[Edaily Reporter YU JIN-HEE ] “The stem cell therapy we are developing has succeeded in substantially restoring damaged vision, overcoming the limitations of existing drugs that merely slowed the progression of the disease. The long-term efficacy demonstrated over 24 months is a decisive indicator proving our unrivaled competitiveness compared to global competitors.”

Keith Dionne, CEO of Luxa Biotechnology (hereinafter “Luxa”), made this statement during an exclusive session at “Bio Plus Co., Ltd. (BIX) 2026,” which opened on the 29th at COEX in Seoul.

Luxa is a joint venture for biopharmaceutical development established in 2019 by Y2 Solution(011690)and the U.S.-based stem cell research institute, the Neural Stem Cell Institute (NSCI). CEO Dionne personally presented the results of the additional follow-up observations from the Phase 1/2a clinical trial of “RPESC-RPE-4W,” a cell therapy candidate for dry age-related macular degeneration, at the event, expressing strong confidence in the development progress.

Low-dose group maintains visual improvement for 24 months… Efficacy surpasses that of competitors

This presentation features key data derived after completing treatment for a total of 18 patients under U.S. Food and Drug Administration (FDA) approval. It focuses on the 24-month long-term follow-up results from the low-dose group (Cohort 1) and the 9-month interim data from the medium-dose group (Cohort 2).

According to the announcement, in the low-dose group, the average best-corrected visual acuity (BCVA) in the treated eye remained stably improved compared to baseline levels for 24 months. This contrasts with typical treatments for ophthalmic diseases, which often produce only a temporary “flash effect” early in treatment or merely slow the progression of the disease.

In particular, among the group of severe patients with very low baseline vision, best-corrected visual acuity improved by an average of 16.6 letters compared to baseline at the 24-month mark. Based on the Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity chart used in the clinical trial, this equates to being able to read more than three additional lines. This indicates that patients who had reached the stage of legal blindness have recovered to a level where they can lead independent daily lives, representing a rate of improvement more than twice that of competing treatments. Even the overall average for the low-dose group showed an improvement of 9.3 letters compared to the baseline, and the difference in visual acuity change compared to the untreated contralateral eye reached 15.3 letters.

Dion’s CEO explained, “The 9-month evaluation of the medium-dose group also fully met all primary endpoints regarding safety and tolerability,” adding, “Clear signs suggesting stabilization of best-corrected visual acuity were also observed, providing strong momentum for subsequent high-dose and commercialization clinical trials.”

Dr. Sally Temple’s Academic Authority… “Will Lead a Global Paradigm Shift”

Behind these clinical achievements lies the unparalleled research expertise of Dr. Sally Temple, co-founder and Chief Scientific Officer (CSO) of Luxa. Dr. Temple, CSO, is a world-renowned scholar who was the first to identify neural stem cells and served as president of the International Society for Stem Cell Research (ISSCR). She also delivered the opening keynote address at BIX 2026, presenting new horizons for the treatment of central nervous system and retinal diseases.

RPESC-RPE-4W, currently under development by Luxa, utilizes retinal pigment epithelium stem cells (RPESCs) isolated from donated adult eyes. The mechanism involves physically replacing damaged retinal pigment epithelium (RPE) cells with healthy ones. In recognition of this innovation, the treatment has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. FDA.

CEO Dion emphasized, “Drugs released by major global pharmaceutical companies have remained mere delaying agents that slow the rate of atrophy by suppressing the complement system, failing to satisfy patients’ desire for vision restoration,” adding, “We will completely transform the market landscape with a mechanism that regenerates the cells themselves.”

A representative from Y2 Solution said, “Since we have once again confirmed safety and efficacy through this Phase 1/2a clinical trial, we will accelerate preparations for global follow-up clinical trials using cryopreserved cells, as well as ongoing discussions regarding technology licensing (L/O).”