Usher Syndrome – Pipeline Review, H2 2019

Global Markets Direct’s, ‘Usher Syndrome – Pipeline Review, H2 2019’, provides an overview of the Usher Syndrome pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Usher 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 Usher Syndrome and features dormant and discontinued projects.

Global Markets Direct’s report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report 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. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report 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.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data.

Scope

– The report provides a snapshot of the global therapeutic landscape of Usher Syndrome

– The report reviews pipeline therapeutics for Usher 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 Usher Syndrome therapeutics and enlists all their major and minor projects

– The report assesses Usher 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 Usher 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 Usher 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 Usher 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

Companies mentioned

Amgen Inc

Clearside BioMedical Inc

Editas Medicine Inc

Eloxx Pharmaceuticals Inc

Ionis Pharmaceuticals Inc

Odylia Therapeutics Inc

ProQR Therapeutics NV

Wave Life Sciences Ltd

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Usher Syndrome Overview

Usher Syndrome Therapeutics Development

Pipeline Overview

Pipeline by Companies

Pipeline by Universities/Institutes

Products under Development by Companies

Products under Development by Universities/Institutes

Usher Syndrome Therapeutics Assessment

Assessment by Target

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Usher Syndrome Companies Involved in Therapeutics Development

Amgen Inc

Editas Medicine Inc

Ionis Pharmaceuticals Inc

ProQR Therapeutics NV

Sanofi

Usher Syndrome Drug Profiles

ASO-29 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

BF-844 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy for Usher Syndrome Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy to Activate CLRN1 for Usher Syndrome Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy to Activate MYO7A for Usher Syndrome Drug Profile

Product Description

Mechanism Of Action

R&D Progress

NT-501 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

QRX-411 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

QRX-421 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

SAR-421869 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Usher Syndrome Dormant Projects

Usher Syndrome Product Development Milestones

Featured News & Press Releases

Sep 05, 2017: ProQR’s Drug Candidate QRX-421 for Usher Syndrome Receives Orphan Drug Designation from FDA and EMA

Jul 05, 2017: ProQR's Drug Candidate QRX-411 for Usher Syndrome Receives Orphan Drug Designation from FDA and EMA

May 01, 2017: ProQR to Present Data on QRX-411 at ARVO

May 01, 2017: ProQR to Present Data on QRX-421 at ARVO

Sep 18, 2012: Neurotech Pharmaceuticals Provides Update On NT-501

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

List of Tables

List of Tables

Number of Products under Development for Usher Syndrome, H2 2019

Number of Products under Development by Companies, H2 2019

Number of Products under Development by Universities/Institutes, H2 2019

Products under Development by Companies, H2 2019

Products under Development by Universities/Institutes, H2 2019

Number of Products by Stage and Target, H2 2019

Number of Products by Stage and Mechanism of Action, H2 2019

Number of Products by Stage and Route of Administration, H2 2019

Number of Products by Stage and Molecule Type, H2 2019

Usher Syndrome – Pipeline by Amgen Inc, H2 2019

Usher Syndrome – Pipeline by Clearside BioMedical Inc, H2 2019

Usher Syndrome – Pipeline by Editas Medicine Inc, H2 2019

Usher Syndrome – Pipeline by Eloxx Pharmaceuticals Inc, H2 2019

Usher Syndrome – Pipeline by Ionis Pharmaceuticals Inc, H2 2019

Usher Syndrome – Pipeline by Odylia Therapeutics Inc, H2 2019

Usher Syndrome – Pipeline by ProQR Therapeutics NV, H2 2019

Usher Syndrome – Pipeline by Wave Life Sciences Ltd, H2 2019

Usher Syndrome – Dormant Projects, H2 2019

Usher Syndrome – Discontinued Products, H2 2019

List of Figures

List of Figures

Number of Products under Development for Usher Syndrome, H2 2019

Number of Products under Development by Companies, H2 2019

Number of Products under Development by Universities/Institutes, H2 2019

Number of Products by Targets, H2 2019

Number of Products by Stage and Targets, H2 2019

Number of Products by Mechanism of Actions, H2 2019

Number of Products by Stage and Mechanism of Actions, H2 2019

Number of Products by Routes of Administration, H2 2019

Number of Products by Stage and Routes of Administration, H2 2019

Number of Products by Molecule Types, H2 2019

Number of Products by Stage and Molecule Types, H2 2019

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