Genetic Ophthalmology Disorders Drug Development Pipeline Review, 2017

“The Report Genetic Ophthalmology Disorders Drug Development Pipeline Review, 2017 provides information on pricing, market analysis, shares, forecast, and company profiles for key industry participants. –



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 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 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.

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– 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

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

Lebers Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) – Overview 11

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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

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