Gene therapy for retinitis pigmentosa due to mutations in the rhodopsin gene
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.
Applications: Gene therapy, therapeutics, ophthalmology
| 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
- Co-development
- Licensing