Beijing-based Chinagene Tech, a specialist in hereditary ophthalmopathy diagnosis and gene therapy, has announced the enrollment and dosing of the first subject in a Phase I/II clinical study for its ophthalmology gene therapy ZVS101e in Tianjin. This study marks the first potentially regulatory clinical trial for a gene therapy targeting Bietti’s crystalline dystrophy (BCD) on a global scale.
ZVS101e: A Promising Gene Therapy Approach
ZVS101e is a recombinant AAV vector that contains the human CYP4V2 gene. Following subretinal administration, it can effectively infect retinal cells and specifically express the CYP4V2 protein within these cells. This mechanism compensates for the loss of protein function caused by gene mutations, thereby playing a crucial role in treating the retinal function of BCD patients. ZVS101e is designed to be applicable to all BCD patients with CYP4V2 gene mutations.
Understanding Bietti’s Crystalline Dystrophy
BCD is a form of retinitis pigmentosa characterized by the deposition of yellow-white glittering crystalline substances in the retina. Clinically, it manifests as progressive visual loss and a narrowing of the field of vision. Patients typically become blind around the age of 40, and currently, there is no effective treatment available. In China, there are an estimated 60,000 to 100,000 BCD patients, making it a significant cause of irreversible blindness in the working-age population and a focal point for blindness prevention and treatment efforts. BCD is caused by mutations in the CYP4V2 gene, which is usually recessive. These mutations lead to the loss of function of the protein encoded by the gene, making it an ideal candidate for gene replacement therapy.-Fineline Info & Tech
