Usher Syndrome-Pipeline Review, H2 2016

Usher Syndrome-Pipeline Review, H2 2016


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  • Pages: 38
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Executive Summary

Usher Syndrome-Pipeline Review, H2 2016

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Usher Syndrome-Pipeline Review, H2 2016, provides an overview of the Usher Syndrome (Genetic Disorders) pipeline landscape.

Usher syndrome is an inherited condition characterized by hearing impairment and progressive vision loss. The major symptoms of Usher syndrome are hearing loss and an eye disorder called retinitis pigmentosa. Risk factors include retinal degeneration, prolonged, unprotected exposure to sunlight may accelerate vision loss. Treatment includes cochlear implants, hearing aids and Vitamin A supplementation.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Usher Syndrome-Pipeline Review, H2 2016, provides comprehensive information on the therapeutics under development for Usher 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 Usher Syndrome (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Usher 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 and Discovery stages are 2 and 2 respectively. Similarly, the Universities portfolio in Preclinical stages comprises 1 molecules, respectively.

Usher 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 Usher Syndrome (Genetic Disorders).

The pipeline guide reviews pipeline therapeutics for Usher 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 Usher Syndrome (Genetic Disorders) therapeutics and enlists all their major and minor projects.

The pipeline guide evaluates Usher 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 Usher 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 Usher 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 Usher 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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Table of Contents

Table of Contents 2

List of Tables 3

List of Figures 4

Introduction 5

Global Markets Direct Report Coverage 5

Usher Syndrome Overview 6

Therapeutics Development 7

Pipeline Products for Usher Syndrome-Overview 7

Pipeline Products for Usher Syndrome-Comparative Analysis 8

Usher Syndrome-Therapeutics under Development by Companies 9

Usher Syndrome-Therapeutics under Investigation by Universities/Institutes 10

Usher Syndrome-Pipeline Products Glance 11

Clinical Stage Products 11

Early Stage Products 12

Usher Syndrome-Products under Development by Companies 13

Usher Syndrome-Products under Investigation by Universities/Institutes 14

Usher Syndrome-Companies Involved in Therapeutics Development 15

Amgen Inc 15

Editas Medicine Inc 16

ProQR Therapeutics NV 17

Sanofi 18

Usher Syndrome-Therapeutics Assessment 19

Assessment by Monotherapy Products 19

Assessment by Target 20

Assessment by Mechanism of Action 22

Assessment by Route of Administration 24

Assessment by Molecule Type 26

Drug Profiles 28

Gene Therapy for Usher Syndrome-Drug Profile 28

Product Description 28

Mechanism Of Action 28

R&D Progress 28

Gene Therapy to Activate MYO7A for Usher Syndrome-Drug Profile 29

Product Description 29

Mechanism Of Action 29

R&D Progress 29

NT-501-Drug Profile 30

Product Description 30

Mechanism Of Action 30

R&D Progress 30

QRX-411-Drug Profile 33

Product Description 33

Mechanism Of Action 33

R&D Progress 33

SAR-421869-Drug Profile 34

Product Description 34

Mechanism Of Action 34

R&D Progress 34

Usher Syndrome-Dormant Projects 36

Appendix 37

Methodology 37

Coverage 37

Secondary Research 37

Primary Research 37

Expert Panel Validation 37

Contact Us 37

Disclaimer 38

List of Figures

Number of Products under Development for Usher Syndrome, H2 2016 7

Number of Products under Development for Usher Syndrome-Comparative Analysis, H2 2016 8

Number of Products under Development by Companies, H2 2016 9

Comparative Analysis by Early Stage Products, H2 2016 12

Assessment by Monotherapy Products, H2 2016 19

Number of Products by Targets, H2 2016 20

Number of Products by Stage and Targets, H2 2016 20

Number of Products by Mechanism of Actions, H2 2016 22

Number of Products by Stage and Mechanism of Actions, H2 2016 22

Number of Products by Routes of Administration, H2 2016 24

Number of Products by Stage and Routes of Administration, H2 2016 24

Number of Products by Molecule Types, H2 2016 26

Number of Products by Stage and Molecule Types, H2 2016 26

List of Tables

Number of Products under Development for Usher Syndrome, H2 2016 7

Number of Products under Development for Usher Syndrome-Comparative Analysis, H2 2016 8

Number of Products under Development by Companies, H2 2016 9

Number of Products under Investigation by Universities/Institutes, H2 2016 10

Comparative Analysis by Clinical Stage Development, H2 2016 11

Comparative Analysis by Early Stage Development, H2 2016 12

Products under Development by Companies, H2 2016 13

Products under Investigation by Universities/Institutes, H2 2016 14

Usher Syndrome-Pipeline by Amgen Inc, H2 2016 15

Usher Syndrome-Pipeline by Editas Medicine Inc, H2 2016 16

Usher Syndrome-Pipeline by ProQR Therapeutics NV, H2 2016 17

Usher Syndrome-Pipeline by Sanofi, H2 2016 18

Assessment by Monotherapy Products, H2 2016 19

Number of Products by Stage and Target, H2 2016 21

Number of Products by Stage and Mechanism of Action, H2 2016 23

Number of Products by Stage and Route of Administration, H2 2016 25

Number of Products by Stage and Molecule Type, H2 2016 27

Usher Syndrome-Dormant Projects, H2 2016 36

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Amgen Inc, Editas Medicine Inc, ProQR Therapeutics NV, Sanofi

Usher Syndrome Therapeutic Products under Development, Key Players in Usher Syndrome Therapeutics, Usher Syndrome Pipeline Overview, Usher Syndrome Pipeline, Usher Syndrome Pipeline Assessment

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