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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. 

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

Luris reference number

INV-086.030

Patent status

  • Patent in national phase
  • EP, US, CN, JP, CA, AU, IL, IN

Data available on request

  • Supporting publications
  • Additional data under CDA

Further information

Ian Nicoud Knowledge Broker (LUMC Div. 4) +31-71-527 5611 i.b.nicoud@luris.nl