Retinitis Pigmentosa (Retinitis) - Pipeline Review, H1 2015

Published: Jun, 2015 | Pages: 143 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

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