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Retinitis Pigmentosa (Retinitis)-Pipeline Review, H1 2015

Retinitis Pigmentosa (Retinitis)-Pipeline Review, H1 2015


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Executive Summary

Retinitis Pigmentosa (Retinitis)-Pipeline Review, H1 2015

Summary

Global Markets Direct's, 'Retinitis Pigmentosa (Retinitis)-Pipeline Review, H1 2015', provides an overview of the Retinitis Pigmentosa (Retinitis)'s therapeutic pipeline.

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 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 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

Table of Contents

Table of Contents 2

List of Tables 8

List of Figures 9

Introduction 10

Global Markets Direct Report Coverage 10

Retinitis Pigmentosa (Retinitis) Overview 11

Therapeutics Development 12

Pipeline Products for Retinitis Pigmentosa (Retinitis)-Overview 12

Pipeline Products for Retinitis Pigmentosa (Retinitis)-Comparative Analysis 13

Retinitis Pigmentosa (Retinitis)-Therapeutics under Development by Companies 14

Retinitis Pigmentosa (Retinitis)-Therapeutics under Investigation by Universities/Institutes 17

Retinitis Pigmentosa (Retinitis)-Pipeline Products Glance 19

Late Stage Products 19

Clinical Stage Products 20

Early Stage Products 21

Retinitis Pigmentosa (Retinitis)-Products under Development by Companies 22

Retinitis Pigmentosa (Retinitis)-Products under Investigation by Universities/Institutes 24

Retinitis Pigmentosa (Retinitis)-Companies Involved in Therapeutics Development 25

Acucela Inc. 25

Amarantus Bioscience Holdings, Inc. 26

Applied Genetic Technologies Corporation 27

Asklepios BioPharmaceutical, Inc. 28

BioDiem Ltd 29

Caladrius Biosciences, Inc. 30

DNAVEC Corporation 31

Dompe Farmaceutici S.p.A. 32

Genable Technologies Limited 33

GenSight Biologics SA 34

Grupo Ferrer Internacional, S.A. 35

International Stem Cell Corporation 36

Isis Pharmaceuticals, Inc. 37

Mimetogen Pharmaceuticals Inc. 38

Neurotech Pharmaceuticals, Inc. 39

Ocata Therapeutics, Inc. 40

Orphagen Pharmaceuticals, Inc. 41

QLT Inc. 42

R-Tech Ueno, Ltd. 43

ReNeuron Group Plc 44

Sanofi 45

Shire Plc 46

Spark Therapeutics, Inc. 47

Sun Pharma Advanced Research Company Ltd. 48

Retinitis Pigmentosa (Retinitis)-Therapeutics Assessment 49

Assessment by Monotherapy Products 49

Assessment by Target 50

Assessment by Mechanism of Action 52

Assessment by Route of Administration 54

Assessment by Molecule Type 56

Drug Profiles 58

AMRS-001-Drug Profile 58

Product Description 58

Mechanism of Action 58

R&D Progress 58

Antibodies to Antagonize IL-6 for Retinitis-Drug Profile 60

Product Description 60

Mechanism of Action 60

R&D Progress 60

BDM-E-Drug Profile 61

Product Description 61

Mechanism of Action 61

R&D Progress 61

BNP-RP-Drug Profile 63

Product Description 63

Mechanism of Action 63

R&D Progress 63

CB-11-Drug Profile 64

Product Description 64

Mechanism of Action 64

R&D Progress 64

ECL-1-Drug Profile 65

Product Description 65

Mechanism of Action 65

R&D Progress 65

emixustat hydrochloride-Drug Profile 66

Product Description 66

Mechanism of Action 66

R&D Progress 66

FIB-111-Drug Profile 68

Product Description 68

Mechanism of Action 68

R&D Progress 68

Gene Therapy for Glaucoma and Retinitis Pigmentosa-Drug Profile 69

Product Description 69

Mechanism of Action 69

R&D Progress 69

Gene Therapy for Ocular Diseases-Drug Profile 70

Product Description 70

Mechanism of Action 70

R&D Progress 70

Gene Therapy for Ophthalmology-Drug Profile 71

Product Description 71

Mechanism of Action 71

R&D Progress 71

Gene Therapy for Retinitis Pigmentosa-Drug Profile 72

Product Description 72

Mechanism of Action 72

R&D Progress 72

Gene Therapy to Activate HDAC4 for Retinitis Pigmentosa-Drug Profile 73

Product Description 73

Mechanism of Action 73

R&D Progress 73

Gene Therapy to Activate RPGR for X-Linked Retinitis Pigmentosa-Drug Profile 74

Product Description 74

Mechanism of Action 74

R&D Progress 74

GS-020-Drug Profile 75

Product Description 75

Mechanism of Action 75

R&D Progress 75

GT-038-Drug Profile 76

Product Description 76

Mechanism of Action 76

R&D Progress 76

ISISRHO-2.5Rx-Drug Profile 78

Product Description 78

Mechanism of Action 78

R&D Progress 78

KIRA-6-Drug Profile 79

Product Description 79

Mechanism of Action 79

R&D Progress 79

NT-501-Drug Profile 80

Product Description 80

Mechanism of Action 80

R&D Progress 80

OCU-100-Drug Profile 83

Product Description 83

Mechanism of Action 83

R&D Progress 83

PRO-015-Drug Profile 84

Product Description 84

Mechanism of Action 84

R&D Progress 84

proinsulin human SR-Drug Profile 85

Product Description 85

Mechanism of Action 85

R&D Progress 85

QLT-091001-Drug Profile 86

Product Description 86

Mechanism of Action 86

R&D Progress 86

Recombinant Human Nerve Growth Factor-Drug Profile 88

Product Description 88

Mechanism of Action 88

R&D Progress 88

ReN-003-Drug Profile 89

Product Description 89

Mechanism of Action 89

R&D Progress 89

Retinal Progenitor Cells for Ophthalmological Disorders-Drug Profile 91

Product Description 91

Mechanism of Action 91

R&D Progress 91

RGX-321-Drug Profile 93

Product Description 93

Mechanism of Action 93

R&D Progress 93

RST-001-Drug Profile 94

Product Description 94

Mechanism of Action 94

R&D Progress 94

SAR-421869-Drug Profile 95

Product Description 95

Mechanism of Action 95

R&D Progress 95

SHP-630-Drug Profile 96

Product Description 96

Mechanism of Action 96

R&D Progress 96

Small Molecule for Retinitis Pigmentosa-Drug Profile 97

Product Description 97

Mechanism of Action 97

R&D Progress 97

Small Molecule to Agonize Neurotrophin Receptor for Retinitis Pigmentosa-Drug Profile 98

Product Description 98

Mechanism of Action 98

R&D Progress 98

Small Molecules for CNS Disorders and Ophthalmology-Drug Profile 99

Product Description 99

Mechanism of Action 99

R&D Progress 99

Small Molecules for Retinitis Pigmentosa-Drug Profile 100

Product Description 100

Mechanism of Action 100

R&D Progress 100

Small Molecules for Retinitis Pigmentosa-Drug Profile 101

Product Description 101

Mechanism of Action 101

R&D Progress 101

Small Molecules to Inhibit Ire1-Alpha for Retinitis Pigmentosa, Type 2 Diabetes and Amyotrophic Lateral Sclerosis-Drug Profile 102

Product Description 102

Mechanism of Action 102

R&D Progress 102

SPK-RPE-65-Drug Profile 103

Product Description 103

Mechanism of Action 103

R&D Progress 103

Stem Cell Therapy for Age Related Macular Degeneration and Retinitis Pigmentosa-Drug Profile 105

Product Description 105

Mechanism of Action 105

R&D Progress 105

Stem Cell Therapy for AMD and Retinitis Pigmentosa-Drug Profile 107

Product Description 107

Mechanism of Action 107

R&D Progress 107

Stem Cell Therapy for Ophthalmic Disorders-Drug Profile 108

Product Description 108

Mechanism of Action 108

R&D Progress 108

Stem Cell Therapy to Activate MFRP for Retinitis Pigmentosa-Drug Profile 109

Product Description 109

Mechanism of Action 109

R&D Progress 109

unoprostone isopropyl-Drug Profile 110

Product Description 110

Mechanism of Action 110

R&D Progress 110

Retinitis Pigmentosa (Retinitis)-Recent Pipeline Updates 112

Retinitis Pigmentosa (Retinitis)-Dormant Projects 132

Retinitis Pigmentosa (Retinitis)-Discontinued Products 133

Retinitis Pigmentosa (Retinitis)-Product Development Milestones 134

Featured News & Press Releases 134

May 14, 2015: Amarantus Enters Into cGMP Manufacturing Agreement With Catalent Biologics for Production of MANF for Human Clinical Studies 134

May 08, 2015: Presentations of two studies on Unoprostone (development code UF-021) for retinitis pigmentosa announced at The Association for Research in Vision and Ophthalmology 2015 134

May 07, 2015: Amarantus Announces the Presentation of Data Showing MANF's Positive Effect on Retinal Function at Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting 135

May 05, 2015: Amarantus Receives Notice of Allowance for U.S. Patent Application Covering Proprietary Compositions of Matter and Methods of Use for MANF 136

Apr 29, 2015: Amarantus Granted European Union Orphan Drug Designation for MANF for the Treatment of Retinitis Pigmentosa 137

Apr 09, 2015: Neostem Announces Extension Of Study Under Grant From California Institute Of Regenerative Medicine To Fund Research Of Retinal Disease 138

Mar 09, 2015: The end of a Phase 3 Clinical Study and Preliminary Findings from a Phase 3 of Unoprostone for treatment of Retinitis Pigmentosa 138

Feb 02, 2015: OptiKira, a New Biotech Start-up, Created to Develop Drugs to Prevent Cell Death Caused by the Unfolded Protein Response 139

Jan 12, 2015: RetroSense Therapeutics to Present at 7th Annual Biotech Showcase Conference 140

Dec 23, 2014: Amarantus Receives Orphan Drug Designation for MANF From U.S. Food and Drug Administration for Treatment of Retinitis Pigmentosa 140

Appendix 141

Methodology 141

Coverage 141

Secondary Research 141

Primary Research 141

Expert Panel Validation 141

Contact Us 141

Disclaimer 142

List of Tables

Number of Products under Development for Retinitis Pigmentosa (Retinitis), H1 2015 12

Number of Products under Development for Retinitis Pigmentosa (Retinitis)-Comparative Analysis, H1 2015 13

Number of Products under Development by Companies, H1 2015 15

Number of Products under Development by Companies, H1 2015 (Contd..1) 16

Number of Products under Investigation by Universities/Institutes, H1 2015 17

Comparative Analysis by Late Stage Development, H1 2015 19

Comparative Analysis by Clinical Stage Development, H1 2015 20

Comparative Analysis by Early Stage Development, H1 2015 21

Products under Development by Companies, H1 2015 22

Products under Development by Companies, H1 2015 (Contd..1) 23

Products under Investigation by Universities/Institutes, H1 2015 24

Retinitis Pigmentosa (Retinitis)-Pipeline by Acucela Inc., H1 2015 25

Retinitis Pigmentosa (Retinitis)-Pipeline by Amarantus Bioscience Holdings, Inc., H1 2015 26

Retinitis Pigmentosa (Retinitis)-Pipeline by Applied Genetic Technologies Corporation, H1 2015 27

Retinitis Pigmentosa (Retinitis)-Pipeline by Asklepios BioPharmaceutical, Inc., H1 2015 28

Retinitis Pigmentosa (Retinitis)-Pipeline by BioDiem Ltd, H1 2015 29

Retinitis Pigmentosa (Retinitis)-Pipeline by Caladrius Biosciences, Inc. , H1 2015 30

Retinitis Pigmentosa (Retinitis)-Pipeline by DNAVEC Corporation, H1 2015 31

Retinitis Pigmentosa (Retinitis)-Pipeline by Dompe Farmaceutici S.p.A., H1 2015 32

Retinitis Pigmentosa (Retinitis)-Pipeline by Genable Technologies Limited, H1 2015 33

Retinitis Pigmentosa (Retinitis)-Pipeline by GenSight Biologics SA, H1 2015 34

Retinitis Pigmentosa (Retinitis)-Pipeline by Grupo Ferrer Internacional, S.A., H1 2015 35

Retinitis Pigmentosa (Retinitis)-Pipeline by International Stem Cell Corporation, H1 2015 36

Retinitis Pigmentosa (Retinitis)-Pipeline by Isis Pharmaceuticals, Inc., H1 2015 37

Retinitis Pigmentosa (Retinitis)-Pipeline by Mimetogen Pharmaceuticals Inc., H1 2015 38

Retinitis Pigmentosa (Retinitis)-Pipeline by Neurotech Pharmaceuticals, Inc., H1 2015 39

Retinitis Pigmentosa (Retinitis)-Pipeline by Ocata Therapeutics, Inc., H1 2015 40

Retinitis Pigmentosa (Retinitis)-Pipeline by Orphagen Pharmaceuticals, Inc., H1 2015 41

Retinitis Pigmentosa (Retinitis)-Pipeline by QLT Inc., H1 2015 42

Retinitis Pigmentosa (Retinitis)-Pipeline by R-Tech Ueno, Ltd., H1 2015 43

Retinitis Pigmentosa (Retinitis)-Pipeline by ReNeuron Group Plc, H1 2015 44

Retinitis Pigmentosa (Retinitis)-Pipeline by Sanofi, H1 2015 45

Retinitis Pigmentosa (Retinitis)-Pipeline by Shire Plc, H1 2015 46

Retinitis Pigmentosa (Retinitis)-Pipeline by Spark Therapeutics, Inc., H1 2015 47

Retinitis Pigmentosa (Retinitis)-Pipeline by Sun Pharma Advanced Research Company Ltd., H1 2015 48

Assessment by Monotherapy Products, H1 2015 49

Number of Products by Stage and Target, H1 2015 51

Number of Products by Stage and Mechanism of Action, H1 2015 53

Number of Products by Stage and Route of Administration, H1 2015 55

Number of Products by Stage and Molecule Type, H1 2015 57

Retinitis Pigmentosa (Retinitis) Therapeutics-Recent Pipeline Updates, H1 2015 112

Retinitis Pigmentosa (Retinitis)-Dormant Projects, H1 2015 132

Retinitis Pigmentosa (Retinitis)-Discontinued Products, H1 2015 133

List of Figures

Number of Products under Development for Retinitis Pigmentosa (Retinitis), H1 2015 12

Number of Products under Development for Retinitis Pigmentosa (Retinitis)-Comparative Analysis, H1 2015 13

Number of Products under Development by Companies, H1 2015 14

Number of Products under Investigation by Universities/Institutes, H1 2015 17

Comparative Analysis by Clinical Stage Development, H1 2015 20

Comparative Analysis by Early Stage Products, H1 2015 21

Assessment by Monotherapy Products, H1 2015 49

Number of Products by Top 10 Targets, H1 2015 50

Number of Products by Stage and Top 10 Targets, H1 2015 50

Number of Products by Top 10 Mechanism of Actions, H1 2015 52

Number of Products by Stage and Top 10 Mechanism of Actions, H1 2015 52

Number of Products by Top 10 Routes of Administration, H1 2015 54

Number of Products by Stage and Top 10 Routes of Administration, H1 2015 54

Number of Products by Top 10 Molecule Types, H1 2015 56

Number of Products by Stage and Top 10 Molecule Types, H1 2015 56

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Acucela Inc.

Amarantus Bioscience Holdings, Inc.

Applied Genetic Technologies Corporation

Asklepios BioPharmaceutical, Inc.

BioDiem Ltd

Caladrius Biosciences, Inc.

DNAVEC Corporation

Dompe Farmaceutici S.p.A.

Genable Technologies Limited

GenSight Biologics SA

Grupo Ferrer Internacional, S.A.

International Stem Cell Corporation

Isis Pharmaceuticals, Inc.

Mimetogen Pharmaceuticals Inc.

Neurotech Pharmaceuticals, Inc.

Ocata Therapeutics, Inc.

Orphagen Pharmaceuticals, Inc.

QLT Inc.

R-Tech Ueno, Ltd.

ReNeuron Group Plc

Sanofi

Shire Plc

Spark Therapeutics, Inc.

Sun Pharma Advanced Research Company Ltd.

Retinitis Pigmentosa (Retinitis) Therapeutic Products under Development, Key Players in Retinitis Pigmentosa (Retinitis) Therapeutics, Retinitis Pigmentosa (Retinitis) Pipeline Overview, Retinitis Pigmentosa (Retinitis) Pipeline, Retinitis Pigmentosa (Retinitis) Pipeline Assessment


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