This technology is a set of targets for reprogramming that rejuvenates the metabolome and retards disease progression in degenerative eye diseases.
There are currently no FDA-approved gene therapy products for retinal degenerative disorders such as Leber congenital amaurosis, retinitis pigmentosa (RP) and age-related macular degeneration (AMD). Those that are currently in development are limited in efficacy by the genetic heterogeneity of the diseases. With over 71 genes associated with RP, any monogenic gene therapy approach will only provide benefit to a small subset of patients. A therapeutic approach targeting an underlying disease metabolism, rather than a single gene, will be needed to create a broadly-applicable treatment option for RP and AMD patient populations. AMD affects 12 million Americans.
This technology describes a method for treating retinal degeneration by correcting aging-related metabolic dysregulation in the retina with gene therapy. Through overexpression of three different reprogramming genes involved in energy generation and antioxidant defense mechanisms, this method should rejuvenate aging, decrease oxidative stress and slow retinal degeneration. Importantly, unlike mutation-specific gene therapy approaches, this method slows disease progression through a broad mechanism. As such, this therapeutic strategy may be applicable to all RP and AMD patients, regardless of genotype.
IR CU18340
Licensing Contact: Kristin Neuman