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 (Luxa), a clinical-stage biotechnology company developing a novel adult retinal pigment epithelial stem cell (RPESC) therapy for dry age-related macular degeneration (AMD), today announced that the U.S. 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).
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 replaces the native RPE cells lost during AMD disease progression. The RPESC program is currently in a Phase 1/2a clinical trial (NCT04627428).
Established under the 21st Century Cures Act, RMAT designation is designed to accelerate the development and review of promising regenerative medicine therapies that treat serious or life-threatening diseases. To obtain this designation, therapies must have demonstrated preliminary clinical evidence that the treatment has the potential to address unmet medical needs for such diseases. RMAT designation provides significant benefits including increased early interactions with FDA leadership to guide and accelerate clinical development, the potential for accelerated approval, and priority review of a product’s biologics license application (BLA).
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