{
  "checked": "2026-09-17",
  "programme_count": 6,
  "programmes": [
    {
      "id": "rv001",
      "programme": "RV-001",
      "evidence": "Sponsor-reported open-label Phase 1/2 interim data: six participants, three per dose cohort. All three high-dose participants reached light perception or better within one month; low-dose one of three around three months.",
      "limitation": "Not normal vision or a randomized demonstration. Primary endpoint safety; updated total target 45. Investigational.",
      "sources": [
        "https://restore-vis.com/en/news/3241674.html",
        "https://jrct.mhlw.go.jp/en-latest-detail/jRCT2033240611"
      ]
    },
    {
      "id": "rpe",
      "programme": "iPSC-derived RPE transplantation",
      "evidence": "2014 autologous RPE-sheet recipient: unchanged visual acuity at one year. Later five-recipient donor-RPE study: graft survival at one year.",
      "limitation": "Support-cell layer, not whole retina. No systemic immunosuppression in matched-donor study did not mean no local steroids or immune reactions.",
      "sources": [
        "https://doi.org/10.1056/NEJMoa1608368",
        "https://doi.org/10.3390/jcm9072217"
      ]
    },
    {
      "id": "retinal-sheets",
      "programme": "Retinal organoids and DSP-3077",
      "evidence": "Two-patient Japanese study demonstrated graft survival; later 208-week registry results showed no ETDRS acuity improvement. DSP-3077 US study plans 12 participants; first treatment sponsor-announced August 2026.",
      "limitation": "Graft survival is not proven functional synaptic integration in humans. US trial focuses on safety; orphan designation is not approval.",
      "sources": [
        "https://pubmed.ncbi.nlm.nih.gov/38065067/",
        "https://jrct.mhlw.go.jp/en-latest-detail/jRCTa050200027",
        "https://clinicaltrials.gov/study/NCT06891885"
      ]
    },
    {
      "id": "cornea",
      "programme": "iPSC corneal epithelial sheets",
      "evidence": "Four-patient Lancet study for limbal stem-cell deficiency: visual acuity and corneal outcomes improved at 52 weeks; no serious adverse events over two years.",
      "limitation": "Small uncontrolled study; non-serious events occurred. Corneal disease, not retinitis pigmentosa.",
      "sources": [
        "https://pubmed.ncbi.nlm.nih.gov/39522528/"
      ]
    },
    {
      "id": "ourep",
      "programme": "OUReP photoelectric film",
      "evidence": "Published rat visual-response and monkey visual-evoked-potential findings.",
      "limitation": "Preclinical outcomes. Verified published human efficacy results and validated treatment price not located. Exact-name ClinicalTrials.gov search is not exhaustive worldwide.",
      "sources": [
        "https://www.okayama-u.ac.jp/user/kouhou/ebulletin/ipe/vol8/ipe_001.html",
        "https://pubmed.ncbi.nlm.nih.gov/29633282/",
        "https://pubmed.ncbi.nlm.nih.gov/28181076/"
      ]
    },
    {
      "id": "mco010",
      "programme": "MCO-010 / MOGENRY",
      "evidence": "Official Japanese pioneering and orphan regenerative-product designations dated January 16, 2026. Sponsor announced FDA BLA acceptance and filing September 9.",
      "limitation": "Designations and application filing are not marketing approval. FDA acceptance letter not independently retrieved. Separate from Restore Vision RV-001.",
      "sources": [
        "https://www.pmda.go.jp/files/000278346.pdf",
        "https://www.pref.saitama.lg.jp/documents/19123/iyakukishin0116-3.pdf",
        "https://nanostherapeutics.com/2026/09/09/nanoscope-therapeutics-announces-u-s-food-and-drug-administration-acceptance-of-biologics-license-application-for-mogenry-for-the-treatment-of-retinitis-pigmentosa-with-severe-vision-loss"
      ]
    }
  ]
}