Genetic Ophthalmology Disorders Drug Development Pipeline Review, 2017

Published: August 2017
No. of Pages: 138
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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.There are a total of 14 products in development for this indication, by 11 companies and one academic institution. Key companies operating in this pipeline space include Astellas, and the biomedical charity Fondazione Telethon.

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. There are a total of nine products in development for this indication, by six companies and two academic institutions. Spark therapeutics, who are fielding two products in this indication, are the most active pipeline developer for Leber congenital amaurosis.

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. Males are affected much more often than females.There are a total of 12 products in development for this indication, by eight companies and one academic institution. Key companies operating in this pipeline space include Biovista, GenSight Biologics and Ixchel Pharma.

Usher syndrome is characterized by hearing impairment and progressive vision loss. The major symptoms of Usher syndrome are hearing loss and secondary retinitis pigmentosa.There are a total of six products in development for this indication, by four companies and one academic institution. ProQR Therapeutics are fielding two products in this indication, while all other players are fielding one each.

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. This has the most active pipeline, with a total of 54 products in development, by 36 companies and 11 academic institutions. Key companies operating in this pipeline space include Spark Therapeutics, Astellas, Horama, ID Pharma, Columbia University Medical Center and John Hopkins University.

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.

The report "Genetic Ophthalmology Disorders Drug Development Pipeline Review, 2017", 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 Stargardtdisease, Lebercongenital amaurosis, Leber’s hereditary optic neuropathy, Usher Syndrome and Retinitis pigmentosa, and features dormant and discontinued projects.


  • 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.

Published By: GBI Research
Product Code: GBI Research1630

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