Zhongyin Therapeutics' Gene Therapy ZVS101e Nominated for Breakthrough Therapy Designation to Halt Vision Loss in Bietti’s Crystalline Dystrophy

The official website of the Center for Drug Evaluation (CDE) of the China National Medical Products Administration recently announced,CHIGENOVO's ZVS101e Injection Application Proposed for Inclusion in Breakthrough Therapy Designation to Treat Crystalline Retinopathy.Public information shows,ZVS101e is a gene replacement therapy drug developed by CHIGENOVO. It has previously been granted Orphan Drug Designation by the U.S. FDA and has received IND approval in both China and the United States.. Currently, the drug has completed Phase 1/2 clinical trials and is about to enter Phase 3 clinical trials. In April this year, a Nature subsidiary published the clinical trial data of this drug.

Screenshot source: CDE official website

Crystalline Retinopathy (BCD) is a hereditary retinal degenerative disease. Patients typically experience night blindness between the ages of 20-30, and as the condition progresses, central vision gradually declines (an average decrease of 4.5 letters per year, equivalent to one line on the vision chart). Most patients become legally blind by around the age of 40 due to severe vision loss and narrowing of the visual field. BCD exhibits autosomal recessive inheritance and is caused by mutations in the CYP4V2 gene. The CYP4V2 gene encodes an ω3-polyunsaturated medium-chain fatty acid hydroxylase, which is highly expressed in retinal pigment epithelial cells (RPE) and plays a role in fatty acid metabolism. Mutations in the CYP4V2 gene lead to abnormal lipid metabolism in the RPE, resulting in photoreceptor degeneration and apoptosis. Currently,There is currently no effective treatment for BCD available worldwide., patients have urgent clinical needs.

ZVS101e Injection is a gene replacement therapy drug developed by CHIGENOVO, intended for the treatment of patients with crystalline retinal degeneration carrying CYP4V2 gene mutations.According to publicly available data from CHIGENOVO, the therapeutic vector contained in ZVS101e has a specific tropism for retinal cells. After being injected into the patient's subretinal space, it can efficiently infect the retinal pigment epithelial (RPE) cells of the retina and specifically express the CYP4V2 protein in RPE cells, compensating for the loss of protein function caused by gene mutations, therebyEffectively restores retinal function in BCD patients。

Author｜Yang Xixia

Editor｜Jiang Yun, Jia Ting

Operator | Zhu Ying

Illustration｜Visual China

Statement: Original content by Jian Shi Ju, please do not reprint without permission.

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