RBIO-101

RBIO-101 is an investigational gene therapy being developed by ReflectionBio for the treatment of Bietti's Crystalline Dystrophy (BCD). BCD is a rare, autosomal recessive retinal dystrophy caused by mutations in the *CYP4V2* gene, which leads to the accumulation of lipid crystals in the retina and progressive vision loss. RBIO-101 utilizes a recombinant adeno-associated virus (AAV) vector to deliver a functional copy of the *CYP4V2* gene to retinal cells via a non-editing gene transfer approach. By restoring CYP4V2 protein expression, the therapy aims to normalize lipid metabolism in the eye and slow or prevent the progression of retinal degeneration. The program is currently in the IND-enabling stage.