Treatment of Retinal Disorders by Transforming Growth Factor-Beta (TFG-beta ) Superfamily Proteins and their Antagonists

Background: Retinal disorders often turn into debilitating diseases, and in most cases only few treatment options are available. With available treatment options, vision is usually not restored and treatments are often limited to delaying disease progression. Recent developments using implanted devices suggest that a limited degree of vision can be restored, but such devices have been problematic and are suboptimal for improving or restoring ocular function. The development and/or regeneration of retinal deficits remains an unmet medical need.

Researchers at the University of California have discovered various compounds that regulate production of retinal neural tissue by interacting with signaling pathway(s) that are associated with transforming growth factor beta (TGF- superfamily members. Specifically, the TGF- superfamily members growth and differentiation factor 11 (GDF11), GDF8, and TGF- antagonists such as follistatin can be utilized to enhance the development and/or regeneration of photoreceptors, amacrine cells, and retinal ganglion cells o the retina. Technology: Growth and differentiation factor 11 (GDF11), a member of the TGF-beta superfamily of signaling factors is expressed in the developing nervous system, including the retina. University of California Irvine researchers, employing developing mice lacking the GDF11 gene, a known GDF11 antagonist and retinal explants from GDF11 null or normal developing mice have shown that GDF11 signaling regulates the production of retinal ganglion, amacrine and photoreceptor cells during development. Interestingly, the mechanism by which GDF11 signaling influences the differentiation of neural cells in the retina is via temporal control over the period of time that progenitor cells are competent to produce progeny cells. In this manner, GDF11 is a negative regulator of retinal cell development. The discovery was reported in Science (2005) Volume 308, page 1927. Patent applications were filed. UCI seeks to develop and commercialize this invention via a license agreement and/or by performing sponsored research in collaboration with industry. Application: UCI researchers have developed a unique assay system for discovery of compositions that modulate the GDF11 and GDF8 signaling pathway. The novel finding that the differentiation of neural progenitor cells can be regulated using GDF11 or GDF8 agonists or antagonists has the potential to effect desired changes in photoreceptors, amacrine cells, and retinal ganglion cells of the retina, and can be used to treat diseases such as retinitis pigmentosa, macular degeneration, Leber's congenital amaurosis, glaucoma, optic nerve injury and retinal ischemia. The therapeutic applications can impact neurodegenerative diseases in general since GDF11 is expressed in several regions of the developing nervous system.

Type of Offer: Licensing



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