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Juvenile Macular Degeneration (Stargardt Disease) - Pipeline Review, H2 2019

  • ID: 4866589
  • Drug Pipelines
  • November 2019
  • Region: Global
  • 93 pages
  • Global Markets Direct
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FEATURED COMPANIES

  • Acucela Inc
  • Beam Therapeutics Inc
  • Biophytis SA
  • Generation Bio Corp
  • IVERIC bio Inc
  • Lin Bioscience Inc
  • MORE
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline Review, H2 2019

Summary

This latest Pharmaceutical and Healthcare disease pipeline guide Juvenile Macular Degeneration - Pipeline Review, H2 2019, 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

This Pharmaceutical and Healthcare latest pipeline guide Juvenile Macular Degeneration - Pipeline Review, H2 2019, 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 1, 5, 2, 9 and 4 respectively. Similarly, the Universities portfolio in Preclinical stages comprises 3 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 the publisher'sproprietary 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.
Note: Product cover images may vary from those shown
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FEATURED COMPANIES

  • Acucela Inc
  • Beam Therapeutics Inc
  • Biophytis SA
  • Generation Bio Corp
  • IVERIC bio Inc
  • Lin Bioscience Inc
  • MORE
Introduction
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
Acucela Inc
Alkeus Pharmaceuticals Inc
Alpine Biotherapeutics Corp
Astellas Pharma Inc
Beam Therapeutics Inc
Biogen Inc
Biophytis SA
Cardax Inc
Copernicus Therapeutics Inc
Generation Bio Corp
Grupo Ferrer Internacional SA
Ichor Therapeutics Inc
IVERIC bio Inc
Katairo GmbH
Lin Bioscience Inc
ProQR Therapeutics NV
Sanofi
Spark Therapeutics Inc
Stargazer Pharmaceuticals Inc
Vision Medicines Inc
WAVE Life Sciences Ltd
Juvenile Macular Degeneration (Stargardt Disease) - Drug Profiles
ALK-001 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
ASP-7317 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
avacincaptad pegol sodium - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
BIO-201 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
CDX-301 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
emixustat hydrochloride - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
FAB-111 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy 1 to Activate ABCA4 for Stargardt Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy for Leber’s Congenital Amaurosis and Stargardt Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate ABCA4 for Stargardt Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
LBS-008 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
NSRABCA-4 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Oligonucleotides to Activate ABCA4 for Stargardt Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
QR-1011 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Recombinant Enzyme for Age Related Macular Degeneration and Stargardt Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SAR-422459 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
soraprazan - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Stem Cell Therapy for Ophthalmology - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
STG-001 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
VM-200 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Juvenile Macular Degeneration (Stargardt Disease) - Dormant Projects
Juvenile Macular Degeneration (Stargardt Disease) - Product Development Milestones
Featured News & Press Releases
Jun 26, 2019: Biophytis announces it presented one oral and one poster presentation at the MaculART meeting in Paris, France
Jun 09, 2019: Acucela receives Orphan Designation From the EMA for Emixustat for the Treatment of Stargardt Disease
Apr 26, 2019: Belite Bio to trial new therapy for macular degeneration
Nov 12, 2018: Acucela enrols first patient in Phase III emixustat trial
Sep 18, 2018: FDA grants Lin BioScience Rare Pediatric Disease status for LBS-008 to treat Stargardt Disease
Jun 27, 2018: Lin BioScience Receives EMA Orphan Drug Status for LBS-008 for the Treatment of Stargardt Disease
Oct 26, 2017: Lin BioScience Receives US FDA Orphan Drug Status for LBS-008 for the Treatment of Stargardt Disease
Sep 10, 2017: Acucela Receives New Patent Covering Method of Use for Emixustat Hydrochloride
Jul 26, 2017: Ophthotech Expands Focus with Development for Ophthalmic Orphan Diseases, Provides Update on Zimura
Jan 26, 2017: Acucela Initiates Phase 2a Study of Emixustat Hydrochloride Addressing Patients with Stargardt Disease
Jan 05, 2017: Acucela Receives Orphan Drug Designation from the FDA for the Treatment of Stargardt Disease
May 26, 2015: Ocata Therapeutics Receives Three New U.S. Patents for its RPE Therapy for Macular Degenerative Diseases
Mar 31, 2015: Ocata Therapeutics Successfully Completes Dosing in Phase 1/2 RPE Studies
Oct 15, 2014: ACT Announces Positive Results from Two Clinical Trials Published in The Lancet Using Differentiated Stem Cell-Derived Retinal Pigment Epithelium (RPE) Cells for the Treatment of Macular Degeneration
Sep 25, 2014: Advanced Cell Technology Announces Final Patient Treated in Stargardt’s Macular Degeneration Phase 1 Trial in the United Kingdom
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer

List of Tables
Table 1: Number of Products under Development for Juvenile Macular Degeneration (Stargardt Disease), H2 2019
Table 2: Number of Products under Development by Companies, H2 2019
Table 3: Number of Products under Development by Companies, H2 2019 (Contd..1), H2 2019
Table 4: Number of Products under Development by Universities/Institutes, H2 2019
Table 5: Products under Development by Companies, H2 2019
Table 6: Products under Development by Companies, H2 2019 (Contd..1), H2 2019
Table 7: Products under Development by Universities/Institutes, H2 2019
Table 8: Number of Products by Stage and Target, H2 2019
Table 9: Number of Products by Stage and Mechanism of Action, H2 2019
Table 10: Number of Products by Stage and Route of Administration, H2 2019
Table 11: Number of Products by Stage and Molecule Type, H2 2019
Table 12: Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Acucela Inc, H2 2019
Table 13: Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Alkeus Pharmaceuticals Inc, H2 2019
Table 14: Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Alpine Biotherapeutics Corp, H2 2019
Table 15: Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Astellas Pharma Inc, H2 2019
Table 16: Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Beam Therapeutics Inc, H2 2019
Table 17: Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Biogen Inc, H2 2019
Table 18: Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Biophytis SA, H2 2019
Table 19: Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Cardax Inc, H2 2019
Table 20: Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Copernicus Therapeutics Inc, H2 2019
Table 21: Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Generation Bio Corp, H2 2019
Table 22: Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Grupo Ferrer Internacional SA, H2 2019
Table 23: Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Ichor Therapeutics Inc, H2 2019
Table 24: Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by IVERIC bio Inc, H2 2019
Table 25: Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Katairo GmbH, H2 2019
Table 26: Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Lin Bioscience Inc, H2 2019
Table 27: Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by ProQR Therapeutics NV, H2 2019
Table 28: Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Sanofi, H2 2019
Table 29: Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Spark Therapeutics Inc, H2 2019
Table 30: Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Stargazer Pharmaceuticals Inc, H2 2019
Table 31: Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Vision Medicines Inc, H2 2019
Table 32: Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by WAVE Life Sciences Ltd, H2 2019
Table 33: Juvenile Macular Degeneration (Stargardt Disease) - Dormant Projects, H2 2019

List of Figures
Figure 1: Number of Products under Development for Juvenile Macular Degeneration (Stargardt Disease), H2 2019
Figure 2: Number of Products under Development by Companies, H2 2019
Figure 3: Number of Products by Targets, H2 2019
Figure 4: Number of Products by Stage and Targets, H2 2019
Figure 5: Number of Products by Mechanism of Actions, H2 2019
Figure 6: Number of Products by Stage and Mechanism of Actions, H2 2019
Figure 7: Number of Products by Routes of Administration, H2 2019
Figure 8: Number of Products by Stage and Top 10 Routes of Administration, H2 2019
Figure 9: Number of Products by Molecule Types, H2 2019
Figure 10: Number of Products by Stage and Top 10 Molecule Types, H2 2019
Note: Product cover images may vary from those shown
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  • Acucela Inc
  • Alkeus Pharmaceuticals Inc
  • Alpine Biotherapeutics Corp
  • Astellas Pharma Inc
  • Beam Therapeutics Inc
  • Biogen Inc
  • Biophytis SA
  • Cardax Inc
  • Copernicus Therapeutics Inc
  • Generation Bio Corp
  • Grupo Ferrer Internacional SA
  • Ichor Therapeutics Inc
  • IVERIC bio Inc
  • Katairo GmbH
  • Lin Bioscience Inc
  • ProQR Therapeutics NV
  • Sanofi
  • Spark Therapeutics Inc
  • Stargazer Pharmaceuticals Inc
  • Vision Medicines Inc
  • WAVE Life Sciences Ltd
Note: Product cover images may vary from those shown
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