Description:
Summary:
The National Eye Institute (NEI) seeks research co-development partners and/or licensees for the development of an eyedrop formulation and/or AVV2-based delivery system to deliver the 17-mer [H105A] peptide to treat retinal degeneration.
Description of Technology:
Retinitis pigmentosa (RP) is one of the most common inherited retinal diseases (IRDs) that is estimated to affect 1 in 4,000 people in the United States and worldwide. Over 100,000 people in the US, and 1.5 million people worldwide suffer from RP.]RP leads to progressive photoreceptor cell degeneration and ultimately vision loss, with more than 90 genes implicated in molecular pathways towards photoreceptor cell death. Due to this high heterogeneity, therapeutic approaches targeting specific genes generally benefit few patients, while for most forms of RP few or no medical options are available. Thus, there remains a need to identify new and more effective treatments for RP and other inherited retinal degenerations. Mutation-independent strategies to protect photoreceptors against continued damage and degradation are appealing approaches.
Researchers at the NEI have developed a pigment epithelium-derived factor (PEDF) peptide of 17 amino acid residues (17-mer) with an amino acid substitution at position 105 from a histidine to an alanine (PEDF 17-mer[H105A]) that exhibits a highly potent effect on photoreceptor protection in in vivo mouse models of RP. The NEI-developed technology described herein relates to: 1- An eyedrop formulation to deliver the 17-mer [H105A] and 2- An AAV2-based delivery system for sustained production and secretion of PEDF and its neurotrophic domain of 17 amino acid residues (17-mer[H105A]) to treat or prevent photoreceptor degradation and vision loss in patients with IRDs.
The protected claims for this invention are directed to composition of matter of both the PEDF 17-mer[H105A] peptide in an eye drop formulation and the AAV2-based delivery system including the nucleic acid encoding the PEDF protein or the PEDF 17-mer[H105A] peptide. The claims also provide methods for using this technology to treat IRDs (e.g. retinitis pigmentosa, Leber congenital amaurosis, cone-rod dystrophy, age-related macular degeneration, Stargardt-like macular degeneration, or a maculopathy). The technology is available for licensing and co-development.
Potential Commercial Applications:
- Potential simple and effective treatment for inherited eye diseases contributing to Retinitis Pigmentosa (RP)
- Potentially prevent patient’s progression to late-stage RP
Competitive Advantages:
- Only known therapeutic treatment for RP that can substantially minimize patient progression to late-stage RP
- Eye drop PEDF formulation accessible to more patients
- Eye drop PEDF formulation provides easy administration route
- PEDF formulation mimics the natural protective process lost in patients with inherited eye diseases contributing to RP