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Juvenile Macular Degeneration (Stargardt Disease) - Pipeline Review, H1 2019

  • ID: 4760834
  • Drug Pipelines
  • 90 pages
  • Global Markets Direct
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FEATURED COMPANIES

  • Acucela Inc
  • Astellas Pharma Inc
  • Biophytis SA
  • Generation Bio Corp
  • Katairo GmbH
  • Lin Bioscience Inc
  • MORE
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline Review, H1 2019

Summary

This latest Pharmaceutical and Healthcare disease pipeline guide Juvenile Macular Degeneration (Stargardt Disease) - Pipeline Review, H1 2019, provides an overview of the Juvenile Macular Degeneration (Stargardt Disease) (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 latest pipeline guide Juvenile Macular Degeneration (Stargardt Disease) - Pipeline Review, H1 2019, provides comprehensive information on the therapeutics under development for Juvenile Macular Degeneration (Stargardt Disease) (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 (Stargardt Disease) (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, 4, 1, 10 and 2 respectively. Similarly, the Universities portfolio in Preclinical stages comprises 3 molecules, respectively.

Juvenile Macular Degeneration (Stargardt Disease) (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 '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 Juvenile Macular Degeneration (Stargardt Disease) (Ophthalmology).
  • The pipeline guide reviews pipeline therapeutics for Juvenile Macular Degeneration (Stargardt Disease) (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 (Stargardt Disease) (Ophthalmology) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Juvenile Macular Degeneration (Stargardt Disease) (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 (Stargardt Disease) (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 (Stargardt Disease) (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 (Stargardt Disease) (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
  • Astellas Pharma Inc
  • Biophytis SA
  • Generation Bio Corp
  • Katairo GmbH
  • 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

Astellas Pharma Inc

Biophytis SA

Copernicus Therapeutics Inc

Generation Bio Corp

Grupo Ferrer Internacional SA

Katairo GmbH

Lin Bioscience Inc

Nightstar Therapeutics PLC

Ophthotech Corp

ProQR Therapeutics NV

Sanofi

Spark Therapeutics 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

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

Gene Therapy to Activate ABCA4 for 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

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

QRX-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 Ophthalmic Disorders - 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

Feb 07, 2019: Oxford BioMedica provides update on its ophthalmology gene therapy programme SAR422459 outlicensed to Sanofi

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

Jul 17, 2013: ACT Secures Approval from Data Safety Monitoring Board to Complete Third Patient Cohort in All Three Clinical Trials

Jun 03, 2013: Oxford BioMedica Halts Recruitment Into Phase I/IIa Study Of StarGen

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), H1 2019

Number of Products under Development by Companies, H1 2019

Number of Products under Development by Universities/Institutes, H1 2019

Products under Development by Companies, H1 2019

Products under Development by Universities/Institutes, H1 2019

Number of Products by Stage and Target, H1 2019

Number of Products by Stage and Mechanism of Action, H1 2019

Number of Products by Stage and Route of Administration, H1 2019

Number of Products by Stage and Molecule Type, H1 2019

Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Acucela Inc, H1 2019

Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Alkeus Pharmaceuticals Inc, H1 2019

Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Astellas Pharma Inc, H1 2019

Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Biophytis SA, H1 2019

Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Copernicus Therapeutics Inc, H1 2019

Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Generation Bio Corp, H1 2019

Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Grupo Ferrer Internacional SA, H1 2019

Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Katairo GmbH, H1 2019

Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Lin Bioscience Inc, H1 2019

Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Nightstar Therapeutics PLC, H1 2019

Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Ophthotech Corp, H1 2019

Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by ProQR Therapeutics NV, H1 2019

Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Sanofi, H1 2019

Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Spark Therapeutics Inc, H1 2019

Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by WAVE Life Sciences Ltd, H1 2019

Juvenile Macular Degeneration (Stargardt Disease) - Dormant Projects, H1 2019

List of Figures

Number of Products under Development for Juvenile Macular Degeneration (Stargardt Disease), H1 2019

Number of Products under Development by Companies, H1 2019

Number of Products by Targets, H1 2019

Number of Products by Stage and Targets, H1 2019

Number of Products by Mechanism of Actions, H1 2019

Number of Products by Stage and Mechanism of Actions, H1 2019

Number of Products by Routes of Administration, H1 2019

Number of Products by Stage and Routes of Administration, H1 2019

Number of Products by Molecule Types, H1 2019

Number of Products by Stage and Top 10 Molecule Types, H1
Note: Product cover images may vary from those shown
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  • Acucela Inc
  • Alkeus Pharmaceuticals Inc
  • Astellas Pharma Inc
  • Biophytis SA
  • Copernicus Therapeutics Inc
  • Generation Bio Corp
  • Grupo Ferrer Internacional SA
  • Katairo GmbH
  • Lin Bioscience Inc
  • Nightstar Therapeutics PLC
  • Ophthotech Corp
  • ProQR Therapeutics NV
  • Sanofi
  • Spark Therapeutics Inc
  • WAVE Life Sciences Ltd
Note: Product cover images may vary from those shown
5 of 5
Note: Product cover images may vary from those shown
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