Evidence map›Paper›PMID 40961946›Full record

Trial reportCell stem cell2025

Safety and tolerability of RPESC-RPE transplantation in patients with dry age-related macular degeneration: Low-dose clinical outcomes.

Rajesh C Rao, Brigitte L Arduini, Susan Borden, Dhruv Sareen, Clive Svendsen, Paul Lee, Charles Ryan, Shilpa Kodati, Caroline Nyaiburi, Keith Wolsieffer and 6 more

Registry-linked trialAbstract readClinical Trial, Phase IClinical Trial, Phase IIMulticenter Study
In one paragraph

Trial report in Cell stem cell, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04627428 (A Phase1/2a, Open-Label Study to Evaluate the Safety and Tolerability of RPE Stem Cell-derived RPE), which is not on this map. Cited by 9 papers.

0numbers the graph read from it
0cells of the map it votes in
9citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT04627428 phase1 / phase2recruitingnot on this map

A Phase1/2a, Open-Label Study to Evaluate the Safety and Tolerability of RPE Stem Cell-derived RPE (RPESC-RPE) Transplantation as Therapy for Dry Age-related Macular Degeneration (AMD)

TypeinterventionalSponsorLuxa Biotechnology, LLCRan2022 to 2026Enrolled18ConditionsDry Age-related Macular DegenerationArmsRPESC-RPE-4W
3 · Its place in the literature

Who cites it

9 citing papers in PubMed.

  1. Review
  2. Review
  3. Article
  4. 3D epigenomic landscape of the human retinal pigment epithelium.Proceedings of the National Academy of Sciences of the United States of America · 2026
    Article
  5. Review
  6. Cell therapy for age-related macular degeneration.Mammalian genome : official journal of the International Mammalian Genome Society · 2026
    Review
  7. Article
  8. Article
  9. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

16 authors.

Rajesh C RaoKellogg Eye Center, University of Michigan Medical Center, 1000 Wall St., Ann Arbor, MI 48105, USA. Electronic address: rajeshr@med.umich.edu.
Brigitte L ArduiniNeural Stem Cell Institute, 150 New Scotland Ave., Albany, NY 12208, USA. Electronic address: brigittearduini@neuralsci.org.
Susan BordenNeural Stem Cell Institute, 150 New Scotland Ave., Albany, NY 12208, USA.
Dhruv SareenCedars-Sinai Biomanufacturing Center, 8687 Melrose Ave., West Hollywood, CA 90069, USA; Board of Governors Regenerative Medicine Institute, Los Angeles, CA 90048, USA.
Clive SvendsenCedars-Sinai Biomanufacturing Center, 8687 Melrose Ave., West Hollywood, CA 90069, USA; Board of Governors Regenerative Medicine Institute, Los Angeles, CA 90048, USA.
Paul LeeKellogg Eye Center, University of Michigan Medical Center, 1000 Wall St., Ann Arbor, MI 48105, USA.
Charles RyanKellogg Eye Center, University of Michigan Medical Center, 1000 Wall St., Ann Arbor, MI 48105, USA.
Shilpa KodatiKellogg Eye Center, University of Michigan Medical Center, 1000 Wall St., Ann Arbor, MI 48105, USA.
Caroline NyaiburiEmmes Corp., 401 N. Washington Street, Suite 700, Rockville, MD 20850, USA.
Keith WolsiefferEmmes Corp., 401 N. Washington Street, Suite 700, Rockville, MD 20850, USA.
Eric OhLuxa Biotechnology, 2050 Center Ave., Suite 435, Fort Lee, NJ 07024, USA.
Shuna ParkLuxa Biotechnology, 2050 Center Ave., Suite 435, Fort Lee, NJ 07024, USA.
Glenna FordLuxa Biotechnology, 2050 Center Ave., Suite 435, Fort Lee, NJ 07024, USA.
Keith DionneLuxa Biotechnology, 2050 Center Ave., Suite 435, Fort Lee, NJ 07024, USA.
Sally TempleNeural Stem Cell Institute, 150 New Scotland Ave., Albany, NY 12208, USA; Luxa Biotechnology, 2050 Center Ave., Suite 435, Fort Lee, NJ 07024, USA.
Jeffrey SternNeural Stem Cell Institute, 150 New Scotland Ave., Albany, NY 12208, USA; Luxa Biotechnology, 2050 Center Ave., Suite 435, Fort Lee, NJ 07024, USA. Electronic address: jeffreystern@neuralsci.org.

Funding

Phase1/2a Clinical Trial of RPESC-derived RPE Transplantation as Therapy for Non-exudative Age-related Macular DegenerationUH3EY031810 · NEI · REGENERATIVE RESEARCH FOUNDATION · PI Jeffrey H Stern · 2021 to 2026
$3.6M
Transplantation of Adult, Tissue-Specific RPE Stem Cells as Therapy for Non-exudative Age-Related Macular Degeneration AMDU01EY030581 · NEI · REGENERATIVE RESEARCH FOUNDATION · PI Jeffrey H Stern · 2019 to 2026
$1.7M
Phase1/2a Clinical Trial of RPESC-derived RPE Transplantation as Therapy for Non-exudative Age-related Macular DegenerationUG3EY031810 · NEI · REGENERATIVE RESEARCH FOUNDATION · PI STERN, JEFFREY H · 2020 to 2020
$201k
NEI NIH HHS U01 EY030581NEI NIH HHS UG3 EY031810NEI NIH HHS UH3 EY031810
6 · The paper itself

Abstract

Retinal pigment epithelium (RPE) cell atrophy in dry age-related macular degeneration (AMD) compromises photoreceptor cell function, leading to vision loss. Stem cell-based RPE replacement therapy aims to reverse disease progression and restore vision. RPESC-RPE-4W, a post-mitotic adult RPE stem cell-derived RPE (RPESC-RPE) progenitor cell product, exhibits consistent safety and efficacy in preclinical studies. The first-in-human clinical trial of RPESC-RPE-4W completed low-dose cohort 1 interventions (NCT04627428). Six subjects received a subretinal suspension of 50,000 RPESC-RPE-4W cells. No significant inflammation, tumor, or product-related serious adverse events were observed. Best-corrected visual acuity in the three worse-seeing group A subjects improved by an average of +21.67 letters from baseline at 12 months. Three better-seeing group B subjects improved by an average of +3.0 letters at 6 months. The positive safety and tolerability outcomes for low-dose cohort 1 enabled dose escalation to mid-dose RPESC-RPE-4W therapy for dry AMD.

Indexed as

Geographic AtrophyMacular DegenerationRetinal Pigment EpitheliumStem CellsStem Cell TransplantationAgedAged, 80 and overFemaleHumansMaleMiddle AgedTreatment OutcomeVisual Acuityadult stem cellcell therapyclinical trialgeographic atrophymacular degenerationretinaretinal pigment epitheliumvision

Identifiers

PMID40961946
PMCPMC12533590

What OpenQuestion holds

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Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.