Retinitis Pigmentosa (RP)

Medical unmet needs
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    High rate of blindness
    The most common inherited retinal diseases, one of the main causes of blindness
    Gradual loss of photoreceptor leading to retina regression/atrophy
    Incidence rate: 1/3,500~4,000 (US/EU)
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    Lack of effective therapies
    Only possible to delay visual loss and to restore partial visual acuity
    Autosomal recessive, dominant, X-linked RP
    Clinical researches on gene therapies and stem cell therapies
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    In need of new therapies
    Almost no clinical trials on Autosomal dominant (Ad RP)
    Ad RP : In need of WT gene expression as well as antimutagenic effect
Frequency of autosomal dominant RP mutations
Stage of Retinal Degeneration
Development of innovative therapies that can treat the main genetic causes
Rhodopsin (RHO in rod photoreceptor) – PR target molecule
RHO protein
RHO mutation : Change in folding or localization/transport of protein Problem in visual circuit / light energy transfer
More than 150 different mutation sites besides P23H in Exon1/ Continuous finding of new mutation sites
Development of innovative therapies which can target a variety of genetic mutations and restore normality simultaneously with alleviating the mutations
Ad RP mutation mRNA correcting RNA replacement enzyme vector-based RP therapies
  • mut RHO targeting

    Targeting dominant mutations to lower mutant : WT

  • RHO expression specificity

    Targeted expressing cell-specific WT protein expression

  • RHO expression level

    Possible to maintain endogenous gene expression level

  • RHO 5’UTR target

    Possible to target all Ad RP mutations with one therapy

  • Multi-functional

    Mutant removal and WT protein expression

  • Safety

    No use of endogenous cell machinery
    No need of extra protein introduction/expression