China-based Yiming (Beijing) Cell Biotechnology Co., Ltd (also known as Ubrigene) and its partner Shanghai Vitalgen Biomed Co., Ltd have received tacit clinical trial approval from the Center for Drug Evaluation (CDE) for their in-house developed gene therapy drug VGR-R01. This marks the second gene therapy from the company to gain clinical approval, following VGB-R04 in July this year.
VGR-R01: A Promising Treatment for BCD
VGR-R01 is a gene replacement therapy designed for patients with Bietti crystalline dystrophy (BCD), a rare genetic disorder caused by mutations in the CYP4V2 gene. The CYP4V2 protein, a member of the P450 enzyme family, is highly expressed in retinal pigment epithelial cells (RPE) and is involved in fat metabolism. VGR-R01 works by delivering a functional copy of the CYP4V2 gene to RPE cells via an AAV capsid protein. Once in the nucleus, the gene expression cassette expresses the CYP4V2 protein, restoring fatty acid hydroxylase activity and potentially preventing or improving structural and functional damage to RPE cells, photoreceptors, and choroids. This mechanism aims to correct visual impairment, protect residual vision, and delay visual deterioration.
The Challenge of BCD
Bietti crystalline dystrophy (BCD) is an autosomal recessive, progressive retinal degeneration disease that affects individuals worldwide, with higher prevalence in China, Japan, and South Korea. Most patients develop night blindness and vision loss between the ages of 20 and 40, and become legally blind by their 50s or 60s. Diagnosis is based on identifying biallelic pathogenic variants in the CYP4V2 gene, but effective treatments have been lacking.
Clinical Progress and Safety Profile
An early clinical study for VGR-R01 was initiated at Beijing’s Tongren Hospital, with preliminary results indicating a favorable safety profile. This approval from the CDE represents a significant step forward in the development of a potential treatment for BCD, offering hope for patients who currently have limited therapeutic options.-Fineline Info & Tech
