Usher Syndrome Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

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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 and prolonged and unprotected exposure to sunlight may accelerate vision loss. Treatment includes cochlear implants, hearing aids, and Vitamin A supplementation.

The Usher syndrome pipeline market research 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 the 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.

Usher Syndrome Pipeline Products Market Segmentation by Targets

The key targets in the Usher syndrome pipeline products market are Usherin, Clarin 1, Harmonin, Unconventional Myosin VIIa, and Usher syndrome Type 1G Protein. Usherin is the leading target in the pipeline.

Usher Syndrome Pipeline Products Market Analysis by Targets

Usher Syndrome Pipeline Products Market Analysis by Targets

For more target insights into the Usher syndrome pipeline products market, download a free report sample

Usher Syndrome Pipeline Products Market Segmentation by Mechanisms of Action

The key mechanisms of action in the Usher syndrome pipeline products market are Usherin Activator, Clarin 1 Activator, Harmonin Activator, Unconventional Myosin VIIa Activator, and Usher syndrome Type 1G Protein Activator. Usherin Activator is the leading MoA in the pipeline market.

Usher Syndrome Pipeline Products Market Analysis by Mechanisms of Action

Usher Syndrome Pipeline Products Market Analysis by Mechanisms of Action

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

Usher Syndrome Pipeline Products Market Segmentation by Routes of Administration

The key routes of administration in the Usher syndrome pipeline products market are intravitreal, intraocular, and parenteral. Intravitreal is the most preferred route for administration.

Usher Syndrome Pipeline Products Market Analysis by Routes of Administration

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

Usher Syndrome Pipeline Products Market Segmentation by Molecule Types

The key molecule types in the Usher syndrome pipeline products market are gene therapy, antisense oligonucleotide, small molecule, and oligonucleotide. The leading molecule is gene therapy.

Usher Syndrome Pipeline Products Market Analysis by Molecule Types

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

Usher Syndrome Pipeline Products Market - Competitive Landscape

Some of the leading companies in the Usher syndrome pipeline products market are Odylia Therapeutics Inc, ProQR Therapeutics NV, GeneToBe LLC, Akouos Inc, Atsena Therapeutics Inc, Clearside BioMedical Inc, Editas Medicine Inc, Eloxx Pharmaceuticals Inc, and Ionis Pharmaceuticals Inc among others. Odylia Therapeutics Inc is the leading company in the Usher syndrome pipeline products market.

Usher Syndrome Pipeline Products Market Analysis by Companies

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

Usher Syndrome Pipeline Products Market Report Overview

Key Targets Usherin, Clarin 1, Harmonin, Unconventional Myosin VIIa, and Usher syndrome Type 1G Protein
Key Mechanisms of Action Usherin Activator, Clarin 1 Activator, Harmonin Activator, Unconventional Myosin VIIa Activator, and Usher syndrome Type 1G Protein Activator
Key Routes of Administration Intravitreal, Intraocular, And Parenteral
Key Molecule Type Gene Therapy, Antisense Oligonucleotide, Small Molecule, and Oligonucleotide
Leading Companies Odylia Therapeutics Inc, ProQR Therapeutics NV, GeneToBe LLC, Akouos Inc, Atsena Therapeutics Inc, Clearside BioMedical Inc, Editas Medicine Inc, Eloxx Pharmaceuticals Inc, and Ionis Pharmaceuticals Inc

Scope

  • The pipeline guide provides a snapshot of the global therapeutic landscape of Usher syndrome.
  • The pipeline guide reviews pipeline therapeutics for Usher syndrome 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 to 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 therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Usher syndrome 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 the latest news related to pipeline therapeutics for Usher syndrome

Reasons to Buy

  • Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
  • Recognize emerging players with potentially strong product portfolios and create effective counter-strategies to gain competitive advantage.
  • Find and recognize significant and varied types of therapeutics under development for Usher syndrome.
  • 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 their most promising pipeline therapeutics.
  • Formulate corrective measures for pipeline projects by understanding the 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.
  • Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from the pipeline.

Akouos Inc
Atsena Therapeutics Inc
Clearside BioMedical Inc
Editas Medicine Inc
Eloxx Pharmaceuticals Inc
GeneToBe LLC
Ionis Pharmaceuticals Inc
IVERIC bio Inc
Locanabio Inc
Nanoscope Therapeutics Inc
Odylia Therapeutics Inc
ProQR Therapeutics NV
Saliogen Therapeutics Inc
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

Akouos Inc

Atsena Therapeutics Inc

Clearside BioMedical Inc

Editas Medicine Inc

Eloxx Pharmaceuticals Inc

GeneToBe LLC

Ionis Pharmaceuticals Inc

IVERIC bio Inc

Locanabio Inc

Nanoscope Therapeutics Inc

Odylia Therapeutics Inc

ProQR Therapeutics NV

Saliogen Therapeutics Inc

Wave Life Sciences Ltd

Usher Syndrome – Drug Profiles

AK-CLRN1 – Drug Profile

Product Description

Mechanism Of Action

ASO-29 – Drug Profile

Product Description

Mechanism Of Action

ATSN-301 – Drug Profile

Product Description

Mechanism Of Action

BF-844 – Drug Profile

Product Description

Mechanism Of Action

EDIT-102 – Drug Profile

Product Description

Mechanism Of Action

ELX-03 – Drug Profile

Product Description

Mechanism Of Action

Gene Therapy for Usher Syndrome – Drug Profile

Product Description

Mechanism Of Action

Gene Therapy for Usher Syndrome – Drug Profile

Product Description

Mechanism Of Action

Gene Therapy for Usher Syndrome and Retinitis Pigmentosa – Drug Profile

Product Description

Mechanism Of Action

Gene Therapy to Activate CLRN1 for Usher Syndrome – Drug Profile

Product Description

Mechanism Of Action

Gene Therapy to Activate CLRN1 for Usher Syndrome – Drug Profile

Product Description

Mechanism Of Action

Gene Therapy to Activate MYO7A for Usher Syndrome – Drug Profile

Product Description

Mechanism Of Action

Gene Therapy to Activate USH1C for Usher Syndrome 1C – Drug Profile

Product Description

Mechanism Of Action

Gene Therapy to Activate USH2A for Usher Syndrome – Drug Profile

Product Description

Mechanism Of Action

GTB-3001 – Drug Profile

Product Description

Mechanism Of Action

GTB-4001 – Drug Profile

Product Description

Mechanism Of Action

miniUSH2A: USH2A -RELATED IRDs – Drug Profile

Product Description

Mechanism Of Action

Oligonucleotides to Activate USH2A for Usher Syndrome – Drug Profile

Product Description

Mechanism Of Action

QR-411 – Drug Profile

Product Description

Mechanism Of Action

QRX-421a – Drug Profile

Product Description

Mechanism Of Action

QRX-461 – Drug Profile

Product Description

Mechanism Of Action

USHER-GT – Drug Profile

Product Description

Mechanism Of Action

VMCO-1 – Drug Profile

Product Description

Mechanism Of Action

Usher Syndrome – Dormant Projects

Usher Syndrome – Discontinued Products

Usher Syndrome – Product Development Milestones

Featured News & Press Releases

May 02, 2022: Atsena Therapeutics announces presentations at ARVO 2022 Annual Meeting and ASGCT 25th Annual Meeting

May 04, 2021: Editas Medicine to present preclinical data demonstrating progress in development of gene editing medicines for the treatment of genetic ocular diseases at the American Society Of Gene And Cell Therapy annual meeting

May 01, 2021: Editas Medicine to present preclinical data demonstrating advancements in in vivo gene editing approach for the treatment of genetic ocular diseases at the Association for Research in Vision and Ophthalmology Annual Meeting

May 02, 2019: Eloxx Pharmaceuticals presents positive new data at the Association for Research in Vision and Ophthalmology “ARVO” 2019 Annual Meeting

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

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

Table

List of Tables

Number of Products under Development for Usher 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 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

Usher Syndrome – Pipeline by Akouos Inc, 2022

Usher Syndrome – Pipeline by Atsena Therapeutics Inc, 2022

Usher Syndrome – Pipeline by Clearside BioMedical Inc, 2022

Usher Syndrome – Pipeline by Editas Medicine Inc, 2022

Usher Syndrome – Pipeline by Eloxx Pharmaceuticals Inc, 2022

Usher Syndrome – Pipeline by GeneToBe LLC, 2022

Usher Syndrome – Pipeline by Ionis Pharmaceuticals Inc, 2022

Usher Syndrome – Pipeline by IVERIC bio Inc, 2022

Usher Syndrome – Pipeline by Locanabio Inc, 2022

Usher Syndrome – Pipeline by Nanoscope Therapeutics Inc, 2022

Usher Syndrome – Pipeline by Odylia Therapeutics Inc, 2022

Usher Syndrome – Pipeline by ProQR Therapeutics NV, 2022

Usher Syndrome – Pipeline by Saliogen Therapeutics Inc, 2022

Usher Syndrome – Pipeline by Wave Life Sciences Ltd, 2022

Usher Syndrome – Dormant Projects, 2022

Usher Syndrome – Discontinued Products, 2022

Figures

List of Figures

Number of Products under Development for Usher Syndrome, 2022

Number of Products under Development by Companies, 2022

Number of Products under Development by Universities/Institutes, 2022

Number of Products by Top 10 Targets, 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

Frequently asked questions

Usher Syndrome Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update standard reports
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