FDA grants RMAT designation to RPESC-RPE-4W transplantation for AMD

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Luxa Biotechnology LLC announced that the US Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to RPESC-RPE-4W transplantation for the treatment of patients with dry age-related macular degeneration (dry AMD).

According to the press release¹ from the company, 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 4 weeks of differentiation (RPESC-RPE-4W) effectively replaces the native RPE cells lost during AMD disease progression. The RPESC program is currently in a Phase 1/2a clinical trial (NCT04627428).

Keith Dionne, PhD, CEO of Luxa, is quoted in the news release¹, on this milestone, saying, “This RMAT designation was granted following the FDA’s review of our compelling preliminary Phase 1 data and underscores the transformative potential of RPESC-RPE-4W in addressing the unmet needs of dry AMD patients who have lost or are losing their vision. We look forward to collaborating closely with the FDA to bring this potential paradigm-changing treatment to patients as efficiently as possible, restoring vision, and improving the quality of life for millions.”

To discuss this process and trial further, Jeffery Stern, MD, PhD, Luxa’s Chief Medical Officer, is scheduled to present safety, tolerability, and early efficacy data of RPESC-RPE-4W transplantation in the first cohort (N=6) of dry AMD patients at the 77th annual Wills Eye Conference which will be held in Philadelphia on March 6, 2025.¹

Reference:

1. Luxa Biotechnology Announces FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to RPESC-RPE-4W Transplantation for the Treatment of Dry Age-Related Macular Degeneration. Luxa Biotechnology LLC. February 18, 2025. Accessed February 21, 2025. https://www.businesswire.com/news/home/20250218104018/en/Luxa-Biotechnology-Announces-FDA-Regenerative-Medicine-Advanced-Therapy-RMAT-Designation-Granted-to-RPESC-RPE-4W-Transplantation-for-the-Treatment-of-Dry-Age-Related-Macular-Degeneration