Evidence map›Paper›PMID 42055007›Full record

ArticleCell reports. Medicine2026

IND-enabling safety and efficacy of RPESC-RPE-4W, an adult RPE progenitor cell therapy for dry age-related macular degeneration.

Brigitte L Arduini, Timothy A Blenkinsop, Michael Naimark, Susan Borden, Carol Charniga, Melissa Campbell, Natasha Rugenstein, Richard J Davis, Nazia Alam, Glen Prusky and 21 more

Abstract read
In one paragraph

Article in Cell reports. Medicine, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing 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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

31 authors.

Brigitte L ArduiniNeural Stem Cell Institute, Albany, NY 12208, USA. Electronic address: brigittearduini@neuralsci.org.
Timothy A BlenkinsopNeural Stem Cell Institute, Albany, NY 12208, USA.
Michael NaimarkNeural Stem Cell Institute, Albany, NY 12208, USA.
Susan BordenNeural Stem Cell Institute, Albany, NY 12208, USA.
Carol CharnigaNeural Stem Cell Institute, Albany, NY 12208, USA.
Melissa CampbellNeural Stem Cell Institute, Albany, NY 12208, USA.
Natasha RugensteinNeural Stem Cell Institute, Albany, NY 12208, USA.
Richard J DavisNeural Stem Cell Institute, Albany, NY 12208, USA.
Nazia AlamBurke Neurological Institute, Weill Cornell Medical College, White Plains, NY 10605, USA.
Glen PruskyBurke Neurological Institute, Weill Cornell Medical College, White Plains, NY 10605, USA.
Amber M FryeNeural Stem Cell Institute, Albany, NY 12208, USA.
Francesca MazzoniDepartment of Biological Sciences, Fordham University, Bronx, NY 10458, USA.
Silvia C FinnemannDepartment of Biological Sciences, Fordham University, Bronx, NY 10458, USA.
Cuiping ZhaoNeural Stem Cell Institute, Albany, NY 12208, USA.
Patricia LedermanNeural Stem Cell Institute, Albany, NY 12208, USA.
Janmeet S SainiNeural Stem Cell Institute, Albany, NY 12208, USA.
Thomas R KiehlNeural Stem Cell Institute, Albany, NY 12208, USA.
Nicole RengaNeural Stem Cell Institute, Albany, NY 12208, USA.
Jade A KozakNeural Stem Cell Institute, Albany, NY 12208, USA.
Penny A RocchioNeural Stem Cell Institute, Albany, NY 12208, USA.
Enrique SaleroNeural Stem Cell Institute, Albany, NY 12208, USA.
Nathan C BolesNeural Stem Cell Institute, Albany, NY 12208, USA.
Susan K GoderieNeural Stem Cell Institute, Albany, NY 12208, USA.
Steven LotzNeural Stem Cell Institute, Albany, NY 12208, USA.
Joan E AdamoUniversity of Rochester, Clinical and Translational Science Institute, Rochester, NY 14642, USA.
Sharyl ZaccaglinoUniversity of Rochester, Clinical and Translational Science Institute, Rochester, NY 14642, USA.
Khadijah OnanugaNeural Stem Cell Institute, Albany, NY 12208, USA.
Michael FiskeUniversity of Rochester, Rochester, NY 14627, USA.
Alexandra CapelaBonovox Lda, Aveiro, Portugal.
Sally TempleNeural Stem Cell Institute, Albany, NY 12208, USA. Electronic address: sallytemple@neuralsci.org.
Jeffrey H SternNeural Stem Cell Institute, Albany, NY 12208, 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

Dry age-related macular degeneration (AMD) is a prevalent blinding disorder characterized by loss of retinal pigment epithelium (RPE) cells and resulting central vision impairment. Here, we describe preclinical studies supporting an investigational new drug (IND) application for adult RPE stem cell-derived RPE (RPESC-RPE) cell replacement therapy. We establish donor-to-donor reproducibility and Good Manufacturing Practice (GMP) processes and evaluate the GMP cell product in preclinical studies conducted in accordance with Good Laboratory Practice principles. Efficacy experiments in Royal College of Surgeons rats show that subretinal implantation of the 4-week, postmitotic, progenitor-stage product (RPESC-RPE-4W) produces significant and durable vision rescue compared to vehicle control. Biodistribution and safety studies in immunocompromised Rowett Nude rats demonstrate a favorable safety profile, with no adverse effects related to the cell product. These studies underpin allowance of an ongoing phase 1/2a clinical trial.

Indexed as

Adult Stem CellsCell- and Tissue-Based TherapyDrugs, InvestigationalMacular DegenerationRetinal Pigment EpitheliumStem CellsStem Cell TransplantationAnimalsFemaleHumansMaleRatsRats, NudeDrugs, Investigationaladult stem cellAMDcell therapyinvestigational new drugmacular degenerationretinal pigment epitheliumRPE

Identifiers

PMID42055007
PMCPMC13198292

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Registered trials

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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.