RZ-004

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
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Stage of Retinal Degeneration
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Development of innovative therapies that can treat the main genetic causes
Rhodopsin (RHO in rod photoreceptor) – PR target molecule
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RHO mRNA
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RHO protein
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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
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  • 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