Gene Therapy for Treatment of CRX-Autosomal Dominant Retinopathies

Description of Technology:

Mutations in the cone rod homeobox (CRX) transcription factor lead to distinct retinopathy phenotypes, including early-onset vision impairment in dominant Leber congenital amaurosis (LCA). Adeno-Associated virus (AAV) vector-mediated delivery of a CRX cDNA under the control of a CRX promoter region partially restored photoreceptor phenotype and expression of phototransduction genes in an in vitro model of CRX-LCA.

Pigment Epithelium-Derived Factor Peptides and Their Use for Treating Retinal Degeneration

Summary:  

The National Eye Institute (NEI) seeks research co-development partners and/or licensees for the development of an AAV2-based delivery system or an eyedrop formulation to deliver a Pigment Epithelium-Derived Factor (PEDF) peptide as a gene-agnostic approach to treating inherited retinal diseases. 

Identification of Biomarkers for Onchocerciasis Control and Elimination

The technology outlined in the document is focused on the discovery of novel biomarkers for Onchocerciasis, a parasitic disease also known as river blindness, which is transmitted by blackfly vectors. The document describes the identification of the first Onchocerca volvulus-encoded molecules that are adult-specific and can be used as markers for macrofilaricidal activity, which is the ability to kill adult worms.