HORA-RLPB1

HORA-RLPB1 is an investigational adeno-associated virus (AAV)-mediated gene replacement therapy being developed by Horama to treat inherited retinal dystrophies caused by biallelic mutations in the RLBP1 gene, including RLBP1-associated retinitis pigmentosa and related phenotypes such as Bothnia-type retinal dystrophy and retinitis punctata albescens.[1][4][17] The product uses a recombinant AAV vector to deliver a functional copy of the human RLBP1 coding sequence to retinal pigment epithelium and Müller cells via subretinal injection, with the aim of restoring expression of cellular retinaldehyde-binding protein (CRALBP), normalizing the visual cycle, improving dark adaptation, and reducing disease-associated retinal deposits.[3][7][11] HORA-RLPB1 has received orphan drug designation in the United States and Europe and is in early-stage clinical development as part of Horama’s pipeline of rAAV-based ocular gene therapies for rare inherited retinal diseases.[1][4][17]