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Genetic Ophthalmology Disorders Drug Development Pipeline Review, 2017

  • Published Date: 01 Aug 2017
  • Number of Pages: 138
  • Category: Healthcare and Medical
  • Country: Global
Genetic Ophthalmology Disorders Drug Development Pipeline Review, 2017

Summary

This report provides an overview of the pipeline landscape for genetic ophthalmological disorders. The report provides comprehensive information on the therapeutics under development and key players involved in therapeutic development for Stargardt disease, Leber congenital amaurosis, Lebers hereditary optic neuropathy, Usher Syndrome and Retinitis pigmentosa, and features dormant and discontinued projects.

Juvenile macular degeneration is a series of inherited eye disorders that affects children and young adults, with the most common form being Stargardt disease, an inherited autosomal recessive syndrome. Leber congenital amaurosis primarily affects the retina, which is the specialized tissue at the back of the eye that detects light and color. It is the most common cause of inherited blindness in childhood. Lebers hereditary optic neuropathy usually begins in a persons teens or twenties, rare cases may appear in early childhood or later in adulthood. Usher syndrome is characterized by hearing impairment and progressive vision loss. The major symptoms of Usher syndrome are hearing loss and secondary retinitis pigmentosa. Finally, retinitis pigmentosa refers to a group of diseases which cause a slow but progressive vision loss. Symptoms include night blindness and loss of peripheral vision.

The size of these pipelines ranges from six products in Usher syndrome to 54 in retinitis pigmentosa. Gene therapies represent the most common type of therapy among these diseases, followed by small molecules. This reflects the therapeutic aim of repairing the defective gene in order to correct the patients phenotype. Likewise, the molecular targets which are acted on are typically clustered around the causative gene within each disease, although there are exceptions. Within retinitis pigmentosa in particular, there is a diversity of molecular targets.

Scope

- Which companies are the most active within the pipeline for genetic ophthalmological disorder therapeutics?
- Which pharmaceutical approaches are the most prominent at each stage of the pipeline and within each indication?
- To what extent do universities and institutions play a role within this pipeline, compared to pharmaceutical companies?
- What are the most important R&D milestones and data publications to have happened in the field of genetic ophthalmological disorders?

Reasons to buy

- Understand the overall pipeline, with an at-a-glance overview of all products in therapeutic development for each indication
- Assess the products in development in granular detail, with an up-to-date overview of each individual pipeline program in each indication, and a comprehensive picture of recent updates and milestones for each
- Analyze the companies, institutions and universities currently operating in the pipeline, and the products being fielded by each of these
- Understand the composition of the pipeline in terms of molecule type, molecular target, mechanism of action and route of administration
Publisher Name : GBI Research

GBI Research Report Guidance 2
Executive Summary 3
Table of Contents 4
List of Tables 6
List of Figures 9
Introduction 11
Genetic Ophthalmology Disorders Report Coverage 11
Juvenile Macular Degeneration (Stargardt Disease) - Overview 11
Leber Congenital Amaurosis (LCA) - Overview 11
Lebers Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Overview 11
Usher Syndrome - Overview 11
Retinitis Pigmentosa (Retinitis) - Overview 11
Therapeutics Development 12
Juvenile Macular Degeneration (Stargardt Disease) 12
Leber Congenital Amaurosis (LCA) 16
Lebers Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 20
Usher Syndrome 23
Retinitis Pigmentosa (Retinitis) 26
Therapeutics Assessment 33
Juvenile Macular Degeneration (Stargardt Disease) 33
Leber Congenital Amaurosis (LCA) 41
Lebers Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 48
Usher Syndrome 55
Retinitis Pigmentosa (Retinitis) 62
Companies Involved in Therapeutics Development 71
Juvenile Macular Degeneration (Stargardt Disease) 71
Leber Congenital Amaurosis (LCA) 74
Lebers Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 77
Usher Syndrome 81
Retinitis Pigmentosa (Retinitis) 83
Dormant Projects 96
Juvenile Macular Degeneration (Stargardt Disease) 96
Leber Congenital Amaurosis (LCA) 96
Lebers Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 96
Usher Syndrome 97
Retinitis Pigmentosa (Retinitis) 97
Discontinued Products 99
Retinitis Pigmentosa (Retinitis) 99
Product Development Milestones 100
Juvenile Macular Degeneration (Stargardt Disease) 100
Leber Congenital Amaurosis (LCA) 108
Lebers Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 117
Usher Syndrome 123
Retinitis Pigmentosa (Retinitis) 124
Appendix 137
Methodology 137
Coverage 137
Secondary Research 137
Primary Research 137
Expert Panel Validation 137
Contact Us 137
Disclaimer 138

List Of Tables


Number of Products under Development for Juvenile Macular Degeneration (Stargardt Disease) 12
Number of Products under Development by Companies, Juvenile Macular Degeneration (Stargardt Disease) 13
Number of Products under Development by Universities/Institutes, Juvenile Macular Degeneration (Stargardt Disease) 14
Products under Development by Companies, Juvenile Macular Degeneration (Stargardt Disease) 14
Products under Development by Universities/Institutes, Juvenile Macular Degeneration (Stargardt Disease) 15
Number of Products under Development for Leber Congenital Amaurosis (LCA) 16
Number of Products under Development by Companies, Leber Congenital Amaurosis (LCA) 17
Number of Products under Development by Universities/Institutes, Leber Congenital Amaurosis (LCA) 18
Products under Development by Companies, Leber Congenital Amaurosis (LCA) 19
Products under Development by Universities/Institutes, Leber Congenital Amaurosis (LCA) 19
Number of Products under Development for Lebers Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 20
Number of Products under Development by Companies, Lebers Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 21
Number of Products under Development by Universities/Institutes, Lebers Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 22
Products under Development by Companies, Lebers Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 22
Products under Development by Universities/Institutes, Lebers Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 23
Number of Products under Development for Usher Syndrome 23
Number of Products under Development by Companies, Usher Syndrome 24
Number of Products under Development by Universities/Institutes, Usher Syndrome 25
Products under Development by Companies, Usher Syndrome 25
Products under Development by Universities/Institutes, Usher Syndrome 25
Number of Products under Development for Retinitis Pigmentosa (Retinitis) 26
Number of Products under Development by Companies, Retinitis Pigmentosa (Retinitis) 28
Number of Products under Development by Universities/Institutes, Retinitis Pigmentosa (Retinitis) 29
Products under Development by Companies, Retinitis Pigmentosa (Retinitis) 30
Products under Development by Universities/Institutes, Retinitis Pigmentosa (Retinitis) 32
Number of Products by Stage and Target, Juvenile Macular Degeneration (Stargardt Disease) 34
Number of Products by Stage and Mechanism of Action, Juvenile Macular Degeneration (Stargardt Disease) 36
Number of Products by Stage and Route of Administration, Juvenile Macular Degeneration (Stargardt Disease) 38
Number of Products by Stage and Molecule Type, Juvenile Macular Degeneration (Stargardt Disease) 40
Number of Products by Stage and Target, Leber Congenital Amaurosis (LCA) 42
Number of Products by Stage and Mechanism of Action, Leber Congenital Amaurosis (LCA) 44
Number of Products by Stage and Route of Administration, Leber Congenital Amaurosis (LCA) 46
Number of Products by Stage and Molecule Type, Leber Congenital Amaurosis (LCA) 48
Number of Products by Stage and Target, Lebers Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 49
Number of Products by Stage and Mechanism of Action, Lebers Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 51
Number of Products by Stage and Route of Administration, Lebers Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 53
Number of Products by Stage and Molecule Type, Lebers Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 55
Number of Products by Stage and Target, Usher Syndrome 56
Number of Products by Stage and Mechanism of Action, Usher Syndrome 58
Number of Products by Stage and Route of Administration, Usher Syndrome 60
Number of Products by Stage and Molecule Type, Usher Syndrome 62
Number of Products by Stage and Target, Retinitis Pigmentosa (Retinitis) 64
Number of Products by Stage and Mechanism of Action, Retinitis Pigmentosa (Retinitis) 66
Number of Products by Stage and Route of Administration, Retinitis Pigmentosa (Retinitis) 68
Number of Products by Stage and Molecule Type, Retinitis Pigmentosa (Retinitis) 70
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Acucela Inc 71
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Alkeus Pharmaceuticals Inc 71
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Astellas Pharma Inc 72
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Copernicus Therapeutics Inc 72
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Grupo Ferrer Internacional SA 73
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Iris Pharma 73
Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Sanofi 74
Leber Congenital Amaurosis (LCA) - Pipeline by AmpliPhi Biosciences Corp 74
Leber Congenital Amaurosis (LCA) - Pipeline by Editas Medicine Inc 75
Leber Congenital Amaurosis (LCA) - Pipeline by Novelion Therapeutics Inc 75
Leber Congenital Amaurosis (LCA) - Pipeline by ProQR Therapeutics NV 76
Leber Congenital Amaurosis (LCA) - Pipeline by Spark Therapeutics Inc 76
Lebers Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Pipeline by Alkeus Pharmaceuticals Inc 77
Lebers Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Pipeline by Biovista Inc 77
Lebers Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Pipeline by GenSight Biologics SA 78
Lebers Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Pipeline by Ixchel Pharma LLC 78
Lebers Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Pipeline by Khondrion BV 79
Lebers Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Pipeline by Mitotech SA 79
Lebers Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Pipeline by Spark Therapeutics Inc 80
Lebers Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Pipeline by Stealth BioTherapeutics Inc 80
Usher Syndrome - Pipeline by Amgen Inc 81
Usher Syndrome - Pipeline by Editas Medicine Inc 81
Usher Syndrome - Pipeline by ProQR Therapeutics NV 82
Usher Syndrome - Pipeline by Sanofi 82
Retinitis Pigmentosa (Retinitis) - Pipeline by Acucela Inc 83
Retinitis Pigmentosa (Retinitis) - Pipeline by Allergan Plc 83
Retinitis Pigmentosa (Retinitis) - Pipeline by Amarantus Bioscience Holdings Inc 84
Retinitis Pigmentosa (Retinitis) - Pipeline by Amgen Inc 84
Retinitis Pigmentosa (Retinitis) - Pipeline by Applied Genetic Technologies Corp 85
Retinitis Pigmentosa (Retinitis) - Pipeline by Asklepios BioPharmaceutical Inc 85
Retinitis Pigmentosa (Retinitis) - Pipeline by Astellas Pharma Inc 86
Retinitis Pigmentosa (Retinitis) - Pipeline by Caladrius Biosciences Inc 86
Retinitis Pigmentosa (Retinitis) - Pipeline by Dompe Farmaceutici SpA 87
Retinitis Pigmentosa (Retinitis) - Pipeline by GenSight Biologics SA 87
Retinitis Pigmentosa (Retinitis) - Pipeline by Grupo Ferrer Internacional SA 88
Retinitis Pigmentosa (Retinitis) - Pipeline by ID Pharma Co Ltd 88
Retinitis Pigmentosa (Retinitis) - Pipeline by InFlectis BioScience 89
Retinitis Pigmentosa (Retinitis) - Pipeline by Ionis Pharmaceuticals Inc 89
Retinitis Pigmentosa (Retinitis) - Pipeline by Ms Science Corp 90
Retinitis Pigmentosa (Retinitis) - Pipeline by Mimetogen Pharmaceuticals Inc 90
Retinitis Pigmentosa (Retinitis) - Pipeline by Nanovector srl 91
Retinitis Pigmentosa (Retinitis) - Pipeline by Novartis AG 91
Retinitis Pigmentosa (Retinitis) - Pipeline by Novelion Therapeutics Inc 92
Retinitis Pigmentosa (Retinitis) - Pipeline by ProQR Therapeutics NV 92
Retinitis Pigmentosa (Retinitis) - Pipeline by ReNeuron Group Plc 93
Retinitis Pigmentosa (Retinitis) - Pipeline by SanBio Inc 93
Retinitis Pigmentosa (Retinitis) - Pipeline by Sanofi 94
Retinitis Pigmentosa (Retinitis) - Pipeline by Shire Plc 94
Retinitis Pigmentosa (Retinitis) - Pipeline by Spark Therapeutics Inc 95
Retinitis Pigmentosa (Retinitis) - Pipeline by Sun Pharma Advanced Research Company Ltd 95
Juvenile Macular Degeneration (Stargardt Disease) - Dormant Projects 96
Leber Congenital Amaurosis (LCA) - Dormant Projects 96
Lebers Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Dormant Projects 96
Usher Syndrome - Dormant Projects 97
Retinitis Pigmentosa (Retinitis) - Dormant Projects 97
Retinitis Pigmentosa (Retinitis) - Discontinued Products 99

List Of Figures


Number of Products under Development for Juvenile Macular Degeneration (Stargardt Disease) 12
Number of Products under Development by Companies, Juvenile Macular Degeneration (Stargardt Disease) 13
Number of Products under Development for Leber Congenital Amaurosis (LCA) 16
Number of Products under Development by Companies, Leber Congenital Amaurosis (LCA) 17
Number of Products under Development by Universities/Institutes, Leber Congenital Amaurosis (LCA) 18
Number of Products under Development for Lebers Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 20
Number of Products under Development by Companies, Lebers Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 21
Number of Products under Development for Usher Syndrome 23
Number of Products under Development by Companies, Usher Syndrome 24
Number of Products under Development for Retinitis Pigmentosa (Retinitis) 26
Number of Products under Development by Companies, Retinitis Pigmentosa (Retinitis) 27
Number of Products under Development by Universities/Institutes, Retinitis Pigmentosa (Retinitis) 29
Number of Products by Targets, Juvenile Macular Degeneration (Stargardt Disease) 33
Number of Products by Stage and Targets, Juvenile Macular Degeneration (Stargardt Disease) 33
Number of Products by Mechanism of Actions, Juvenile Macular Degeneration (Stargardt Disease) 35
Number of Products by Stage and Mechanism of Actions, Juvenile Macular Degeneration (Stargardt Disease) 35
Number of Products by Routes of Administration, Juvenile Macular Degeneration (Stargardt Disease) 37
Number of Products by Stage and Routes of Administration, Juvenile Macular Degeneration (Stargardt Disease) 37
Number of Products by Molecule Types, Juvenile Macular Degeneration (Stargardt Disease) 39
Number of Products by Stage and Molecule Types, Juvenile Macular Degeneration (Stargardt Disease) 39
Number of Products by Targets, Leber Congenital Amaurosis (LCA) 41
Number of Products by Stage and Targets, Leber Congenital Amaurosis (LCA) 41
Number of Products by Mechanism of Actions, Leber Congenital Amaurosis (LCA) 43
Number of Products by Stage and Mechanism of Actions, Leber Congenital Amaurosis (LCA) 43
Number of Products by Routes of Administration, Leber Congenital Amaurosis (LCA) 45
Number of Products by Stage and Routes of Administration, Leber Congenital Amaurosis (LCA) 45
Number of Products by Molecule Types, Leber Congenital Amaurosis (LCA) 47
Number of Products by Stage and Molecule Types, Leber Congenital Amaurosis (LCA) 47
Number of Products by Targets, Lebers Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 48
Number of Products by Stage and Targets, Lebers Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 49
Number of Products by Mechanism of Actions, Lebers Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 50
Number of Products by Stage and Mechanism of Actions, Lebers Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 50
Number of Products by Routes of Administration, Lebers Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 52
Number of Products by Stage and Routes of Administration, Lebers Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 52
Number of Products by Molecule Types, Lebers Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 54
Number of Products by Stage and Molecule Types, Lebers Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 54
Number of Products by Targets, Usher Syndrome 55
Number of Products by Stage and Targets, Usher Syndrome 56
Number of Products by Mechanism of Actions, Usher Syndrome 57
Number of Products by Stage and Mechanism of Actions, Usher Syndrome 57
Number of Products by Routes of Administration, Usher Syndrome 59
Number of Products by Stage and Routes of Administration, Usher Syndrome 59
Number of Products by Molecule Types, Usher Syndrome 61
Number of Products by Stage and Molecule Types, Usher Syndrome 61
Number of Products by Top 10 Targets, Retinitis Pigmentosa (Retinitis) 62
Number of Products by Stage and Top 10 Targets, Retinitis Pigmentosa (Retinitis) 63
Number of Products by Top 10 Mechanism of Actions, Retinitis Pigmentosa (Retinitis) 65
Number of Products by Stage and Top 10 Mechanism of Actions, Retinitis Pigmentosa (Retinitis) 65
Number of Products by Routes of Administration, Retinitis Pigmentosa (Retinitis) 67
Number of Products by Stage and Routes of Administration, Retinitis Pigmentosa (Retinitis) 67
Number of Products by Molecule Types, Retinitis Pigmentosa (Retinitis) 69
Number of Products by Stage and Molecule Types, Retinitis Pigmentosa (Retinitis) 69

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