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Overview of Genetic Ophthalmology Disorders Drug Development Pipeline Review, 2017 - Market Research Report
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.
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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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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
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
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