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

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    Report

  • 96 Pages
  • April 2022
  • Region: Global
  • Global Markets Direct
  • ID: 5589918
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The 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


The 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 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 it’s 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

IntroductionReport CoverageJuvenile 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 ProjectsJuvenile 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 Emixustat’s 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
AppendixMethodologyCoverageSecondary ResearchPrimary ResearchExpert Panel ValidationContact the Analyst
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 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
  • 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

Companies Mentioned (Partial List)

A selection of companies mentioned in this report includes, but is not limited to:

  • 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