Beijing-based ChinaGene Technology Co., Ltd. has announced that it has received tacit clinical approval from the Center for Drug Evaluation (CDE) for its ophthalmic gene therapy ZVS101e, designed to treat retinitis pigmentosa (RP) patients with the RHO-R135W (RHO c.403C>T) mutation. This follows a tacit trial approval granted in the United States in December of last year.
ZVS203e, another candidate developed using CRISPR/Cas9 technology, received orphan drug designation (ODD) status in the U.S. in July 2023 and commenced an investigator-initiated trial (ITT) in China in September 2023.
Retinitis pigmentosa is a progressive genetic disorder characterized by the gradual loss of retinal photoreceptors and retinal pigment epithelial cells, affecting approximately 1 in 3,500 people worldwide. It is a leading cause of irreversible blindness in working-age individuals, with RHO gene mutations being the most prevalent in autosomal dominant RP. Initial symptoms typically include night blindness, with a progressive narrowing of the visual field and loss of central vision, often leading to legal blindness between the ages of 20 and 65. Currently, no targeted therapies exist globally for this condition.- Flcube.com
