Report On: Juvenile Macular Degeneration (Stargardt Disease) Industry 2015-2020

This report provides comprehensive information on the therapeutic development for JuvenileMacular Degeneration (Stargardt Disease), complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type, along with latest updates, and featured news and press releases. It also reviews key players involved in the therapeutic development for Juvenile Macular Degeneration (Stargardt Disease) and special features on late-stage 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, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together by Global Markets Direct's team. Drug profiles/records featured in the report undergoes periodic updation following a stringent set of processes that ensures that all the profiles are updated with the latest set of information. Additionally, processes including live news & deals tracking, browser based alert-box and clinical trials registries tracking ensure that the most recent developments are captured on a real time basis.

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Scope

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

- The report reviews key pipeline products under drug profile section which includes, product description, MoA and R&D brief, licensing and collaboration details & other developmental activities

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

- The report summarizes all the dormant and discontinued pipeline projects

- A review of the Juvenile Macular Degeneration (Stargardt Disease) products under development by companies and universities/research institutes based on information derived from company and industry-specific sources

- Pipeline products coverage based on various stages of development ranging from pre-registration till discovery and undisclosed stages

- A detailed assessment of monotherapy and combination therapy pipeline projects

- Coverage of the Juvenile Macular Degeneration (Stargardt Disease) pipeline on the basis of target, MoA, route of administration and molecule type

- Latest news and deals relating related to pipeline products

Table Of Content

Table of Contents 2

List of Tables 4

List of Figures 4

Introduction 5

Global Markets Direct Report Coverage 5

Juvenile Macular Degeneration (Stargardt Disease) Overview 6

Therapeutics Development 7

Pipeline Products for Juvenile Macular Degeneration (Stargardt Disease) - Overview 7

Pipeline Products for Juvenile Macular Degeneration (Stargardt Disease) - Comparative Analysis 8

Juvenile Macular Degeneration (Stargardt Disease) - Therapeutics under Development by Companies 9

Juvenile Macular Degeneration (Stargardt Disease) - Therapeutics under Investigation by Universities/Institutes 10

Juvenile Macular Degeneration (Stargardt Disease) - Pipeline Products Glance 11

Clinical Stage Products 11

Early Stage Products 12

Juvenile Macular Degeneration (Stargardt Disease) - Products under Development by Companies 13

Juvenile Macular Degeneration (Stargardt Disease) - Products under Investigation by Universities/Institutes 14

Juvenile Macular Degeneration (Stargardt Disease) - Companies Involved in Therapeutics Development 15

Acucela Inc. 15

Alkeus Pharmaceuticals, Inc. 16

Ocata Therapeutics, Inc. 17

Sanofi 18

Juvenile Macular Degeneration (Stargardt Disease) - Therapeutics Assessment 19

Assessment by Monotherapy Products 19

Assessment by Target 20

Assessment by Mechanism of Action 22

Assessment by Route of Administration 24

Assessment by Molecule Type 26

Drug Profiles 28

ALK-001 - Drug Profile 28

Product Description 28

Mechanism of Action 28

R&D Progress 28

echothiophate iodide - Drug Profile 30

Product Description 30

Mechanism of Action 30

R&D Progress 30

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emixustat hydrochloride - Drug Profile 31

Product Description 31

Mechanism of Action 31

R&D Progress 31

Gene Therapy for Stargardt Disease - Drug Profile 33

Product Description 33

Mechanism of Action 33

R&D Progress 33

MA09-hRPE - Drug Profile 34

Product Description 34

Mechanism of Action 34

R&D Progress 34

SAR-422459 - Drug Profile 36

Product Description 36

Mechanism of Action 36

R&D Progress 36

Small Molecule for Stargardt Disease - Drug Profile 37

Product Description 37

Mechanism of Action 37

R&D Progress 37

Stem Cell Therapy for Ophthalmic Disorders - Drug Profile 38

Product Description 38

Mechanism of Action 38

R&D Progress 38

VSM-20R - Drug Profile 39

Product Description 39

Mechanism of Action 39

R&D Progress 39

Juvenile Macular Degeneration (Stargardt Disease) - Recent Pipeline Updates 40

Juvenile Macular Degeneration (Stargardt Disease) - Product Development Milestones 53

Featured News & Press Releases 53

May 26, 2015: Ocata Therapeutics Receives Three New U.S. Patents for its RPE Therapy for Macular Degenerative Diseases 53

May 04, 2015: European Medicines Agency Grants Orphan Designation for Makindus' MI-100 for Stargardt's Disease 53

Mar 31, 2015: Ocata Therapeutics Successfully Completes Dosing in Phase 1/2 RPE Studies 54

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 54

Sep 25, 2014: Advanced Cell Technology Announces Final Patient Treated in Stargardt's Macular Degeneration Phase 1 Trial in the United Kingdom 55

Jul 17, 2013: ACT Secures Approval from Data Safety Monitoring Board to Complete Third Patient Cohort in All Three Clinical Trials 56

May 16, 2013: ACT Confirms Clinical Trial Participant Shows Improvement In Vision Following Treatment With Human Embryonic Stem Cells 56

Apr 23, 2013: ACT Initiates Higher-Dosage Patient Treatment In European Phase I Study For Macular Degeneration 57

Apr 15, 2013: ACT Treats First Patient With Better Vision In Clinical Trial For Stargardt's Macular Dystrophy 57

Apr 01, 2013: ACT Initiates Treatment Of Higher-dosage Cohort In Clinical Trials For Dry Age-related Macular Degeneration And Stargardt's Macular Dystrophy 58

Appendix 59

Methodology 59

Coverage 59

Secondary Research 59

Primary Research 59

Expert Panel Validation 59

Contact Us 59

Disclaimer 60

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