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Juvenile Macular Degeneration (Stargardt Disease) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

Industry Report I 2022-04-29 I 96 Pages I Global Markets Direct

Juvenile Macular Degeneration (Stargardt Disease) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Juvenile Macular Degeneration - Drugs In Development, 2022, provides an overview of the Juvenile Macular Degeneration (Ophthalmology) pipeline landscape.

Juvenile macular degeneration is a series of inherited eye disorders that affects children and young adults. The most common form of juvenile macular degeneration is Stargardt disease. Stargardt's disease is an inherited autosomal recessive syndrome. Signs and symptoms include blurry or fuzzy vision, dark, empty spots in the center of vision and difficulty reading or performing detail work. Risk factors include arteriosclerosis, hypercholesterolemia, smoking and hypertension.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Juvenile Macular Degeneration - Drugs In Development, 2022, provides comprehensive information on the therapeutics under development for Juvenile Macular Degeneration (Ophthalmology), 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 Juvenile Macular Degeneration (Ophthalmology) pipeline guide also reviews of key players involved in therapeutic development for Juvenile Macular Degeneration (Stargardt Disease) and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, Phase II, Phase I, Preclinical and Discovery stages are 2, 7, 1, 14 and 12 respectively. Similarly, the Universities portfolio in Preclinical stages comprises 2 molecules, respectively.

Juvenile Macular Degeneration (Ophthalmology) 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 Directs 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 Juvenile Macular Degeneration (Ophthalmology).
- The pipeline guide reviews pipeline therapeutics for Juvenile Macular Degeneration (Ophthalmology) 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 Juvenile Macular Degeneration (Ophthalmology) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Juvenile Macular Degeneration (Ophthalmology) 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 Juvenile Macular Degeneration (Ophthalmology)

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 Juvenile Macular Degeneration (Ophthalmology).
- 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 its most promising pipeline therapeutics.
- Formulate corrective measures for pipeline projects by understanding Juvenile Macular Degeneration (Ophthalmology) 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
List of Tables
List of Figures
Introduction
Global Markets Direct Report Coverage
Juvenile Macular Degeneration (Stargardt Disease) - Overview
Juvenile Macular Degeneration (Stargardt Disease) - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Juvenile Macular Degeneration (Stargardt Disease) - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Juvenile Macular Degeneration (Stargardt Disease) - Companies Involved in Therapeutics Development
4P-Pharma SAS
Alkeus Pharmaceuticals Inc
Alnylam Pharmaceuticals Inc
Alpine Biotherapeutics Corp
Applied Genetic Technologies Corp
Astellas Pharma Inc
Beam Therapeutics Inc
Belite Bio Inc
Biophytis SA
Brooklyn ImmunoTherapeutics Inc
Cardax Inc
Coave Therapeutics
Copernicus Therapeutics Inc
Curative Biotechnology Inc
Fera Pharmaceuticals LLC
Generation Bio Co
Grupo Ferrer Internacional SA
IVERIC bio Inc
jCyte Inc
Katairo GmbH
Kubota Vision Inc
Lysoclear Inc
Mediphage Bioceuticals Inc
Nanoscope Therapeutics Inc
Oak bay Biosciences Ltd
ProQR Therapeutics NV
Ray Therapeutics Inc
ReVision Therapeutics Inc
Saliogen Therapeutics Inc
Shape Therapeutics Inc
Spark Therapeutics Inc
Splice Bio SL
Stargazer Pharmaceuticals Inc
Juvenile Macular Degeneration (Stargardt Disease) - Drug Profiles
4P-020 - Drug Profile
Product Description
Mechanism Of Action
ALK-001 - Drug Profile
Product Description
Mechanism Of Action
History of Events
Aptamer to Inhibit CFD for Juvenile Macular Degeneration (Stargardt Disease) - Drug Profile
Product Description
Mechanism Of Action
ASP-7317 - Drug Profile
Product Description
Mechanism Of Action
History of Events
avacincaptad pegol sodium - Drug Profile
Product Description
Mechanism Of Action
History of Events
BIO-201 - Drug Profile
Product Description
Mechanism Of Action
History of Events
CDX-301 - Drug Profile
Product Description
Mechanism Of Action
CTxABCA-4 - Drug Profile
Product Description
Mechanism Of Action
ecothiopate iodide - Drug Profile
Product Description
Mechanism Of Action
History of Events
emixustat hydrochloride - Drug Profile
Product Description
Mechanism Of Action
History of Events
FAB-111 - Drug Profile
Product Description
Mechanism Of Action
Gene Therapy 1 to Activate ABCA4 for Stargardt Disease - Drug Profile
Product Description
Mechanism Of Action
History of Events
Gene therapy for Juvenile Macular Degeneration (Stargardt Disease) - Drug Profile
Product Description
Mechanism Of Action
Gene Therapy to Activate ABCA4 for Stargardt Disease - Drug Profile
Product Description
Mechanism Of Action
History of Events
Gene Therapy to Activate ABCA4 for Stargardt Disease - Drug Profile
Product Description
Mechanism Of Action
History of Events
Gene Therapy to Activate ABCA4 for Stargardt Disease - Drug Profile
Product Description
Mechanism Of Action
History of Events
Gene Therapy to Activate ABCA4 for Stargardt Disease - Drug Profile
Product Description
Mechanism Of Action
Gene Therapy To Activate ABCA4 for Stargardt Disease - Drug Profile
Product Description
Mechanism Of Action
Gene Therapy to Activate ABCA4 for Stargardt Disease - Drug Profile
Product Description
Mechanism Of Action
GTSG - Drug Profile
Product Description
Mechanism Of Action
Jcell - Drug Profile
Product Description
Mechanism Of Action
History of Events
Lead program in Stargardt disease - Drug Profile
Product Description
Mechanism Of Action
metformin - Drug Profile
Product Description
Mechanism Of Action
History of Events
miniABCA4: STGD1 - Drug Profile
Product Description
Mechanism Of Action
History of Events
QR-1011 - Drug Profile
Product Description
Mechanism Of Action
RAY-001 - Drug Profile
Product Description
Mechanism Of Action
Recombinant Enzyme for Age Related Macular Degeneration and Stargardt Disease - Drug Profile
Product Description
Mechanism Of Action
History of Events
REV-0100 - Drug Profile
Product Description
Mechanism Of Action
History of Events
SHP-261 - Drug Profile
Product Description
Mechanism Of Action
soraprazan - Drug Profile
Product Description
Mechanism Of Action
History of Events
Stargardt - Drug Profile
Product Description
Mechanism Of Action
Stargardt Disease - Drug Profile
Product Description
Mechanism Of Action
History of Events
Stem Cell Therapy for Ophthalmology - Drug Profile
Product Description
Mechanism Of Action
History of Events
STG-001 - Drug Profile
Product Description
Mechanism Of Action
History of Events
TFB for Dry Age-Related Macular Degeneration, Stargardt Macular Dystrophy - Drug Profile
Product Description
Mechanism Of Action
tinlarebant - Drug Profile
Product Description
Mechanism Of Action
History of Events
VMCO-1 - Drug Profile
Product Description
Mechanism Of Action
History of Events
vutrisiran - Drug Profile
Product Description
Mechanism Of Action
History of Events
Juvenile Macular Degeneration (Stargardt Disease) - Dormant Projects
Juvenile Macular Degeneration (Stargardt Disease) - Product Development Milestones
Featured News & Press Releases
Apr 12, 2022: Fera Pharmaceuticals announces FDA Orphan Designation of Phospholine Iodide for the treatment of Stargardt disease
Jan 25, 2022: Nanoscope Therapeutics announces FDA clearance of IND for MCO-010 gene therapy in Stargardt macular degeneration patients
Jan 10, 2022: Nanoscope awarded key U.S. patent protecting its multi-characteristic Opsin Gene Therapy platform for reversing blindness
Aug 25, 2021: Aequus strengthens clinical experience in Stargardt disease with pediatric ophthalmology expertise
Jul 14, 2021: FDA grants Alkeus Pharmaceuticals Breakthrough Therapy designation for ALK-001 (C20-D3-vitamin A) for the treatment of Stargardt Disease
Jun 30, 2021: Belite Bio announces start of LBS-008 phase 3 Stargardt Disease trial
Mar 03, 2021: Nanoscope president to deliver talk on gene therapy for retinal diseases at Association for Ocular Pharmacology and Therapeutics conference
Jan 25, 2021: Nanoscope Therapeutics receives Orphan Drug Designation for gene therapy of blindness
Nov 24, 2020: Kubota Vision announces publication of Emixustats pharmacodynamic effects in patients with stargardt disease
Oct 28, 2020: reVision Therapeutics announces US FDA Grant of rare pediatric disease and orphan-drug designation for REV-0100 for the treatment of Stargardt disease
Aug 20, 2020: Kubota Vision receives orphan products clinical trials grants to Emixustat for stargardt disease
Jul 20, 2020: Belite Bio announces positive results from phase 1 clinical trials of LBS-008
May 01, 2020: Kubota Vision completes enrollment in the Emixustat phase 3 clinical trial in patients with Stargardt disease
Feb 13, 2020: Acucela provides update on Emixustat phase 3 clinical trial in patients with Stargardt disease
Jan 06, 2020: Acucela announces publication assessing the role of Emixustat Hydrochloride in retinal degeneration treatment
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer

List of Tables
Number of Products under Development for Juvenile Macular Degeneration (Stargardt Disease), 2022
Number of Products under Development by Companies, 2022
Number of Products under Development by Companies, 2022 (Contd..1)
Number of Products under Development by Universities/Institutes, 2022
Products under Development by Companies, 2022
Products under Development by Companies, 2022 (Contd..1)
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
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by 4P-Pharma SAS, 2022
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Alkeus Pharmaceuticals Inc, 2022
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Alnylam Pharmaceuticals Inc, 2022
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Alpine Biotherapeutics Corp, 2022
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Applied Genetic Technologies Corp, 2022
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Astellas Pharma Inc, 2022
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Beam Therapeutics Inc, 2022
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Belite Bio Inc, 2022
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Biophytis SA, 2022
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Brooklyn ImmunoTherapeutics Inc, 2022
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Cardax Inc, 2022
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Coave Therapeutics, 2022
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Copernicus Therapeutics Inc, 2022
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Curative Biotechnology Inc, 2022
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Fera Pharmaceuticals LLC, 2022
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Generation Bio Co, 2022
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Grupo Ferrer Internacional SA, 2022
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by IVERIC bio Inc, 2022
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by jCyte Inc, 2022
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Katairo GmbH, 2022
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Kubota Vision Inc, 2022
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Lysoclear Inc, 2022
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Mediphage Bioceuticals Inc, 2022
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Nanoscope Therapeutics Inc, 2022
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Oak bay Biosciences Ltd, 2022
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by ProQR Therapeutics NV, 2022
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Ray Therapeutics Inc, 2022
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by ReVision Therapeutics Inc, 2022
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Saliogen Therapeutics Inc, 2022
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Shape Therapeutics Inc, 2022
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Spark Therapeutics Inc, 2022
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Splice Bio SL, 2022
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Stargazer Pharmaceuticals Inc, 2022
Juvenile Macular Degeneration (Stargardt Disease) - Dormant Projects, 2022

List of Figures
Number of Products under Development for Juvenile Macular Degeneration (Stargardt Disease), 2022
Number of Products under Development by Companies, 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 Routes of Administration, 2022
Number of Products by Stage and Routes of Administration, 2022
Number of Products by Molecule Types, 2022
Number of Products by Stage and Molecule Types, 2022

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