VGR-R01

VGR-R01 is an investigational gene therapy developed by Shanghai Vitalgen BioPharma for the treatment of Bietti crystalline dystrophy (BCD), a rare inherited retinal disorder also known as crystalline retinitis pigmentosa. The therapy utilizes a recombinant adeno-associated virus (AAV) vector to deliver a functional copy of the human CYP4V2 gene to retinal cells via subretinal injection. By replacing the defective or missing Cytochrome P450 family 4 subfamily V member 2 (CYP4V2) protein in affected individuals, VGR-R01 aims to restore normal cellular function and slow or halt disease progression. Clinical studies have demonstrated that subretinal administration of VGR-R01 is generally well-tolerated and may result in improvements in visual function[1][2][5][6][7].