Inc
Denali Therapeutics Inc
Esteve Pharmaceuticals SA
GC Biopharma Corp
Generium
Homology Medicines Inc
Immusoft Corp
JCR Pharmaceuticals Co Ltd
RegenxBio Inc
ReqMed Co Ltd
Sigilon 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

Apteeus SAS

AVROBIO Inc

Bioasis Technologies Inc

Biorchestra Co Ltd

Capsida Biotherapeutics Inc

Denali Therapeutics Inc

Esteve Pharmaceuticals SA

GC Biopharma Corp

Generium

Homology Medicines Inc

Immusoft Corp

JCR Pharmaceuticals Co Ltd

RegenxBio Inc

ReqMed Co Ltd

Sigilon Therapeutics Inc

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

AVRRD-05 – Drug Profile

Product Description

Mechanism Of Action

BMRD-001 – Drug Profile

Product Description

Mechanism Of Action

CAP-001 – Drug Profile

Product Description

Mechanism Of Action

DNL-310 – Drug Profile

Product Description

Mechanism Of Action

DUOC-01 – Drug Profile

Product Description

Mechanism Of Action

EGT-301 – Drug Profile

Product Description

Mechanism Of Action

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

Product Description

Mechanism Of Action

GNR-055 – Drug Profile

Product Description

Mechanism Of Action

HMI-203 – Drug Profile

Product Description

Mechanism Of Action

idursulfase beta – Drug Profile

Product Description

Mechanism Of Action

ISP-002 – Drug Profile

Product Description

Mechanism Of Action

Mucopolysaccharidosis Type II – Drug Profile

Product Description

Mechanism Of Action

pabinafusp alfa – Drug Profile

Product Description

Mechanism Of Action

pentosan polysulfate sodium – Drug Profile

Product Description

Mechanism Of Action

Recombinant Iduronate 2-Sulfatase Replacement for Mucopolysaccharidosis II – Drug Profile

Product Description

Mechanism Of Action

RGX-121 – Drug Profile

Product Description

Mechanism Of Action

SIG-018 – Drug Profile

Product Description

Mechanism Of Action

xB-3008 – Drug Profile

Product Description

Mechanism Of Action

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

Sep 15, 2022: UK MHRA approves Avrobio’s Phase I/II Hunter syndrome therapy trial

Jul 14, 2022: Avrobio receives orphan drug designation for Hunter syndrome gene therapy

Feb 15, 2022: JCR announces first patient dosed in phase 3 global clinical trial of JR-141 for treatment of MPS II (Hunter Syndrome)

Feb 10, 2022: Homology medicines announces presentations on HMI-203 investigational gene therapy for Hunter syndrome and broad applicability of AAVHSC platform for lysosomal storage disorders at the 18th Annual WORLDSymposium meeting

Feb 09, 2022: JCR pharmaceuticals to present posters on JR-141 at the 18th annual WORLDSymposium 2022

Feb 03, 2022: JCR Pharmaceuticals receives the WORLDSymposium new treatment award for IZCARGO (Pabinafusp Alfa)

Nov 29, 2021: Immusoft receives $4M in funding from the California Institute for Regenerative Medicine (CIRM)

Nov 08, 2021: AVROBIO to present preclinical data on AVRRD-05 at the 14th ICIEM Conference

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.

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

Table

List of Tables

Number of Products under Development for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ), 2022

Number of Products under Development by Companies, 2022

Number of Products under Development by Universities/Institutes, 2022

Products under Development by Companies, 2022

Products under Development by Companies, 2022 (Contd..1)

Products under Development by Universities/Institutes, 2022

Number of Products by Stage and Target, 2022

Number of Products by Stage and Mechanism of Action, 2022

Number of Products by Stage and Route of Administration, 2022

Number of Products by Stage and Molecule Type, 2022

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) – Pipeline by AngioChem Inc, 2022

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) – Pipeline by Apteeus SAS, 2022

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) – Pipeline by AVROBIO Inc, 2022

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) – Pipeline by Bioasis Technologies Inc, 2022

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) – Pipeline by Biorchestra Co Ltd, 2022

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) – Pipeline by Capsida Biotherapeutics Inc, 2022

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) – Pipeline by Denali Therapeutics Inc, 2022

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) – Pipeline by Esteve Pharmaceuticals SA, 2022

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) – Pipeline by GC Biopharma Corp, 2022

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) – Pipeline by Generium, 2022

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) – Pipeline by Homology Medicines Inc, 2022

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) – Pipeline by Immusoft Corp, 2022

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) – Pipeline by JCR Pharmaceuticals Co Ltd, 2022

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) – Pipeline by RegenxBio Inc, 2022

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) – Pipeline by ReqMed Co Ltd, 2022

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) – Pipeline by Sigilon Therapeutics Inc, 2022

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) – Dormant Projects, 2022

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) – Discontinued Products, 2022

Figures

List of Figures

Number of Products under Development for Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ), 2022

Number of Products under Development by Companies, 2022

Number of Products under Development by Universities/Institutes, 2022

Number of Products by Stage and Top 10 Targets, 2022

Number of Products by Top 10 Mechanism of Actions, 2022

Number of Products by Stage and Top 10 Mechanism of Actions, 2022

Number of Products by Top 10 Routes of Administration, 2022

Number of Products by Stage and Top 10 Routes of Administration, 2022

Number of Products by Top 10 Molecule Types, 2022

Number of Products by Stage and Top 10 Molecule Types, 2022

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