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Related Experiment Video

Updated: May 10, 2026

Efficient Derivation of Retinal Pigment Epithelium Cells from Stem Cells
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Retinal progenitor cells (jCell) for retinitis pigmentosa.

Jing Yang1,2, Baruch D Kuppermann2, David Liao3

  • 1Stem Cell Research Center, University of California, Irvine, Irvine, CA, United States.

Frontiers in Cellular Neuroscience
|September 10, 2025
PubMed
Summary

Intravitreal injection of human retinal progenitor cells (RPCs) was safe and well-tolerated in patients with retinitis pigmentosa (RP). Exploratory data suggest potential vision improvement, especially at higher doses, warranting further study.

Keywords:
allogeneic transplantationblindnesscell therapyintravitrealphotoreceptor dystrophy

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Area of Science:

  • Ophthalmology
  • Regenerative Medicine
  • Cell Therapy

Background:

  • Retinitis pigmentosa (RP) is a group of inherited retinal diseases causing progressive vision loss.
  • Current treatments for RP are limited, highlighting the need for novel therapeutic strategies.
  • Human retinal progenitor cells (RPCs) offer a potential cell-based therapy for retinal degeneration.

Purpose of the Study:

  • To evaluate the safety and tolerability of intravitreal RPC injections in adults with non-syndromic RP.
  • To assess the potential efficacy of RPCs in improving visual acuity and other visual functions.

Main Methods:

  • A prospective, multicenter, open-label, single-arm Phase I/IIa safety study involving 28 adults with RP.
  • Participants were divided into two cohorts based on baseline visual acuity.
  • Subjects received a single intravitreal injection of RPCs at doses ranging from 0.5 to 3.0 × 10^6 cells.

Main Results:

  • Intravitreal RPC injection was generally well-tolerated, with mostly mild, transient adverse events.
  • No signs of graft rejection were observed.
  • Exploratory efficacy assessments indicated potential improvements in best-corrected visual acuity (BCVA) at all doses, with a possible dose-response relationship at higher RPC concentrations.

Conclusions:

  • Intravitreal RPC injection is a safe and tolerable treatment for RP patients.
  • The study provides preliminary evidence of potential visual function improvement in RP patients treated with RPCs.
  • Further investigation in larger, controlled trials is warranted to confirm these findings and explore the therapeutic potential of RPCs in RP.