Genetic Ophthalmology Disorders Drug Development Pipeline Review, 2017

Genetic Ophthalmology Disorders Drug Development Pipeline Review, 2017


  • Products Id :- GBIHC015IDB
  • |
  • Pages: 138
  • |
  • |
  • |
  Request for Sample Report
  Request A Quote

Executive Summary

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, Leber's 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. Leber's hereditary optic neuropathy usually begins in a person's 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 patient's 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



Read More



Contact Us:
Ken Research
Ankur Gupta, Head Marketing & Communications

+91-9015378249

Table of Contents

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

Leber's 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

Leber's 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

Leber's 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

Leber's 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

Leber's 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

Leber's 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 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 Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 20

Number of Products under Development by Companies, Leber's 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, Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 48

Number of Products by Stage and Targets, Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 49

Number of Products by Mechanism of Actions, Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 50

Number of Products by Stage and Mechanism of Actions, Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 50

Number of Products by Routes of Administration, Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 52

Number of Products by Stage and Routes of Administration, Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 52

Number of Products by Molecule Types, Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 54

Number of Products by Stage and Molecule Types, Leber's 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

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 Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 20

Number of Products under Development by Companies, Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 21

Number of Products under Development by Universities/Institutes, Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 22

Products under Development by Companies, Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 22

Products under Development by Universities/Institutes, Leber's 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, Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 49

Number of Products by Stage and Mechanism of Action, Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 51

Number of Products by Stage and Route of Administration, Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 53

Number of Products by Stage and Molecule Type, Leber's 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

Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Pipeline by Alkeus Pharmaceuticals Inc 77

Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Pipeline by Biovista Inc 77

Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Pipeline by GenSight Biologics SA 78

Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Pipeline by Ixchel Pharma LLC 78

Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Pipeline by Khondrion BV 79

Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Pipeline by Mitotech SA 79

Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Pipeline by Spark Therapeutics Inc 80

Leber's 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 M's 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

Leber's 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

Single User License:
Report can be used by individual purchaser only

Site License:
Report can be shared by unlimited users within one corporate location, e.g. a regional office

Corporate User License:
Report can be shared globally by unlimited users within the purchasing corporation e.g. all employees of a single company

To know more information on Purchase by Section, please send a mail to query [@] kenresearch.com
 

select a license

Single User License
USD 3995 INR 256719
Site License
USD 7990 INR 513437
Corporate User License
USD 11985 INR 770156

NEWSLETTER BY CATEGORY




Testimonials

The report sent to us was on the point, and its information was quite extensive, well structured, and well researched. More importantly what we valued was your response time and professionalism. As a leading global consulting firm, our clients expect high quality deliverables in short periods of time, so a reliable research partner is essential. For the price that you have charged the quality of your services were exceptional. We look forward to continue our relationship with your team on future engagements....

This is with regards to your report on India PVC pipes and fitting market outlook industry outlook to 2019. This is a very well written report and accept my compliments on the same.... ...

I am pleased to see the very useful and productive information in the report..Very well structured great presentation reflect professionalism in your research studies....

While we still are studying and analysing your reports about Vietnam & Thailand, we would like to convey to your research team that they have done a very good job in compiling so much of information together. We hope to see more of such well researched reports. All the best & keep it up. ...

We would like to appreciate Ken Research for their great efforts and wonderful support in providing the Market Intelligence Report for ITPC. The information, statistics and research are well understandable and very clear to the point. We are happy with Ken research for their good client service, on-time delivery of the report and as said the report itself. Thank you Ken research for bringing the valuable output for us. We would be looking forward to have more research with you in the near future. Wish you a success in your business!...



Related Products in vertical
Ken Research Pvt. Ltd. Unit 14, Tower B3, Spaze I Tech Business Park, Sohna Road, sector 49
     Gurgaon, Haryana - 122001, India

download

Company Brochure

Engage with Us

query [@] kenresearch.com