Retinal Degeneration - Pipeline Review, H2 2015 Is

Released
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
2

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
3

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
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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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