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Gene therapy for retinitis pigmentosa due to mutations in the rhodopsin gene

Image from Licence Details: Gene therapy for retinitis pigmentosa due to mutations in the rhodopsin gene

Applications: Gene therapy, therapeutics, ophthalmology

1 in 3,000-7,000 people suffers from retinitis pigmentosa (RP), with 25-30% of RP cases being autosomal dominant RP (adRP). Of these, 25% are due to mutations in the rhodopsin gene (RHO). The clinical phenotypes of rhodopsin-mediated adRP (RHO-adRP) are diverse and range from mild night blindness to severe visual impairment. There are currently no universally effective treatments or cures for RHO-adRP.

Features Benefits
  • AAV-based gene therapy for retinitis pigmentosa caused by mutations in the rhodopsin gene.
  • The developmental product offers a potential long-lasting therapy for all RHO-adRP sufferers.
  • Delivers artificial mirtrons (intron-derived microRNAs) to suppress the expression of the endogenous mutated rhodopsin.
  • Pre-clinical development ongoing at Oxford via an MRC DPFS-funded project, the principal objective of which is to deliver a clinical candidate.
  • Also delivers a functional version of the rhodopsin gene to replace the endogenous mutated form.
  • Oxford has a track record in the development of gene therapies for inherited retinal diseases and possesses extensive pre-clinical development, regulatory and clinical trials experience.
  • Patent applications pending in the US, Europe and Japan.


Patented and available for:

  • Licensing
  • Co-development
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