Retinal progenitor cells (jCell) for retinitis pigmentosa.

ABSTRACT:

Objective

To assess the safety and tolerability of intravitreal injection of human retinal progenitor cells (RPCs) at multiple dose levels in adults with non-syndromic retinitis pigmentosa (RP).

Design

A prospective, multicenter, open-label, single-arm, Phase I/IIa safety study of RPCs in adults with RP (n = 28). Two patient cohorts were studied: Cohort 1: BCVA no better than 20/200 and no worse than Hand Motions, and Cohort 2: BCVA no better than 20/40 and no worse than 20/200).

Subjects

Adults (n = 28) with a clinical diagnosis of RP confirmed by electroretinogram, consenting to gene mutation typing for genes involved in inherited retinal degenerations and related disorders, and willing to undergo human leukocyte antigen (HLA) typing.

Methods

Su

SUBMITTER: Yang J 

PROVIDER: S-EPMC12414927 | biostudies-literature | 2025

REPOSITORIES: biostudies-literature