Luxa Biotechnology Awarded $4 Million CIRM Grant to Support Clinical Trial of Adult RPESC-RPE-4W Therapy for Dry Age-related Macular Degeneration

Luxa Biotechnology (LuxaBio), a joint venture between Y2 Solution Co. Ltd, Seoul, South Korea and the Neural Stem Cell Institute (NSCI) in Rensselaer, New York, today announced receipt of a $4 million grant from the California Institute for Regeneration Medicine (CIRM) to support the ongoing Phase 1/2a study (NCT04627428) of RPESC-RPE-4W transplantation for the treatment of dry age-related macular degeneration (dry AMD). The funding will accelerate manufacturing and clinical activities based in California.

RPESC-RPE-4W is a cell product derived from adult retinal pigment epithelial stem cells (RPESC) that generate retinal pigmented epithelium (RPE) cell progeny (RPESC-RPE). Transplantation of progenitor-stage RPESC-RPE cells obtained after four weeks of differentiation (RPESC-RPE-4W) effectively replace the native RPE cells lost during AMD disease progression.

"We are honored to have CIRM recognize the potential of our unique technology utilizing precursor-stage RPE cells. Replacement of lost RPE cells with RPESC-RPE-4W aims to restore vision loss in patients with dry AMD and offers the potential to improve the quality of life," said Jeffrey Stern, PhD, MD, co-founder of the NSCI.

Dry AMD is a common eye disorder in people over 50. It is caused by the deterioration of the cells of the macula, as retinal cells die off and are not renewed. The condition results in reduced central vision and there are currently no treatments available to reverse the damage.

"RPESC-RPE-4W therapy represents a potential breakthrough in the treatment of dry AMD," said Dr. Abla Creasey, PhD, Vice President of Therapeutics Development at CIRM. "We are impressed with the stem cell-based replacement approach developed by Luxa Biotechnology and delighted to support their groundbreaking efforts to restore vision and hope to patients with dry AMD."

Dr. Stern will present an update on the RPESC-RPE-4W trial on May 3, 2024, at the Foundation Fighting Blindness / Casey Eye Center Symposium, taking place in Seattle, Washington.

About Luxa Biotechnology

Luxa Biotechnology (LuxaBio) is a clinical-stage biotechnology company developing a novel adult RPE stem cell (RPESC) therapy for dry AMD. The proprietary adult RPESC-derived RPE stem cell product was developed at and licensed from the Neural Stem Cell Institute (NSCI). LuxaBio is a partnership between the NSCI research institute and Y2 Solution, a Korean company, to evaluate RPESC in a clinical trial to test the safety and efficacy of RPE progenitor stage progeny as a therapy for dry AMD. LuxaBio maintains a robust research program at NSCI to develop the RPESC as an effective, commercially viable cell product. The Phase 1/2a clinical trial of RPESC-RPE-4W for the Treatment of Dry Age-related Macular Degeneration includes the Cedars Sinai Biomanufacturing Center, Emmes, the University of Michigan Kellogg Eye Center, the National Eye Institute For more information, please contact [email protected]

About NSCI

In 2005, Dr. Sally Temple and Dr. Jeffrey Stern founded the Regenerative Research Foundation and the Neural Stem Cell Institute (NSCI), the first independent stem cell research institute in the country. Rooted in discovery research, the administratively lean NSCI effectively advances laboratory discoveries to clinical application of regenerative stem cell therapies that address diseases of the central nervous system. NSCI is a non-profit discovery research Institute led by Dr. Sally Temple, the scientific director. The staff of 40 research staff is dedicated to advancing the understanding of neural stem cell biology work in cutting edge laboratories equipped for stem cell research. The retinal pigment epithelial stem cell (RPESC) was discovered at NSCI where in-depth characterization of this unique stem cell is ongoing. In 2022, NSCI partnered with Y2 Solution to create Luxa Biotechnology to advance the RPESC as therapy for dry age-related macular degeneration, in collaboration with the NIH National Eye Institute, Cedars-Sinai Biomanufacturing Center, Emmes Corporation, and the University of Michigan Kellogg Eye Center.

About CIRM

At CIRM, we never forget that we were created by the people of California to accelerate stem cell treatments to patients with unmet medical needs, and act with a sense of urgency to succeed in that mission. To meet this challenge, our team of highly trained and experienced professionals actively partners with both academia and industry in a hands-on, entrepreneurial environment to fast track the development of today's most promising stem cell technologies. With $5.5 billion in funding and more than 150 active stem cell programs in our portfolio, CIRM is one of the world's largest institutions dedicated to helping people by bringing the future of cellular medicine closer to reality. For more information go to www.cirm.ca.gov