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Retinal Degeneration - Pipeline Review, H2 2015 Is Released

Press Release   •   Jan 29, 2016 16:57 IST

Retinal Degeneration - Pipeline Review, H2 2015 Summary Global Markets Direct s, Retinal Degeneration - Pipeline Review, H2 2015, provides an overview of the Retinal Degenerations 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 Directs 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 Directs 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.

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Table of Contents

ntroduction 8

Global Markets Direct Report Coverage 8

Retinal Degeneration Overview 9

Therapeutics Development 10

Pipeline Products for Retinal Degeneration - Overview 10

Pipeline Products for Retinal Degeneration - Comparative Analysis 11

Retinal Degeneration - Therapeutics under Development by Companies 12

Retinal Degeneration - Therapeutics under Investigation by Universities/Institutes 13

Retinal Degeneration - Pipeline Products Glance 14

Late Stage Products 14

Clinical Stage Products 15

Early Stage Products 16

Unknown Stage Products 17

Retinal Degeneration - Products under Development by Companies 18

Retinal Degeneration - Products under Investigation by Universities/Institutes 19

Retinal Degeneration - Companies Involved in Therapeutics Development 20

2-BBB Medicines BV 20

Alkeus Pharmaceuticals, Inc. 21

Applied Genetic Technologies Corporation 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

Retinal Degeneration - Therapeutics Assessment 29

Assessment by Monotherapy Products 29

Assessment by Target 30

Assessment by Mechanism of Action 32

Assessment by Route of Administration 34

Assessment by Molecule Type 36

Drug Profiles 38

BVA-202 - Drug Profile 38

Product Description 38

Mechanism of Action 38

R&D Progress 38

BVA-203 - Drug Profile 39

Product Description 39

Mechanism of Action 39

R&D Progress 39

CB-11 - Drug Profile 40

Product Description 40

Mechanism of Action 40

R&D Progress 40

CNS-102 - Drug Profile 41

Product Description 41

Mechanism of Action 41

R&D Progress 41

Drug for Lebers Hereditary Optic Neuropathy - Drug Profile 42

Product Description 42

Mechanism of Action 42

R&D Progress 42

Gene Therapy for X-linked retinoschisis - Drug Profile 43

Product Description 43

Mechanism of Action 43

R&D Progress 43

Gene Therapy to Activate ND4 for Leber's Hereditary Optic Neuropathy - Drug Profile 44

Product Description 44

Mechanism of Action 44

R&D Progress 44

Gene Therapy to Activate Retinoschisin for X-Linked Retinoschisis - Drug Profile 45

Product Description 45

Mechanism of Action 45

R&D Progress 45

GS-010 - Drug Profile 46

Product Description 46

Mechanism of Action 46

R&D Progress 46

GS-011 - Drug Profile 48

Product Description 48

Mechanism of Action 48

R&D Progress 48

idebenone - Drug Profile 49

Product Description 49

Mechanism of Action 49

R&D Progress 49

KH-176 - Drug Profile 52

Product Description 52

Mechanism of Action 52

R&D Progress 52

MTP-131 - Drug Profile 53

Product Description 53

Mechanism of Action 53

R&D Progress 53

SB-623 - 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 57

Product Description 57

Mechanism of Action 57

R&D Progress 57

Small Molecules for Inherited Retinal Diseases - Drug Profile 58

Product Description 58

Mechanism of Action 58

R&D Progress 58

Small Molecules to Agonize Neurotrophin Receptor for CNS and Ophthalmological Disorders - 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 74

Retinal Degeneration - Product Development Milestones 75

Featured News & Press Releases 75

Jun 26, 2015: CHMP recommends granting a marketing authorization for Santhera's Raxone for the treatment of Leber's Hereditary Optic Neuropathy 75

Jun 05, 2014: European Medicines Agency Validates Santhera s Marketing Authorization Application for Raxone in Lebers Hereditary Optic Neuropathy 75

May 07, 2014: Santhera Files Marketing Authorization Application in European Union for Raxone in the Treatment of LHON 76

Mar 04, 2014: Santhera Presents New Efficacy Data from an Expanded Access Program with Raxone for the Treatment of Leber's Hereditary Optic Neuropathy 77

Jan 21, 2014: Temporary Authorization for Use Granted for Santhera's Raxone for the Treatment of Leber's Hereditary Optic Neuropathy in France 78

Jul 11, 2013: Santhera Initiates Collaboration with the European Vision Institute Clinical Research Network in LHON 79

Mar 22, 2013: Santhera Pharma Withdraws Marketing Authorization Application for Raxone In LHON 79

Jan 18, 2013: Santhera Pharma Receives Negative Opinion from EMA's CHMP On Marketing Authorization Application For Raxone 80

Nov 16, 2012: Santhera Pharma Expects CHMP Opinion on MAA for Raxone In Early 2013 81

Jul 26, 2011: Catena Improves Sight for Patients With Inherited Blindness, Newcastle University Study Finds 81

Appendix 83

Methodology 83

Coverage 83

Secondary Research 83

Primary Research 83

Expert Panel Validation 83

Contact Us 83

Disclaimer 84

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