Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2021 Update

Pages: 115 Published: November 30, 2021 Report Code: GMDGDHC13175IDB

  • Overview
  • Key Players
  • Contents
  • Tables
  • Figures
  • FAQ
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The Mucopolysaccharidosis III pipeline market research report provides comprehensive information on the therapeutics under development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome), 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 provides an overview of key players involved in therapeutic development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) and features dormant and discontinued projects.

What are the key targets in the Mucopolysaccharidosis III pipeline market?

The key targets in the Mucopolysaccharidosis III pipeline market are Alpha N-Acetylglucosaminidase, N Sulphoglucosamine Sulphohydrolase, Polyadenylate Binding Protein 2, Heparan Alpha Glucosaminide N-Acetyltransferase, and Interleukin 1 Receptor Type 1, and N-Acetylgalactosamine 6 Sulfatase. Alpha N-Acetylglucosaminidase has the highest number of pipeline products in the Mucopolysaccharidosis III pipeline market.

Mucopolysaccharidosis III pipeline market, by targets

Mucopolysaccharidosis III pipeline market, by targets

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What are the key MoA in the Mucopolysaccharidosis III pipeline market?

The key MoA in the Mucopolysaccharidosis III pipeline market are Alpha N Acetylglucosaminidase Activator, Alpha N Acetylglucosaminidase Replacement, N Sulphoglucosamine Sulphohydrolase Activator, N Sulphoglucosamine Sulphohydrolase Replacement, Polyadenylate Binding Protein 2 (Poly(A) Binding Protein 2 or Nuclear PolyInhibitor, Heparan Alpha Glucosaminide N-Acetyltransferase Activator, Interleukin 1 Receptor Type 1 Antagonist 1, and N-Acetylgalactosamine 6 Sulfatase Replacement. Alpha N Acetylglucosaminidase Activator has the highest number of pipeline products in the Mucopolysaccharidosis III pipeline market.

Mucopolysaccharidosis III pipeline market, by MoA

Mucopolysaccharidosis III pipeline market, by MoA

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What are the key RoA in the Mucopolysaccharidosis III pipeline market?

The key RoA in the Mucopolysaccharidosis III pipeline market are Intravenous, Intrathecal, Intracerebral, Intravenous Bolus, Intravenous Drip, Nasal, Oral, Parenteral, Periarticular, and Subcutaneous. Intravenous has the highest number of pipeline products in the Mucopolysaccharidosis III pipeline market.

Mucopolysaccharidosis III pipeline market, by RoA

Mucopolysaccharidosis III pipeline market, by RoA

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What are the key molecule type in the Mucopolysaccharidosis III pipeline market?

The key molecule type in the Mucopolysaccharidosis III pipeline market are Gene Therapy, Recombinant Enzyme, Fusion Protein, Gene-Modified Cell Therapy, Small Molecule, Enzyme, Protein, and Recombinant Protein. Gene Therapy has the highest number of pipeline products in the Mucopolysaccharidosis III pipeline market.

Mucopolysaccharidosis III pipeline market, by molecule type

Mucopolysaccharidosis III pipeline market, by molecule type

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What are the key companies in the Mucopolysaccharidosis III pipeline market?

The key companies in the Mucopolysaccharidosis III pipeline market are Phoenix Nest Inc, Abeona Therapeutics Inc, Allievex Corp, Amicus Therapeutics Inc, ArmaGen Inc, Denali Therapeutics Inc, Esteve Pharmaceuticals SA, JCR Pharmaceuticals Co Ltd, M6P Therapeutics, and Orchard Therapeutics Plc.

Mucopolysaccharidosis III pipeline market, by company

Mucopolysaccharidosis III pipeline market, by company

To know more about key company , download a free report sample

Market report overview

Key Targets Alpha N-Acetylglucosaminidase, N Sulphoglucosamine Sulphohydrolase, Polyadenylate Binding Protein 2, Heparan Alpha Glucosaminide N-Acetyltransferase, and Interleukin 1 Receptor Type 1, and N-Acetylgalactosamine 6 Sulfatase
Key MoA Alpha N Acetylglucosaminidase Activator, Alpha N Acetylglucosaminidase Replacement, N Sulphoglucosamine Sulphohydrolase Activator, N Sulphoglucosamine Sulphohydrolase Replacement, Polyadenylate Binding Protein 2 (Poly(A) Binding Protein 2 or Nuclear PolyInhibitor, Heparan Alpha Glucosaminide N-Acetyltransferase Activator, Interleukin 1 Receptor Type 1 Antagonist 1, and N-Acetylgalactosamine 6 Sulfatase Replacement
Key RoA Intravenous, Intrathecal, Intracerebral, Intravenous Bolus, Intravenous Drip, Nasal, Oral, Parenteral, Periarticular, and Subcutaneous
Key Molecule Type Gene Therapy, Recombinant Enzyme, Fusion Protein, Gene-Modified Cell Therapy, Small Molecule, Enzyme, Protein, and Recombinant Protein
Key companies Phoenix Nest Inc, Abeona Therapeutics Inc, Allievex Corp, Amicus Therapeutics Inc, ArmaGen Inc, Denali Therapeutics Inc, Esteve Pharmaceuticals SA, JCR Pharmaceuticals Co Ltd, M6P Therapeutics, and Orchard Therapeutics Plc

Scope

  • The report provides a snapshot of the global therapeutic landscape of Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)
  • The report reviews pipeline therapeutics for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) by companies and universities/research institutes based on information derived from company and industry-specific sources
  • The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages
  • The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities
  • The report reviews key players involved Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) therapeutics and enlists all their major and minor projects
  • The report assesses Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) therapeutics based on drug target, mechanism of action (MoA), route of administration (RoA) and molecule type
  • The report summarizes all the dormant and discontinued pipeline projects
  • The report reviews latest news related to pipeline therapeutics for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)

Reasons to Buy

  • Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies
  • Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
  • Identify and understand important and diverse types of therapeutics under development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)
  • Identify potential new clients or partners in the target demographic
  • Develop strategic initiatives by understanding the focus areas of leading companies
  • Plan mergers and acquisitions effectively by identifying key players and it’s most promising pipeline therapeutics
  • Devise corrective measures for pipeline projects by understanding Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 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
  • Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline

Key Players

Abeona Therapeutics Inc

Allievex Corp

Amicus Therapeutics Inc

ArmaGen Inc

Denali Therapeutics Inc

Esteve Pharmaceuticals SA

JCR Pharmaceuticals Co Ltd

Jupiter Neurosciences Inc

Lacerta Therapeutics Inc

Lysogene SAS

M6P Therapeutics

Neurogt Inc

Orchard Therapeutics Plc

Phoenix Nest Inc

Seelos Therapeutics, Inc.

Swedish Orphan Biovitrum AB

Tega Therapeutics Inc

Table of Contents

Table of Contents

Introduction

Global Markets Direct Report Coverage

Mucopolysaccharidosis III – Overview

Mucopolysaccharidosis III – Therapeutics Development

Mucopolysaccharidosis III – Therapeutics Assessment

Mucopolysaccharidosis III – Companies Involved in Therapeutics Development

Mucopolysaccharidosis III – Drug Profiles

Mucopolysaccharidosis III – Dormant Projects

Mucopolysaccharidosis III – Discontinued Products

Mucopolysaccharidosis III – Product Development Milestones

Appendix

List of Tables

List of Tables

Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo 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 III (MPS III) (Sanfilippo Syndrome ) – Pipeline by Abeona Therapeutics Inc, 2021

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) – Pipeline by Allievex Corp, 2021

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) – Pipeline by Amicus Therapeutics Inc, 2021

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) – Pipeline by ArmaGen Inc, 2021

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) – Pipeline by Denali Therapeutics Inc, 2021

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) – Pipeline by Esteve Pharmaceuticals SA, 2021

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) – Pipeline by JCR Pharmaceuticals Co Ltd, 2021

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) – Pipeline by Jupiter Neurosciences Inc, 2021

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) – Pipeline by Lacerta Therapeutics Inc, 2021

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) – Pipeline by Lysogene SAS, 2021

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) – Pipeline by M6P Therapeutics, 2021

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) – Pipeline by Neurogt Inc, 2021

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) – Pipeline by Orchard Therapeutics Plc, 2021

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) – Pipeline by Phoenix Nest Inc, 2021

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) – Pipeline by Seelos Therapeutics, Inc., 2021

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) – Pipeline by Swedish Orphan Biovitrum AB, 2021

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) – Pipeline by Tega Therapeutics Inc, 2021

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) – Dormant Projects, 2021

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) – Discontinued Products, 2021

List of Figures

List of Figures

Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ), 2021

Number of Products under Development by Companies, 2021

Number of Products by Targets, 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 Top 10 Routes of Administration, 2021

Number of Products by Stage and Top 10 Routes of Administration, 2021

Number of Products by Top 10 Molecule Types, 2021

Number of Products by Stage and Top 10 Molecule Types, 2021

Frequently Asked Questions

The key targets in the Mucopolysaccharidosis III pipeline market are Alpha N-Acetylglucosaminidase, N Sulphoglucosamine Sulphohydrolase, Polyadenylate Binding Protein 2, Heparan Alpha Glucosaminide N-Acetyltransferase, and Interleukin 1 Receptor Type 1, and N-Acetylgalactosamine 6 Sulfatase. Alpha N-Acetylglucosaminidase has the highest number of pipeline products in the Mucopolysaccharidosis III pipeline market.

The key MoA in the Mucopolysaccharidosis III pipeline market are Alpha N Acetylglucosaminidase Activator, Alpha N Acetylglucosaminidase Replacement, N Sulphoglucosamine Sulphohydrolase Activator, N Sulphoglucosamine Sulphohydrolase Replacement, Polyadenylate Binding Protein 2 (Poly(A) Binding Protein 2 or Nuclear PolyInhibitor, Heparan Alpha Glucosaminide N-Acetyltransferase Activator, Interleukin 1 Receptor Type 1 Antagonist 1, and N-Acetylgalactosamine 6 Sulfatase Replacement. Alpha N Acetylglucosaminidase Activator has the highest number of pipeline products in the Mucopolysaccharidosis III pipeline market.

The key RoA in the Mucopolysaccharidosis III pipeline market are Intravenous, Intrathecal, Intracerebral, Intravenous Bolus, Intravenous Drip, Nasal, Oral, Parenteral, Periarticular, and Subcutaneous. Intravenous has the highest number of pipeline products in the Mucopolysaccharidosis III pipeline market.

The key molecule type in the Mucopolysaccharidosis III pipeline market are Gene Therapy, Recombinant Enzyme, Fusion Protein, Gene-Modified Cell Therapy, Small Molecule, Enzyme, Protein, and Recombinant Protein. Gene Therapy has the highest number of pipeline products in the Mucopolysaccharidosis III pipeline market.

The key companies in the Mucopolysaccharidosis III pipeline market are Phoenix Nest Inc, Abeona Therapeutics Inc, Allievex Corp, Amicus Therapeutics Inc, ArmaGen Inc, Denali Therapeutics Inc, Esteve Pharmaceuticals SA, JCR Pharmaceuticals Co Ltd, M6P Therapeutics, and Orchard Therapeutics Plc.

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