Luxa Biotechnology announces paper on 'Identification of Biomarkers for Diversity and Transplantation Efficacy of Retinal Pigment Epithelial Cells' by Dr. Sally Temple.
Luxa Biotechnology (LuxaBio), a joint venture between Y2 Solution Co. Ltd, Seoul, South Korea and the Neural Stem Cell Institute (NSCI), Rensselaer, New York, today announced the publication of a paper titled "Identification of Biomarkers for Diversity and Transplantation Efficacy of Retinal Pigment Epithelial Cells" in the Journal of Experimental Medicine(JEM). Luxa Biotechnology supported this research to provide in-depth characterization of retinal pigment epithelial stem cell (RPESC)-derived RPE (RPESC-RPE) product identity and critical quality attributes that affect efficacy and safety of the RPE cell product for patients with retinal disease.
Retinal degenerative diseases such as dry age-related dry macular degeneration (AMD) are a major cause of blindness. Currently there are no vision-improving treatment options for dry AMD. This study identified biomarkers to enhance the efficacy and safety of RPE cell transplantation therapy for dry AMD. The research team isolated subpopulations of adult RPESC from donated eyes and analyzed their gene expression using single-cell RNA sequencing technology. They evaluated the integration of RPESC-RPE subpopulations into mature RPE monolayers and assessed their efficacy at vision rescue in the Royal College of Surgeons (RCS) rat animal model.
This research revealed 13 subpopulations of cells within the RPESC-RPE product, each with a unique gene expression pattern. Some subpopulations exhibited characteristics of the RPE progenitor cells that are more effective at RCS rat vision rescue after transplantation than mature RPE cells. One subpopulation showed high expression of a gene belonging to the polycomb transcription factor family, associated with cell regeneration, differentiation, in vivo efficacy, and cell integration ability. This noteworthy finding identified the long non-coding RNA(lncRNA) TREX as a biomarker for in vitro engraftment and in vivo efficacy at vision rescue. TREX inhibition decreased cell integration, while overexpression increased cell integration, demonstrating TREX's involvement in this processes critical to RPE cell transplantation. Vision improvements were associated with a threshold TREX level, further confirming TREX as a biomarker predicting the efficacy of RPE cell replacement therapy.
Dr. Sally Temple commented, "I am delighted that this research has provided new markers for the development of cell therapies for macular degeneration and other retinal degenerative diseases." She added, "In future research and development, we will utilize the TREX gene to improve the RPESC-RPE cell product. Our goal is to restore vision and provide improved quality of life for dry AMD patients."
LuxaBio is currently conducting a Phase 1/2a clinical trial of RPESC-RPE-4W therapy for dry AMD. The clinical trial conducted in the United States is actively enrolling patients aged 55 and older with vision loss due to dry AMD.
The paper can be found at the following link:
https://rupress.org/jem/article-abstract/220/12/e20230913/276285/Identifying-biomarkers-of-heterogeneity-and?redirectedFrom=fulltext
