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The publisher's latest Pharmaceutical and Healthcare disease pipeline guide Usher Syndrome - Drugs In Development, 2022, provides an overview of the Usher Syndrome (Genetic Disorders) pipeline landscape.Speak directly to the analyst to clarify any post sales queries you may have.
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
The publisher's Pharmaceutical and Healthcare latest pipeline guide Usher Syndrome - Drugs In Development, 2022, 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 III, Preclinical and Discovery stages are 1, 14 and 4 respectively. Similarly, the Universities portfolio in Phase I and Preclinical stages comprises 1 and 3 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 the publisher'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.
Table of Contents
- Introduction
- 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
- 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
- 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
Companies Mentioned (Partial List)
A selection of companies mentioned in this report includes, but is not limited to:
- 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
