Treating Familial Blindness

About

An adeno-associated virus-based recombinant gene therapy vector for the treatment of a retinal disorder, specifically Leber's congenital amaurosis and retinitis pigmentosa, due to mutations in Crumbs homologue-1. LCA and RP are rare genetic disorders, caused by mutation in the Crumbs homologue-1 (CRB-1) gene, with prevalences of 1/40,000 and 1/3,000 respectively. Scientists from Leiden University Medical Center (LUMC) and the Netherlands Institute for Neuroscience of the Royal Netherlands Academy of Arts and Sciences (KNAW) have developed an adeno-associated virus (AAV)-based recombinant gene therapy vector for the treatment of a retinal disorder, specifically Leber's congenital amaurosis (LCA) and retinitis pigmentosa (RP), due to mutations in Crumbs homologue-1 (CRB-1). Presently there are no therapeutics or effective treatments available to prevent, delay or treat LCA or RP in humans. Therefore, there is a need for methods and means for the treatment of retinal disorders due to mutations in CRB1. LUMC has attracted significant funding for the preclinical development and Phase I/IIa clinical trial. We are currently looking for a development partner to collaborate, contribute expertise and / or provide matching funds for the clinical development stage, in return for a license or exclusive option to license the know-how and global patent family. This project is a unique opportunity for a public-private partnership to develop a treatment for a rare orphan disease.  Key Benefits Novel treatment for orphan disease Promising in-vivo data Applications Retinal disorders: Leber's congenital amaurosis Retinitis pigmentosa Development Stage Positive in-vivo testing Start of GMP validation Patent Status Patent in national phase EP, US, CN, JP, CA, AU, IL, IN