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.
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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.
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.
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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?
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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
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Michelle Thoras
201 Spear Street 1100,
Suite 3036, San Francisco,
CA 94105, United States
Tel: 1-415-349-0054
Toll Free: 1-888-928-9744
Mail: sales@radiantinsights.com
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