Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline Review, H1 2020

June 2020 | 110 pages | ID: M9F6E8FA3D6EN
Global Markets Direct

Abstracts

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline Review, H1 2020

SUMMARY

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline Review, H1 2020, provides an overview of the Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) (Genetic Disorders) pipeline landscape.
MPS I (Mucopolysaccharidosis I) is an inherited lysosomal storage disorder caused by a deficiency of alpha-L-iduronidase, a lysosomal enzyme normally required for the breakdown of certain complex carbohydrates known as glycosaminoglycans (GAGs). Symptoms include abnormal bones in the spine, claw hand, cloudy corneas, deafness and heart valve problems. Treatment includes bone marrow transplantation, enzyme therapy and gene therapy.

REPORT HIGHLIGHTS

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline Review, H1 2020, provides comprehensive information on the therapeutics under development for Mucopolysaccharidosis I (MPS I) (Hurler 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 I (MPS I) (Hurler Syndrome ) (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies/Universities/Institutes, the molecules developed by Companies in Phase II, Phase I, Preclinical, Discovery and Unknown stages are 5, 2, 10, 1 and 1 respectively. Similarly, the Universities portfolio in Discovery stages comprises 2 molecules, respectively.
Mucopolysaccharidosis I (MPS I) (Hurler 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 I (MPS I) (Hurler Syndrome ) (Genetic Disorders).
• The pipeline guide reviews pipeline therapeutics for Mucopolysaccharidosis I (MPS I) (Hurler 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 I (MPS I) (Hurler Syndrome ) (Genetic Disorders) therapeutics and enlists all their major and minor projects.
• The pipeline guide evaluates Mucopolysaccharidosis I (MPS I) (Hurler 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 I (MPS I) (Hurler 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 I (MPS I) (Hurler 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 I (MPS I) (Hurler 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.

Contents

Introduction
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Overview
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Therapeutics Development
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Therapeutics Assessment
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Companies Involved in Therapeutics Development
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Drug Profiles
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Dormant Projects
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Discontinued Products
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Product Development Milestones
Appendix

List Of Tables

LIST OF TABLES

Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ), H1 2020
Number of Products under Development by Companies, H1 2020
Number of Products under Development by Universities/Institutes, H1 2020
Products under Development by Companies, H1 2020
Products under Development by Companies, H1 2020 (Contd..1), H1 2020
Products under Development by Universities/Institutes, H1 2020
Number of Products by Stage and Target, H1 2020
Number of Products by Stage and Mechanism of Action, H1 2020
Number of Products by Stage and Route of Administration, H1 2020
Number of Products by Stage and Molecule Type, H1 2020
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by ArmaGen Inc, H1 2020
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by BioStrategies LC, H1 2020
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by bluebird bio Inc, H1 2020
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by EdiGene Inc, H1 2020
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Eloxx Pharmaceuticals Inc, H1 2020
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by GT Gain Therapeutics SA, H1 2020
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Immusoft Corp, H1 2020
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by JCR Pharmaceuticals Co Ltd, H1 2020
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Jupiter Orphan Therapeutics Inc, H1 2020
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Magenta Therapeutics Inc, H1 2020
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Orchard Therapeutics Plc, H1 2020
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Ossianix Inc, H1 2020
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Paradigm Biopharmaceuticals Ltd, H1 2020
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by RegenxBio Inc, H1 2020
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by ReqMed Co Ltd, H1 2020
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Sangamo Therapeutics Inc, H1 2020
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Sigilon Therapeutics Inc, H1 2020
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Tamid Bio Inc, H1 2020
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Tega Therapeutics Inc, H1 2020
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Dormant Projects, H1 2020
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Discontinued Products, H1 2020

List Of Figures

LIST OF FIGURES

Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ), H1 2020
Number of Products under Development by Companies, H1 2020
Number of Products by Top 10 Targets, H1 2020
Number of Products by Stage and Top 10 Targets, H1 2020
Number of Products by Top 10 Mechanism of Actions, H1 2020
Number of Products by Stage and Top 10 Mechanism of Actions, H1 2020
Number of Products by Top 10 Routes of Administration, H1 2020
Number of Products by Stage and Top 10 Routes of Administration, H1 2020
Number of Products by Top 10 Molecule Types, H1 2020
Number of Products by Stage and Top 10 Molecule Types, H1 2020

COMPANIES MENTIONED

ArmaGen Inc
BioStrategies LC
bluebird bio Inc
EdiGene Inc
Eloxx Pharmaceuticals Inc
GT Gain Therapeutics SA
Immusoft Corp
JCR Pharmaceuticals Co Ltd
Jupiter Orphan Therapeutics Inc
Magenta Therapeutics Inc
Orchard Therapeutics Plc
Ossianix Inc
Paradigm Biopharmaceuticals Ltd
RegenxBio Inc
ReqMed Co Ltd
Sangamo Therapeutics Inc
Sigilon Therapeutics Inc
Tamid Bio Inc
Tega Therapeutics Inc