04/28/2026
Available now Cell Reports Medicine: “IND-enabling Safety and Efficacy of RPESC-RPE-4W, an Adult RPE Progenitor Cell Therapy for Dry Age-related Macular Degeneration.” This paper describes the pathway to an Investigational New Drug (IND) allowance from the FDA, a prerequisite for our ongoing clinical trial. Strong preclinical safety and efficacy, along with GMP process development and promising clinical results, also contributed to designation from the FDA in 2025.
Led by Sally Temple and Jeffrey Stern, this multi-institutional effort included Brigitte Arduini, Timothy Blenkinsop, Michael Naimark, Susan Borden, Carol Charniga, Melissa Campbell, Natasha Rugenstein, Richard Davis, Nazia Alam, Glen Prusky, Amber Frye, Francesca Mazzoni, Silvia Finnemann, Cuiping Zhao, Patricia Lederman, Janmeet Saini, Thomas Kiehl, Nicole Renga, Jade Kozak, Penny Rocchio, Enrique Salero, Nathan Boles, Susan Goderie, Steven Lotz, Joan Adamo, Sharyl Zaccaglino, Khadijah Onanuga, Michael Fiske and Alexandra Capela.
The Neural Stem Cell Institute received support from NYSTEM (New York State Department of Health), the National Eye Institute and Luxa Biotechnology. The UR CTSI is supported in part by a Clinical and Translational Science Award from the National Center for Advancing Translational Sciences, a component of the National Institutes of Health.
We are grateful to the eye donors and their families, and to the eye banks that enabled this work, including Lions Eye Bank of the Northeast in Albany, Central Florida Lions Eye in Tampa, Lions Gift of Sight of the University of Minnesota, the Eye Bank of New York, the San Diego Eye Bank, Eversight, ConnectLife, and the National Disease Research Interchange (NDRI).
Fordham University
Free Full-Text | https://www.cell.com/cell-reports-medicine/fulltext/S2666-3791(26)00196-5