Retinitis Pigmentosa (Retinitis) - Pipeline Review, H2 2014

Published: Nov, 2014 | Pages: 127 | Publisher: Global Markets Direct
Industry: Healthcare | Report Format: Electronic (PDF)

Retinitis Pigmentosa (Retinitis) - Pipeline Review, H2 2014

Summary

Global Markets Direct's, 'Retinitis Pigmentosa (Retinitis) - Pipeline Review, H2 2014', 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 7
List of Figures 8
Introduction 9
Global Markets Direct Report Coverage 9
Retinitis Pigmentosa (Retinitis) Overview 10
Therapeutics Development 11
Pipeline Products for Retinitis Pigmentosa (Retinitis) - Overview 11
Pipeline Products for Retinitis Pigmentosa (Retinitis) - Comparative Analysis 12
Retinitis Pigmentosa (Retinitis) - Therapeutics under Development by Companies 13
Retinitis Pigmentosa (Retinitis) - Therapeutics under Investigation by Universities/Institutes 15
Retinitis Pigmentosa (Retinitis) - Pipeline Products Glance 16
Late Stage Products 16
Clinical Stage Products 17
Early Stage Products 18
Retinitis Pigmentosa (Retinitis) - Products under Development by Companies 19
Retinitis Pigmentosa (Retinitis) - Products under Investigation by Universities/Institutes 21
Retinitis Pigmentosa (Retinitis) - Companies Involved in Therapeutics Development 22
Acucela Inc. 22
Amarantus Bioscience Holdings, Inc. 23
Applied Genetic Technologies Corporation 24
Asklepios BioPharmaceutical, Inc. 25
BBB Therapeutics B.V. 26
BioDiem Ltd 27
DNAVEC Corporation 28
Dompe Farmaceutici S.p.A. 29
Genable Technologies Limited 30
GenSight Biologics 31
Grupo Ferrer Internacional, S.A. 32
International Stem Cell Corporation 33
Mimetogen Pharmaceuticals Inc. 34
NeoStem, Inc. 35
Neurotech Pharmaceuticals, Inc. 36
Ocata Therapeutics, Inc. 37
Orphagen Pharmaceuticals, Inc. 38
QLT Inc. 39
R-Tech Ueno, Ltd. 40
ReGenX Biosciences, LLC 41
ReNeuron Group plc 42
Sanofi 43
Shire Plc 44
Retinitis Pigmentosa (Retinitis) - Therapeutics Assessment 45
Assessment by Monotherapy Products 45
Assessment by Target 46
Assessment by Mechanism of Action 48
Assessment by Route of Administration 50
Assessment by Molecule Type 52
Drug Profiles 54
AMRS-001 - Drug Profile 54
Product Description 54
Mechanism of Action 54
R&D Progress 54
BDM-E - Drug Profile 56
Product Description 56
Mechanism of Action 56
R&D Progress 56
BNP-RP - Drug Profile 58
Product Description 58
Mechanism of Action 58
R&D Progress 58
CB-11 - Drug Profile 59
Product Description 59
Mechanism of Action 59
R&D Progress 59
emixustat hydrochloride - Drug Profile 60
Product Description 60
Mechanism of Action 60
R&D Progress 60
FIB-111 - Drug Profile 62
Product Description 62
Mechanism of Action 62
R&D Progress 62
Gene Therapy for Ocular Diseases - Drug Profile 63
Product Description 63
Mechanism of Action 63
R&D Progress 63
Gene Therapy for Ophthalmology - Drug Profile 64
Product Description 64
Mechanism of Action 64
R&D Progress 64
Gene Therapy for Retinitis Pigmentosa - Drug Profile 65
Product Description 65
Mechanism of Action 65
R&D Progress 65
Gene Therapy for Retinitis Pigmentosa - Drug Profile 66
Product Description 66
Mechanism of Action 66
R&D Progress 66
Gene Therapy for Retinitis Pigmentosa and Glaucoma - Drug Profile 67
Product Description 67
Mechanism of Action 67
R&D Progress 67
Gene Therapy to Activate HDAC4 for Retinitis Pigmentosa - Drug Profile 68
Product Description 68
Mechanism of Action 68
R&D Progress 68
Gene Therapy to Activate RPGR for X-Linked Retinitis Pigmentosa - Drug Profile 69
Product Description 69
Mechanism of Action 69
R&D Progress 69
Gene Therapy to Activate RPGR for X-Linked Retinitis Pigmentosa - Drug Profile 70
Product Description 70
Mechanism of Action 70
R&D Progress 70
GT-038 - Drug Profile 71
Product Description 71
Mechanism of Action 71
R&D Progress 71
KIRA-6 - Drug Profile 72
Product Description 72
Mechanism of Action 72
R&D Progress 72
NT-501 - Drug Profile 73
Product Description 73
Mechanism of Action 73
R&D Progress 73
PRO-015 - Drug Profile 75
Product Description 75
Mechanism of Action 75
R&D Progress 75
proinsulin human SR - Drug Profile 76
Product Description 76
Mechanism of Action 76
R&D Progress 76
QLT-091001 - Drug Profile 77
Product Description 77
Mechanism of Action 77
R&D Progress 77
Recombinant Human Nerve Growth Factor - Drug Profile 79
Product Description 79
Mechanism of Action 79
R&D Progress 79
ReN-003 - Drug Profile 80
Product Description 80
Mechanism of Action 80
R&D Progress 80
RST-001 - Drug Profile 82
Product Description 82
Mechanism of Action 82
R&D Progress 82
SHP-630 - Drug Profile 83
Product Description 83
Mechanism of Action 83
R&D Progress 83
Small Molecule 5 for Retinitis Pigmentosa - Drug Profile 84
Product Description 84
Mechanism of Action 84
R&D Progress 84
Small Molecule 9 for Retinitis Pigmentosa - Drug Profile 85
Product Description 85
Mechanism of Action 85
R&D Progress 85
Small Molecule for Retinitis Pigmentosa - Drug Profile 86
Product Description 86
Mechanism of Action 86
R&D Progress 86
Small Molecule to Agonize Neurotrophin Receptor for Retinitis Pigmentosa - Drug Profile 87
Product Description 87
Mechanism of Action 87
R&D Progress 87
Small Molecules for CNS Disorders and Ophthalmology - Drug Profile 88
Product Description 88
Mechanism of Action 88
R&D Progress 88
Small Molecules for Inherited Retinal Diseases - Drug Profile 89
Product Description 89
Mechanism of Action 89
R&D Progress 89
Small Molecules to Modulate Nuclear Receptors for Retinitis Pigmentosa - Drug Profile 90
Product Description 90
Mechanism of Action 90
R&D Progress 90
Stem Cell Therapy for Age Related Macular Degeneration and Retinitis Pigmentosa - Drug Profile 91
Product Description 91
Mechanism of Action 91
R&D Progress 91
Stem Cell Therapy for AMD and Retinitis Pigmentosa - Drug Profile 93
Product Description 93
Mechanism of Action 93
R&D Progress 93
Stem Cell Therapy for Ophthalmic Disorders - Drug Profile 94
Product Description 94
Mechanism of Action 94
R&D Progress 94
Stem Cell Therapy for Ophthalmological Disorders - Drug Profile 95
Product Description 95
Mechanism of Action 95
R&D Progress 95
Stem Cell Therapy for Retinitis Pigmentosa - Drug Profile 96
Product Description 96
Mechanism of Action 96
R&D Progress 96
Stem Cell Therapy to Activate MFRP for Retinitis Pigmentosa - Drug Profile 97
Product Description 97
Mechanism of Action 97
R&D Progress 97
unoprostone isopropyl - Drug Profile 98
Product Description 98
Mechanism of Action 98
R&D Progress 98
UshStat - Drug Profile 100
Product Description 100
Mechanism of Action 100
R&D Progress 100
Retinitis Pigmentosa (Retinitis) - Recent Pipeline Updates 101
Retinitis Pigmentosa (Retinitis) - Dormant Projects 118
Retinitis Pigmentosa (Retinitis) - Discontinued Products 119
Retinitis Pigmentosa (Retinitis) - Product Development Milestones 120
Featured News & Press Releases 120
Oct 30, 2014: RetroSense Therapeutics Granted Orphan Drug Designation for Lead Product RST-001 for Retinitis Pigmentosa 120
Oct 16, 2014: Amarantus Applies to FDA for MANF Orphan Drug Designation in Retinitis Pigmentosa 120
Sep 10, 2014: Amarantus to Present Orphan Ocular Data at Targeting Ocular Disorders Conference 2014 121
Sep 09, 2014: Amarantus Announces Positive MANF Ocular Toxicology Data 121
Jul 02, 2014: Amarantus Announces Positive Interim Toxicology Data for MANF in Retinitis Pigmentosa 122
Apr 25, 2014: Sucampo Pharma To Present Preclinical Data For Unoprostone Isopropyl At Experimental Biology Conference 2014 122
Apr 03, 2014: First Patient is Enrolled in Lumos Study. Objective: to Evaluate Safety and Potential Efficacy of rhNGF in the Treatment of Retinitis Pigmentosa 123
Mar 11, 2014: ReNeuron to receive support from Foundation Fighting Blindness for its retinal stem cell therapy candidate for treatment of retinitis pigmentosa 123
Feb 27, 2014: QLT Announces Positive Preliminary Results From Phase 1b Retreatment Trial of QLT091001 in Subjects With Leber Congenital Amaurosis and Retinitis Pigmentosa Due to Mutations in LRAT or RPE65 124
Feb 06, 2014: California Stem Cell Partners on CIRM Grant to Develop Transplantable 3D Retinas 125
Appendix 126
Methodology 126
Coverage 126
Secondary Research 126
Primary Research 126
Expert Panel Validation 126
Contact Us 127
Disclaimer 127

List of Tables
Number of Products under Development for Retinitis Pigmentosa (Retinitis), H2 2014 11
Number of Products under Development for Retinitis Pigmentosa (Retinitis) - Comparative Analysis, H2 2014 12
Number of Products under Development by Companies, H2 2014 13
Number of Products under Investigation by Universities/Institutes, H2 2014 15
Comparative Analysis by Late Stage Development, H2 2014 16
Comparative Analysis by Clinical Stage Development, H2 2014 17
Comparative Analysis by Early Stage Development, H2 2014 18
Products under Development by Companies, H2 2014 19
Products under Investigation by Universities/Institutes, H2 2014 21
Retinitis Pigmentosa (Retinitis) - Pipeline by Acucela Inc., H2 2014 22
Retinitis Pigmentosa (Retinitis) - Pipeline by Amarantus Bioscience Holdings, Inc., H2 2014 23
Retinitis Pigmentosa (Retinitis) - Pipeline by Applied Genetic Technologies Corporation, H2 2014 24
Retinitis Pigmentosa (Retinitis) - Pipeline by Asklepios BioPharmaceutical, Inc., H2 2014 25
Retinitis Pigmentosa (Retinitis) - Pipeline by BBB Therapeutics B.V., H2 2014 26
Retinitis Pigmentosa (Retinitis) - Pipeline by BioDiem Ltd, H2 2014 27
Retinitis Pigmentosa (Retinitis) - Pipeline by DNAVEC Corporation, H2 2014 28
Retinitis Pigmentosa (Retinitis) - Pipeline by Dompe Farmaceutici S.p.A., H2 2014 29
Retinitis Pigmentosa (Retinitis) - Pipeline by Genable Technologies Limited, H2 2014 30
Retinitis Pigmentosa (Retinitis) - Pipeline by GenSight Biologics, H2 2014 31
Retinitis Pigmentosa (Retinitis) - Pipeline by Grupo Ferrer Internacional, S.A., H2 2014 32
Retinitis Pigmentosa (Retinitis) - Pipeline by International Stem Cell Corporation, H2 2014 33
Retinitis Pigmentosa (Retinitis) - Pipeline by Mimetogen Pharmaceuticals Inc., H2 2014 34
Retinitis Pigmentosa (Retinitis) - Pipeline by NeoStem, Inc., H2 2014 35
Retinitis Pigmentosa (Retinitis) - Pipeline by Neurotech Pharmaceuticals, Inc., H2 2014 36
Retinitis Pigmentosa (Retinitis) - Pipeline by Ocata Therapeutics, Inc., H2 2014 37
Retinitis Pigmentosa (Retinitis) - Pipeline by Orphagen Pharmaceuticals, Inc., H2 2014 38
Retinitis Pigmentosa (Retinitis) - Pipeline by QLT Inc., H2 2014 39
Retinitis Pigmentosa (Retinitis) - Pipeline by R-Tech Ueno, Ltd., H2 2014 40
Retinitis Pigmentosa (Retinitis) - Pipeline by ReGenX Biosciences, LLC, H2 2014 41
Retinitis Pigmentosa (Retinitis) - Pipeline by ReNeuron Group plc, H2 2014 42
Retinitis Pigmentosa (Retinitis) - Pipeline by Sanofi, H2 2014 43
Retinitis Pigmentosa (Retinitis) - Pipeline by Shire Plc, H2 2014 44
Assessment by Monotherapy Products, H2 2014 45
Number of Products by Stage and Target, H2 2014 47
Number of Products by Stage and Mechanism of Action, H2 2014 49
Number of Products by Stage and Route of Administration, H2 2014 51
Number of Products by Stage and Molecule Type, H2 2014 53
Retinitis Pigmentosa (Retinitis) Therapeutics - Recent Pipeline Updates, H2 2014 101
Retinitis Pigmentosa (Retinitis) - Dormant Projects, H2 2014 118
Retinitis Pigmentosa (Retinitis) - Discontinued Products, H2 2014 119