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Research report explores the genetic ophthalmological disorders treatment pipeline review

Medical Market Research

Genetic Ophthalmological Disorders Treatment Pipeline Review 2017

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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.

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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.


- 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 access

- 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 Content: Key Points

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



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