Juvenile Macular Degeneration (Stargardt Disease) - Pipeline Review, H2 2018

Global Markets Direct’s, ‘Juvenile Macular Degeneration (Stargardt Disease) - Pipeline Review, H2 2018’, provides an overview of the Juvenile Macular Degeneration (Stargardt Disease) pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Juvenile Macular Degeneration (Stargardt Disease), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Juvenile Macular Degeneration (Stargardt Disease) and features dormant and discontinued projects.

Global Markets Direct’s report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report 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. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report 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.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data.

Scope

- The report provides a snapshot of the global therapeutic landscape of Juvenile Macular Degeneration (Stargardt Disease)

- The report reviews pipeline therapeutics for Juvenile Macular Degeneration (Stargardt Disease) by companies and universities/research institutes based on information derived from company and industry-specific sources

- The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages

- The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities

- The report reviews key players involved Juvenile Macular Degeneration (Stargardt Disease) therapeutics and enlists all their major and minor projects

- The report assesses Juvenile Macular Degeneration (Stargardt Disease) therapeutics based on drug target, mechanism of action (MoA), route of administration (RoA) and molecule type

- The report summarizes all the dormant and discontinued pipeline projects

- The report reviews latest news related to pipeline therapeutics for Juvenile Macular Degeneration (Stargardt Disease)

Reasons to buy

- Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies

- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage

- Identify and understand important and diverse types of therapeutics under development for Juvenile Macular Degeneration (Stargardt Disease)

- Identify potential new clients or partners in the target demographic

- Develop strategic initiatives by understanding the focus areas of leading companies

- Plan mergers and acquisitions effectively by identifying key players and it’s most promising pipeline therapeutics

- Devise corrective measures for pipeline projects by understanding Juvenile Macular Degeneration (Stargardt Disease) 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

- Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline

Companies mentioned

Acucela Inc

Alkeus Pharmaceuticals Inc

Astellas Pharma Inc

Biophytis SA

Copernicus Therapeutics Inc

Grupo Ferrer Internacional SA

Iris Pharma

Katairo GmbH

Lin Bioscience Inc

Nightstar ...

Acucela Inc

Alkeus Pharmaceuticals Inc

Astellas Pharma Inc

Biophytis SA

Copernicus Therapeutics Inc

Grupo Ferrer Internacional SA

Iris Pharma

Katairo GmbH

Lin Bioscience Inc

Nightstar Therapeutics PLC

Ophthotech Corp

ProQR Therapeutics NV

Sanofi

Table of Contents

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 ...

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

Acucela Inc

Alkeus Pharmaceuticals Inc

Astellas Pharma Inc

Biophytis SA

Copernicus Therapeutics Inc

Grupo Ferrer Internacional SA

Iris Pharma

Katairo GmbH

Lin Bioscience Inc

Nightstar Therapeutics PLC

Ophthotech Corp

ProQR Therapeutics NV

Sanofi

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

echothiophate iodide – 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 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

QRX-1011 – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ramiprilat – 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

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

May 04, 2015: European Medicines Agency Grants Orphan Designation for Makindus' MI-100 for Stargardt’s Disease

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

May 16, 2013: ACT Confirms Clinical Trial Participant Shows Improvement In Vision Following Treatment With Human Embryonic Stem Cells

Apr 23, 2013: ACT Initiates Higher-Dosage Patient Treatment In European Phase I Study For Macular Degeneration

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 Juvenile Macular Degeneration (Stargardt Disease), H2 2018

Number of Products under Development by Companies, H2 2018 ...

List of Tables

Number of Products under Development for Juvenile Macular Degeneration (Stargardt Disease), H2 2018

Number of Products under Development by Companies, H2 2018

Number of Products under Development by Universities/Institutes, H2 2018

Products under Development by Companies, H2 2018

Products under Development by Companies, H2 2018 (Contd..1), H2 2018

Products under Development by Universities/Institutes, H2 2018

Number of Products by Stage and Target, H2 2018

Number of Products by Stage and Mechanism of Action, H2 2018

Number of Products by Stage and Route of Administration, H2 2018

Number of Products by Stage and Molecule Type, H2 2018

Juvenile Macular Degeneration (Stargardt Disease) – Pipeline by Acucela Inc, H2 2018

Juvenile Macular Degeneration (Stargardt Disease) – Pipeline by Alkeus Pharmaceuticals Inc, H2 2018

Juvenile Macular Degeneration (Stargardt Disease) – Pipeline by Astellas Pharma Inc, H2 2018

Juvenile Macular Degeneration (Stargardt Disease) – Pipeline by Biophytis SA, H2 2018

Juvenile Macular Degeneration (Stargardt Disease) – Pipeline by Copernicus Therapeutics Inc, H2 2018

Juvenile Macular Degeneration (Stargardt Disease) – Pipeline by Grupo Ferrer Internacional SA, H2 2018

Juvenile Macular Degeneration (Stargardt Disease) – Pipeline by Iris Pharma, H2 2018

Juvenile Macular Degeneration (Stargardt Disease) – Pipeline by Katairo GmbH, H2 2018

Juvenile Macular Degeneration (Stargardt Disease) – Pipeline by Lin Bioscience Inc, H2 2018

Juvenile Macular Degeneration (Stargardt Disease) – Pipeline by Nightstar Therapeutics PLC, H2 2018

Juvenile Macular Degeneration (Stargardt Disease) – Pipeline by Ophthotech Corp, H2 2018

Juvenile Macular Degeneration (Stargardt Disease) – Pipeline by ProQR Therapeutics NV, H2 2018

Juvenile Macular Degeneration (Stargardt Disease) – Pipeline by Sanofi, H2 2018

Juvenile Macular Degeneration (Stargardt Disease) – Dormant Projects, H2 2018

List of Figures

List of Figures

Number of Products under Development for Juvenile Macular Degeneration (Stargardt Disease), H2 2018

Number of Products under Development by Companies, H2 2018 ...

List of Figures

Number of Products under Development for Juvenile Macular Degeneration (Stargardt Disease), H2 2018

Number of Products under Development by Companies, H2 2018

Number of Products by Targets, H2 2018

Number of Products by Stage and Targets, H2 2018

Number of Products by Mechanism of Actions, H2 2018

Number of Products by Stage and Mechanism of Actions, H2 2018

Number of Products by Routes of Administration, H2 2018

Number of Products by Stage and Routes of Administration, H2 2018

Number of Products by Molecule Types, H2 2018

Number of Products by Stage and Molecule Types, H2 2018

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