Retinal Degeneration - Pipeline Review, H1 2015

Published: May, 2015 | Pages: 82 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Retinal Degeneration - Pipeline Review, H1 2015

Summary

Global Markets Direct's, 'Retinal Degeneration - Pipeline Review, H1 2015', provides an overview of the Retinal Degeneration's therapeutic pipeline.

This report provides comprehensive information on the therapeutic development for Retinal Degeneration, 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 Retinal Degeneration 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 Retinal Degeneration
- 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 Retinal Degeneration and enlists all their major and minor projects
- The report summarizes all the dormant and discontinued pipeline projects 
- A review of the Retinal Degeneration 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 Retinal Degeneration 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 Retinal Degeneration
- Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline
- Devise corrective measures for pipeline projects by understanding Retinal Degeneration 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 5
List of Figures 6
Introduction 7
Global Markets Direct Report Coverage 7
Retinal Degeneration Overview 8
Therapeutics Development 9
Pipeline Products for Retinal Degeneration - Overview 9
Pipeline Products for Retinal Degeneration - Comparative Analysis 10
Retinal Degeneration - Therapeutics under Development by Companies 11
Retinal Degeneration - Therapeutics under Investigation by Universities/Institutes 12
Retinal Degeneration - Pipeline Products Glance 13
Late Stage Products 13
Clinical Stage Products 14
Early Stage Products 15
Unknown Stage Products 16
Retinal Degeneration - Products under Development by Companies 17
Retinal Degeneration - Products under Investigation by Universities/Institutes 18
Retinal Degeneration - Companies Involved in Therapeutics Development 19
Alkeus Pharmaceuticals, Inc. 19
Applied Genetic Technologies Corporation 20
Aprogen, Inc. 21
BBB Therapeutics B.V. 22
Bionature E.A. Ltd. 23
Biovista Inc. 24
GenSight Biologics SA 25
SanBio, Inc. 26
Santhera Pharmaceuticals Holding AG 27
Senju Pharmaceutical Co., Ltd. 28
Spark Therapeutics, Inc. 29
Retinal Degeneration - Therapeutics Assessment 30
Assessment by Monotherapy Products 30
Assessment by Target 31
Assessment by Mechanism of Action 33
Assessment by Route of Administration 35
Assessment by Molecule Type 37
Drug Profiles 39
AP-202 - Drug Profile 39
Product Description 39
Mechanism of Action 39
R&D Progress 39
BVA-202 - Drug Profile 40
Product Description 40
Mechanism of Action 40
R&D Progress 40
BVA-203 - Drug Profile 41
Product Description 41
Mechanism of Action 41
R&D Progress 41
CB-11 - Drug Profile 42
Product Description 42
Mechanism of Action 42
R&D Progress 42
Drug for Lebers Hereditary Optic Neuropathy - Drug Profile 43
Product Description 43
Mechanism of Action 43
R&D Progress 43
Gene Therapy for Choroideremia - Drug Profile 44
Product Description 44
Mechanism of Action 44
R&D Progress 44
Gene Therapy for Retinal Degenerative Disease - Drug Profile 45
Product Description 45
Mechanism of Action 45
R&D Progress 45
Gene Therapy to Activate Retinoschisin for X-Linked Retinoschisis - Drug Profile 46
Product Description 46
Mechanism of Action 46
R&D Progress 46
GS-010 - Drug Profile 47
Product Description 47
Mechanism of Action 47
R&D Progress 47
idebenone - Drug Profile 48
Product Description 48
Mechanism of Action 48
R&D Progress 48
MTP-131 - Drug Profile 50
Product Description 50
Mechanism of Action 50
R&D Progress 50
rAAV2tYFCB-hRS1 - Drug Profile 52
Product Description 52
Mechanism of Action 52
R&D Progress 52
SB-623 - Drug Profile 53
Product Description 53
Mechanism of Action 53
R&D Progress 53
Small Molecule 8 for Retinitis Pigmentosa - Drug Profile 55
Product Description 55
Mechanism of Action 55
R&D Progress 55
Small Molecule to Activate NF-E2 Related Factor 2 for Central Nervous System, Metabolic Disorders, Cardiovascular and Ophthalmology - Drug Profile 56
Product Description 56
Mechanism of Action 56
R&D Progress 56
Small Molecules for Inherited Retinal Diseases - Drug Profile 57
Product Description 57
Mechanism of Action 57
R&D Progress 57
Small Molecules to Agonize Neurotrophin Receptor for CNS and Ophthalmological Disorders - Drug Profile 58
Product Description 58
Mechanism of Action 58
R&D Progress 58
SNC-121 - Drug Profile 59
Product Description 59
Mechanism of Action 59
R&D Progress 59
SNJ-1945 - Drug Profile 60
Product Description 60
Mechanism of Action 60
R&D Progress 60
Stem Cell Therapy for Cancer, Pain, Retinal Degeneration and Niemann-Pick Disease - Drug Profile 61
Product Description 61
Mechanism of Action 61
R&D Progress 61
Retinal Degeneration - Recent Pipeline Updates 62
Retinal Degeneration - Dormant Projects 72
Retinal Degeneration - Dormant Projects 72
Retinal Degeneration - Product Development Milestones 73
Featured News & Press Releases 73
Jun 05, 2014: European Medicines Agency Validates Santhera's Marketing Authorization Application for Raxone in Leber's Hereditary Optic Neuropathy 73
May 07, 2014: Santhera Files Marketing Authorization Application in European Union for Raxone in the Treatment of LHON 74
Mar 04, 2014: Santhera Presents New Efficacy Data from an Expanded Access Program with Raxone for the Treatment of Leber's Hereditary Optic Neuropathy 74
Jan 21, 2014: Temporary Authorization for Use Granted for Santhera's Raxone for the Treatment of Leber's Hereditary Optic Neuropathy in France 75
Mar 22, 2013: Santhera Pharma Withdraws Marketing Authorization Application For Raxone In LHON 76
Jan 18, 2013: Santhera Pharma Receives Negative Opinion From EMA's CHMP On Marketing Authorization Application For Raxone 77
Nov 16, 2012: Santhera Pharma Expects CHMP Opinion On MAA For Raxone In Early 2013 77
Jul 26, 2011: Catena Improves Sight For Patients With Inherited Blindness, Newcastle University Study Finds 78
Jul 22, 2011: EMA Accepts Santhera's Marketing Authorization Application Of Idebenone For Review In LHON 79
Jun 06, 2011: Santhera Pharma Presents Nonclinical Data In LHON At Joint Congress Of SOE/AAO 2011 79
Appendix 81
Methodology 81
Coverage 81
Secondary Research 81
Primary Research 81
Expert Panel Validation 81
Contact Us 81
Disclaimer 82

List of Tables

Number of Products under Development for Retinal Degeneration, H1 2015 9
Number of Products under Development for Retinal Degeneration - Comparative Analysis, H1 2015 10
Number of Products under Development by Companies, H1 2015 11
Number of Products under Investigation by Universities/Institutes, H1 2015 12
Comparative Analysis by Late Stage Development, H1 2015 13
Comparative Analysis by Clinical Stage Development, H1 2015 14
Comparative Analysis by Early Stage Development, H1 2015 15
Comparative Analysis by Unknown Stage Development, H1 2015 16
Products under Development by Companies, H1 2015 17
Products under Investigation by Universities/Institutes, H1 2015 18
Retinal Degeneration - Pipeline by Alkeus Pharmaceuticals, Inc., H1 2015 19
Retinal Degeneration - Pipeline by Applied Genetic Technologies Corporation, H1 2015 20
Retinal Degeneration - Pipeline by Aprogen, Inc., H1 2015 21
Retinal Degeneration - Pipeline by BBB Therapeutics B.V., H1 2015 22
Retinal Degeneration - Pipeline by Bionature E.A. Ltd., H1 2015 23
Retinal Degeneration - Pipeline by Biovista Inc., H1 2015 24
Retinal Degeneration - Pipeline by GenSight Biologics SA, H1 2015 25
Retinal Degeneration - Pipeline by SanBio, Inc., H1 2015 26
Retinal Degeneration - Pipeline by Santhera Pharmaceuticals Holding AG, H1 2015 27
Retinal Degeneration - Pipeline by Senju Pharmaceutical Co., Ltd., H1 2015 28
Retinal Degeneration - Pipeline by Spark Therapeutics, Inc., H1 2015 29
Assessment by Monotherapy Products, H1 2015 30
Number of Products by Stage and Target, H1 2015 32
Number of Products by Stage and Mechanism of Action, H1 2015 34
Number of Products by Stage and Route of Administration, H1 2015 36
Number of Products by Stage and Molecule Type, H1 2015 38
Retinal Degeneration Therapeutics - Recent Pipeline Updates, H1 2015 62
Retinal Degeneration - Dormant Projects, H1 2015 72