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ISTEM-01

Development stage: Phase 2
Lead developer: Centre d'Etude des Cellules Souches
Modality: Stem Cell Therapies → Cell Therapies
Administration: Ophthalmic

Overview

ISTEM-01 is an investigational cell therapy product consisting of retinal pigment epithelium (RPE) cells derived from human embryonic stem cells (hESCs). Developed by the Centre d'Etude des Cellules Souches (I-Stem), it is formulated as a medicinal patch for subretinal implantation. The therapy is intended to treat retinitis pigmentosa caused by monogenic mutations (such as RPE65, LRAT, or MerTK) by replacing damaged or lost RPE cells that are critical for photoreceptor survival. It is currently in Phase 1/2 clinical development (STREAM trial), where it is evaluated for safety, tolerability, and preliminary efficacy in patients with advanced visual loss.

Other names: hESC-derived RPEHuman Embryonic Stem Cell Derived Retinal Pigment Epithelium

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