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Retinitis Pigmentosa (Retinitis) - Pipeline Review, H2 2015 Is Released

Press Release   •   Feb 02, 2016 18:38 IST

This report provides comprehensive information on the therapeutic development for Retinitis Pigmentosa (Retinitis), 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 Retinitis Pigmentosa (Retinitis) 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 Directs 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 Retinitis Pigmentosa (Retinitis) - 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 Retinitis Pigmentosa (Retinitis) and enlists all their major and minor projects - The report summarizes all the dormant and discontinued pipeline projects - A review of the Retinitis Pigmentosa (Retinitis) 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 Retinitis Pigmentosa (Retinitis) 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 Retinitis Pigmentosa (Retinitis) - Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline - Devise corrective measures for pipeline projects by understanding Retinitis Pigmentosa (Retinitis) 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

Table of Contents 2

List of Tables 5

List of Figures 6

Introduction 7

Global Markets Direct Report Coverage 7

Retinitis Pigmentosa (Retinitis) Overview 8

Therapeutics Development 9

Pipeline Products for Retinitis Pigmentosa (Retinitis) - Overview 9

Pipeline Products for Retinitis Pigmentosa (Retinitis) - Comparative Analysis 10

Retinitis Pigmentosa (Retinitis) - Therapeutics under Development by Companies 11

Retinitis Pigmentosa (Retinitis) - Therapeutics under Investigation by Universities/Institutes 13

Retinitis Pigmentosa (Retinitis) - Pipeline Products Glance 14

Late Stage Products 14

Clinical Stage Products 15

Early Stage Products 16

Retinitis Pigmentosa (Retinitis) - Products under Development by Companies 17

Retinitis Pigmentosa (Retinitis) - Products under Investigation by Universities/Institutes 20

Retinitis Pigmentosa (Retinitis) - Companies Involved in Therapeutics Development 21

Acucela Inc. 21

Amarantus Bioscience Holdings, Inc. 22

Applied Genetic Technologies Corporation 23

Asklepios BioPharmaceutical, Inc. 24

Caladrius Biosciences, Inc. 25

DNAVEC Corporation 26

Dompe Farmaceutici S.p.A. 27

Genable Technologies Limited 28

Genethon 29

GenSight Biologics SA 30

Grupo Ferrer Internacional, S.A. 31

InFlectis BioScience 32

International Stem Cell Corporation 33

Isis Pharmaceuticals, Inc. 34

Mimetogen Pharmaceuticals Inc. 35

Neurotech Pharmaceuticals, Inc. 36

Ocata Therapeutics, Inc. 37

Orphagen Pharmaceuticals, Inc. 38

QLT Inc. 39

RegenxBio Inc. 40

ReNeuron Group Plc 41

SanBio, Inc. 42

Sanofi 43

Shire Plc 44

Spark Therapeutics, Inc. 45

Sucampo Pharmaceuticals, Inc. 46

Sun Pharma Advanced Research Company Ltd. 47

Retinitis Pigmentosa (Retinitis) - Therapeutics Assessment 48

Assessment by Monotherapy Products 48

Assessment by Target 49

Assessment by Mechanism of Action 51

Assessment by Route of Administration 53

Assessment by Molecule Type 56

Drug Profiles 58

AMRS-001 - Drug Profile 58

Antibodies to Antagonize IL-6 for Retinitis - Drug Profile 61

BNP-RP - Drug Profile 62

CB-11 - Drug Profile 63

ECL-1 - Drug Profile 64

emixustat hydrochloride - Drug Profile 65

FIB-111 - Drug Profile 67

Gene Therapy for Glaucoma and Retinitis Pigmentosa - Drug Profile 68

Gene Therapy for Ocular Diseases - Drug Profile 69

Gene Therapy for Ophthalmology - Drug Profile 70

Gene Therapy for Retinitis Pigmentosa - Drug Profile 71

Gene Therapy for Retinitis Pigmentosa - Drug Profile 72

Gene Therapy to Activate Glutamate Cysteine Ligase and Glutathione Synthase for Retinitis Pigmentosa - Drug Profile 73

Gene Therapy to Activate HDAC4 for Retinitis Pigmentosa - Drug Profile 74

Gene Therapy to Activate RPE65 for Leber Congenital Amaurosis - Drug Profile 75

Gene Therapy to Activate RPGR for X-Linked Retinitis Pigmentosa - Drug Profile 76

Gene Therapy to Activate RPGR for X-Linked Retinitis Pigmentosa - Drug Profile 77

GS-030 - Drug Profile 78

GT-038 - Drug Profile 80

IFB-088 - Drug Profile 82

ISIS-RHO2.5Rx - Drug Profile 83

KIRA-6 - Drug Profile 84

NT-501 - Drug Profile 85

OCU-100 - Drug Profile 88

PRO-015 - Drug Profile 89

proinsulin human SR - Drug Profile 90

Recombinant Protein to Agonize p75NTR and Trk for Ophthalmology - Drug Profile 91

ReN-003 - Drug Profile 93

Retinal Progenitor Cells for Ophthalmological Disorders - Drug Profile 95

RGX-321 - Drug Profile 97

RST-001 - Drug Profile 98

SAR-421869 - Drug Profile 99

SB-623 - Drug Profile 101

SHP-630 - Drug Profile 103

Small Molecule for Retinitis Pigmentosa - Drug Profile 104

Small Molecule to Agonize Neurotrophin Receptor for Retinitis Pigmentosa - Drug Profile 105

Small Molecules for CNS Disorders and Ophthalmology - Drug Profile 106

Small Molecules for Retinitis Pigmentosa - Drug Profile 107

Small Molecules for Retinitis Pigmentosa - Drug Profile 108

Small Molecules to Inhibit Ire1-Alpha for Retinitis Pigmentosa, Type 2 Diabetes and Amyotrophic Lateral Sclerosis - Drug Profile 109

SPKRPE-65 - Drug Profile 110

Stem Cell Therapy for Age Related Macular Degeneration and Retinitis Pigmentosa - Drug Profile 113

Stem Cell Therapy for AMD and Retinitis Pigmentosa - Drug Profile 115

Stem Cell Therapy for Ophthalmic Disorders - Drug Profile 116

Stem Cell Therapy to Activate MFRP for Retinitis Pigmentosa - Drug Profile 117

unoprostone isopropyl - Drug Profile 118

zuretinol acetate - Drug Profile 120

Retinitis Pigmentosa (Retinitis) - Recent Pipeline Updates 122

Retinitis Pigmentosa (Retinitis) - Dormant Projects 145

Retinitis Pigmentosa (Retinitis) - Discontinued Products 146

Retinitis Pigmentosa (Retinitis) - Product Development Milestones 147

Featured News & Press Releases 147

Appendix 156

Methodology 156

Coverage 156

Secondary Research 156

Primary Research 156

Expert Panel Validation 156

Contact Us 156

Disclaimer 157" width="80" height="40" />Table of Contents

Table of Contents 2

List of Tables 5

List of Figures 6

Introduction 7

Global Markets Direct Report Coverage 7

Retinitis Pigmentosa (Retinitis) Overview 8

Therapeutics Development 9

Pipeline Products for Retinitis Pigmentosa (Retinitis) - Overview 9

Pipeline Products for Retinitis Pigmentosa (Retinitis) - Comparative Analysis 10

Retinitis Pigmentosa (Retinitis) - Therapeutics under Development by Companies 11

Retinitis Pigmentosa (Retinitis) - Therapeutics under Investigation by Universities/Institutes 13

Retinitis Pigmentosa (Retinitis) - Pipeline Products Glance 14

Late Stage Products 14

Clinical Stage Products 15

Early Stage Products 16

Retinitis Pigmentosa (Retinitis) - Products under Development by Companies 17

Retinitis Pigmentosa (Retinitis) - Products under Investigation by Universities/Institutes 20

Retinitis Pigmentosa (Retinitis) - Companies Involved in Therapeutics Development 21

Acucela Inc. 21

Amarantus Bioscience Holdings, Inc. 22

Applied Genetic Technologies Corporation 23

Asklepios BioPharmaceutical, Inc. 24

Caladrius Biosciences, Inc. 25

DNAVEC Corporation 26

Dompe Farmaceutici S.p.A. 27

Genable Technologies Limited 28

Genethon 29

GenSight Biologics SA 30

Grupo Ferrer Internacional, S.A. 31

InFlectis BioScience 32

International Stem Cell Corporation 33

Isis Pharmaceuticals, Inc. 34

Mimetogen Pharmaceuticals Inc. 35

Neurotech Pharmaceuticals, Inc. 36

Ocata Therapeutics, Inc. 37

Orphagen Pharmaceuticals, Inc. 38

QLT Inc. 39

RegenxBio Inc. 40

ReNeuron Group Plc 41

SanBio, Inc. 42

Sanofi 43

Shire Plc 44

Spark Therapeutics, Inc. 45

Sucampo Pharmaceuticals, Inc. 46

Sun Pharma Advanced Research Company Ltd. 47

Retinitis Pigmentosa (Retinitis) - Therapeutics Assessment 48

Assessment by Monotherapy Products 48

Assessment by Target 49

Assessment by Mechanism of Action 51

Assessment by Route of Administration 53

Assessment by Molecule Type 56

Drug Profiles 58

AMRS-001 - Drug Profile 58

Antibodies to Antagonize IL-6 for Retinitis - Drug Profile 61

BNP-RP - Drug Profile 62

CB-11 - Drug Profile 63

ECL-1 - Drug Profile 64

emixustat hydrochloride - Drug Profile 65

FIB-111 - Drug Profile 67

Gene Therapy for Glaucoma and Retinitis Pigmentosa - Drug Profile 68

Gene Therapy for Ocular Diseases - Drug Profile 69

Gene Therapy for Ophthalmology - Drug Profile 70

Gene Therapy for Retinitis Pigmentosa - Drug Profile 71

Gene Therapy for Retinitis Pigmentosa - Drug Profile 72

Gene Therapy to Activate Glutamate Cysteine Ligase and Glutathione Synthase for Retinitis Pigmentosa - Drug Profile 73

Gene Therapy to Activate HDAC4 for Retinitis Pigmentosa - Drug Profile 74

Gene Therapy to Activate RPE65 for Leber Congenital Amaurosis - Drug Profile 75

Gene Therapy to Activate RPGR for X-Linked Retinitis Pigmentosa - Drug Profile 76

Gene Therapy to Activate RPGR for X-Linked Retinitis Pigmentosa - Drug Profile 77

GS-030 - Drug Profile 78

GT-038 - Drug Profile 80

IFB-088 - Drug Profile 82

ISIS-RHO2.5Rx - Drug Profile 83

KIRA-6 - Drug Profile 84

NT-501 - Drug Profile 85

OCU-100 - Drug Profile 88

PRO-015 - Drug Profile 89

proinsulin human SR - Drug Profile 90

Recombinant Protein to Agonize p75NTR and Trk for Ophthalmology - Drug Profile 91

ReN-003 - Drug Profile 93

Retinal Progenitor Cells for Ophthalmological Disorders - Drug Profile 95

RGX-321 - Drug Profile 97

RST-001 - Drug Profile 98

SAR-421869 - Drug Profile 99

SB-623 - Drug Profile 101

SHP-630 - Drug Profile 103

Small Molecule for Retinitis Pigmentosa - Drug Profile 104

Small Molecule to Agonize Neurotrophin Receptor for Retinitis Pigmentosa - Drug Profile 105

Small Molecules for CNS Disorders and Ophthalmology - Drug Profile 106

Small Molecules for Retinitis Pigmentosa - Drug Profile 107

Small Molecules for Retinitis Pigmentosa - Drug Profile 108

Small Molecules to Inhibit Ire1-Alpha for Retinitis Pigmentosa, Type 2 Diabetes and Amyotrophic Lateral Sclerosis - Drug Profile 109

SPKRPE-65 - Drug Profile 110

Stem Cell Therapy for Age Related Macular Degeneration and Retinitis Pigmentosa - Drug Profile 113

Stem Cell Therapy for AMD and Retinitis Pigmentosa - Drug Profile 115

Stem Cell Therapy for Ophthalmic Disorders - Drug Profile 116

Stem Cell Therapy to Activate MFRP for Retinitis Pigmentosa - Drug Profile 117

unoprostone isopropyl - Drug Profile 118

zuretinol acetate - Drug Profile 120

Retinitis Pigmentosa (Retinitis) - Recent Pipeline Updates 122

Retinitis Pigmentosa (Retinitis) - Dormant Projects 145

Retinitis Pigmentosa (Retinitis) - Discontinued Products 146

Retinitis Pigmentosa (Retinitis) - Product Development Milestones 147

Featured News & Press Releases 147

Appendix 156

Methodology 156

Coverage 156

Secondary Research 156

Primary Research 156

Expert Panel Validation 156

Contact Us 156

Disclaimer 157

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