Usher Syndrome - Drugs in Development, 2021

November 2021 | 82 pages | ID: U7FE6AFADD82EN
Global Markets Direct

Abstracts

Usher Syndrome - Drugs in Development, 2021

SUMMARY

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Usher Syndrome - Drugs In Development, 2021, 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 - Drugs In Development, 2021, 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, Preclinical and Discovery stages are 1, 13 and 2 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 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.

Contents

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
Ionis Pharmaceuticals Inc
IVERIC bio Inc
Locanabio Inc
Nanoscope Therapeutics Inc
Odylia Therapeutics Inc
ProQR Therapeutics NV
Wave Life Sciences Ltd
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
EDIT-102 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
ELX-03 - 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 for Usher Syndrome and Retinitis Pigmentosa - 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 CLRN1 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
Gene Therapy to Activate MYO7A for Usher Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate USH1C for Usher Syndrome 1C - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate USH1G for Usher Type I Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate USH2A for Retinitis Pigmentosa and Usher Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate USH2A for Usher Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Oligonucleotides to Activate USH2A for Usher Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
QR-411 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
QRX-421a - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
QRX-461 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
VMCO-1 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Usher Syndrome - Dormant Projects
Usher Syndrome - Discontinued Products
Usher Syndrome - Product Development Milestones
Featured News & Press Releases
Sep 06, 2021: ProQR announces virtual presentation at EURETINA 2021
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
Mar 24, 2021: ProQR announces positive results from clinical trial of QR-421a in Usher Syndrome and plans to start pivotal trials
Feb 16, 2021: ProQR announces expert perspectives call on disease education and endpoints in usher syndrome
Jun 12, 2020: ProQR announces expert perspectives conference call series
Mar 31, 2020: ProQR announces positive findings from an interim analysis in the phase 1/2 trial of QR-421a for Usher Syndrome and provides business update
Mar 25, 2020: ProQR to present findings from phase 1/2 interim analysis of QR-421a for Usher Syndrome
Jan 31, 2020: FDA grants RPD designation to ProQR’s retinitis pigmentosa therapy
May 02, 2019: Eloxx Pharmaceuticals presents positive new data at the Association for Research in Vision and Ophthalmology “ARVO” 2019 Annual Meeting
Apr 22, 2019: ProQR announces presentation of abstracts on QR-421a at scientific conferences in April
Mar 11, 2019: ProQR doses first patient in Phase 1/2 stellar trial of QR-421a for Usher Syndrome Type
Jan 02, 2019: ProQR receives fast track designation from FDA for QR-421a for usher syndrome type
Dec 05, 2018: ProQR receives FDA approval for Phase I/II QR-421a trial
Jul 12, 2018: ProQR Announces Presentation on QR-421a in Ophthalmology at Conferences in July
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, 2021
Number of Products under Development by Companies, 2021
Number of Products under Development by Universities/Institutes, 2021
Products under Development by Companies, 2021
Products under Development by Universities/Institutes, 2021
Number of Products by Stage and Target, 2021
Number of Products by Stage and Mechanism of Action, 2021
Number of Products by Stage and Route of Administration, 2021
Number of Products by Stage and Molecule Type, 2021
Usher Syndrome - Pipeline by Akouos Inc, 2021
Usher Syndrome - Pipeline by Atsena Therapeutics Inc, 2021
Usher Syndrome - Pipeline by Clearside BioMedical Inc, 2021
Usher Syndrome - Pipeline by Editas Medicine Inc, 2021
Usher Syndrome - Pipeline by Eloxx Pharmaceuticals Inc, 2021
Usher Syndrome - Pipeline by Ionis Pharmaceuticals Inc, 2021
Usher Syndrome - Pipeline by IVERIC bio Inc, 2021
Usher Syndrome - Pipeline by Locanabio Inc, 2021
Usher Syndrome - Pipeline by Nanoscope Therapeutics Inc, 2021
Usher Syndrome - Pipeline by Odylia Therapeutics Inc, 2021
Usher Syndrome - Pipeline by ProQR Therapeutics NV, 2021
Usher Syndrome - Pipeline by Wave Life Sciences Ltd, 2021
Usher Syndrome - Dormant Projects, 2021
Usher Syndrome - Discontinued Products, 2021

List Of Figures

LIST OF FIGURES

Number of Products under Development for Usher Syndrome, 2021
Number of Products under Development by Companies, 2021
Number of Products under Development by Universities/Institutes, 2021
Number of Products by Targets, 2021
Number of Products by Stage and Targets, 2021
Number of Products by Mechanism of Actions, 2021
Number of Products by Stage and Mechanism of Actions, 2021
Number of Products by Routes of Administration, 2021
Number of Products by Stage and Routes of Administration, 2021
Number of Products by Molecule Types, 2021
Number of Products by Stage and Molecule Types, 2021