Juvenile Macular Degeneration (Stargardt Disease) - Pipeline Review, H2 2014

Published: Nov, 2014 | Pages: 60 | Publisher: Global Markets Direct
Industry: Healthcare | Report Format: Electronic (PDF)

Juvenile Macular Degeneration (Stargardt Disease) - Pipeline Review, H2 2014

Summary

Global Markets Direct's, 'Juvenile Macular Degeneration (Stargardt Disease) - Pipeline Review, H2 2014', provides an overview of the Juvenile Macular Degeneration (Stargardt Disease)'s therapeutic pipeline.

This report provides comprehensive information on the therapeutic development for Juvenile Macular 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.

The report enhances decision making capabilities and help to create effective counter strategies to gain competitive advantage. It strengthens R&D pipelines by identifying new targets and MOAs to produce first-in-class and best-in-class products.

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

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

Reasons to buy

- Provides strategically significant competitor information, analysis, and insights to formulate effective R&D development strategies
- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
- Develop strategic initiatives by understanding the focus areas of leading companies
- Identify and understand important and diverse types of therapeutics under development for Juvenile Macular Degeneration (Stargardt Disease)
- Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline
- 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

 Table of Contents
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
Unknown Stage Products 13
Juvenile Macular Degeneration (Stargardt Disease) - Products under Development by Companies 14
Juvenile Macular Degeneration (Stargardt Disease) - Products under Investigation by Universities/Institutes 15
Juvenile Macular Degeneration (Stargardt Disease) - Companies Involved in Therapeutics Development 16
Acucela Inc. 16
Advanced Cell Technology, Inc. 17
Alkeus Pharmaceuticals, Inc. 18
Sanofi 19
Juvenile Macular Degeneration (Stargardt Disease) - Therapeutics Assessment 20
Assessment by Monotherapy Products 20
Assessment by Target 21
Assessment by Mechanism of Action 23
Assessment by Route of Administration 25
Assessment by Molecule Type 27
Drug Profiles 29
ALK-001 - Drug Profile 29
Product Description 29
Mechanism of Action 29
R&D Progress 29
echothiophate iodide - Drug Profile 31
Product Description 31
Mechanism of Action 31
R&D Progress 31
emixustat hydrochloride - Drug Profile 32
Product Description 32
Mechanism of Action 32
R&D Progress 32
Gene Therapy for Stargardt Disease - Drug Profile 34
Product Description 34
Mechanism of Action 34
R&D Progress 34
MA09-hRPE - Drug Profile 35
Product Description 35
Mechanism of Action 35
R&D Progress 35
Small Molecule for Stargardt Disease - Drug Profile 37
Product Description 37
Mechanism of Action 37
R&D Progress 37
StarGen - Drug Profile 38
Product Description 38
Mechanism of Action 38
R&D Progress 38
VSM-20R - Drug Profile 40
Product Description 40
Mechanism of Action 40
R&D Progress 40
Juvenile Macular Degeneration (Stargardt Disease) - Recent Pipeline Updates 41
Juvenile Macular Degeneration (Stargardt Disease) - Product Development Milestones 52
Featured News & Press Releases 52
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 52
Sep 25, 2014: Advanced Cell Technology Announces Final Patient Treated in Stargardt's Macular Degeneration Phase 1 Trial in the United Kingdom 53
Jul 17, 2013: ACT Secures Approval from Data Safety Monitoring Board to Complete Third Patient Cohort in All Three Clinical Trials 53
May 16, 2013: ACT Confirms Clinical Trial Participant Shows Improvement In Vision Following Treatment With Human Embryonic Stem Cells 54
Apr 23, 2013: ACT Initiates Higher-Dosage Patient Treatment In European Phase I Study For Macular Degeneration 54
Apr 15, 2013: ACT Treats First Patient With Better Vision In Clinical Trial For Stargardt's Macular Dystrophy 55
Apr 01, 2013: ACT Initiates Treatment Of Higher-dosage Cohort In Clinical Trials For Dry Age-related Macular Degeneration And Stargardt's Macular Dystrophy 55
Mar 14, 2013: Advanced Cell Technology Receives DSMB Approval To Initiate Treatment Of Third Patient Cohort In All Three Clinical Trials Using hESC-derived RPE Cells 56
Jan 08, 2013: Advanced Cell Technology Achieves Clinical Milestone 57
Dec 17, 2012: ACT Completes Higher-Dosage Cohort In European Clinical Trial For Stargardt's Macular Dystrophy Using Retinal Pigment Epithelial Cells 58
Appendix 59
Methodology 59
Coverage 59
Secondary Research 59
Primary Research 59
Expert Panel Validation 59
Contact Us 60
Disclaimer 6

List of Tables
Number of Products under Development for Juvenile Macular Degeneration (Stargardt Disease), H2 2014 7
Number of Products under Development for Juvenile Macular Degeneration (Stargardt Disease) - Comparative Analysis, H2 2014 8
Number of Products under Development by Companies, H2 2014 9
Number of Products under Investigation by Universities/Institutes, H2 2014 10
Comparative Analysis by Clinical Stage Development, H2 2014 11
Comparative Analysis by Early Stage Development, H2 2014 12
Comparative Analysis by Unknown Stage Development, H2 2014 13
Products under Development by Companies, H2 2014 14
Products under Investigation by Universities/Institutes, H2 2014 15
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Acucela Inc., H2 2014 16
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Advanced Cell Technology, Inc., H2 2014 17
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Alkeus Pharmaceuticals, Inc., H2 2014 18
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Sanofi, H2 2014 19
Assessment by Monotherapy Products, H2 2014 20
Number of Products by Stage and Target, H2 2014 22
Number of Products by Stage and Mechanism of Action, H2 2014 24
Number of Products by Stage and Route of Administration, H2 2014 26
Number of Products by Stage and Molecule Type, H2 2014 28
Juvenile Macular Degeneration (Stargardt Disease) Therapeutics - Recent Pipeline Updates, H2 2014 41