Treatment of familial blindness
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.
- Novel treatment for orphan disease
- Promising in-vivo data
- Leber's congenital amaurosis
- Retinitis pigmentosa
- Positive in-vivo testing
- Start of GMP validation
Luris reference numberINV-086.030
- Patent in national phase
- EP, US, CN, JP, CA, AU, IL, IN
Data available on request
- Supporting publications
- Additional data under CDA