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Correcting visual impairment: VGR-R01 project IND application accepted by Center for Drug Evaluation

A VGR-R01 project IND application from uBriGene partner Vitalgen has been accepted by the CDE, aiming to correct visual impairment

On August 9th, 2022, a partner of uBriGene Biosciences Inc, Shanghai Vitalgen Bio-Pharma Co. (hereinafter referred to as " Vitalgen") announced that its IND application for VGR-R01 injectant (rAAV2/8-CYP4V2) has been accepted by the Center for Drug Evaluation (CDE) of the State Drug Administration. VGR-R01 is a gene therapy product developed by Vitalgen for the treatment of Bietti’s crystalline dystrophy (BCD) caused by CYP4V2 gene mutation. This is the first gene therapeutic drug application in the world for BCD for clinical trials employing target gene. The working principle of VGR-R01 is using target gene substitution.

Figure 1. Notice of acceptance of VGR-R01 injectant IND application

"BCD is an inherited retinal disease that is relatively prevalent in China but rare in Western countries,” said Wei Li, founder and senior researcher of Vitalgen. "With this in mind, we have a greater sense of mission to develop a medicine for those patients suffering from BCD, a rare disease that has received little attention. In the past four years over which we cooperated with Prof. Wei Wenbin's team at Beijing Tongren Hospital of Capital Medical University, we have accumulated data in preclinical studies of BCD and solidified the foundation to translate research findings to the clinic application. For BCD patients, the IND filing of VGR-R01 is an important milestone. For those of us who are in the pharmaceutical industry, it is also a gratifying moment, and we look forward to using this specific gene therapy method to help more BCD patients", Mr. Li added.

About Bietti’s crystalline dystrophy, BCD

Bietti crystalline dystrophy (BCD), also known as Bietti’s crystalline retinopathy, is an autosomal recessive ocular disease which involves the progressive degradation of the retina. The mutation is located in the CYP4V2 gene on chromosome 4q35. CYP4V2 enzyme has been shown to play a role in fatty acid and steroid metabolism. The disease is caused by homozygous or complex heterozygous mutations. Most patients with BCD develop night blindness and vision loss between their 20s and 40s. They then progress to legal blindness between their 50s and 60s. Although it is a serious genetic eye disease, basic and clinical research on BCD is relatively scarce compared to some of more widely focused on genetic diseases. There is still no cure for BCD, even after 80 years since its initial discovery and naming by professor Bietti.

About VGR-R01

VGR-R01 is a gene therapy product for patients with BCD caused by mutations in the CYP4V2 gene. The CYP4V2 protein is a member of the P450 enzyme family that is highly expressed in the retinal pigment epithelium (RPE). It has fatty acid hydroxylase activity and is associated with lipid metabolism. The working mechanism of VGR-R01 is gene substitution therapy. The mutated CYP4V2 gene copy number is complemented by the VGR-R01 product, which enables the cell to express the CYP4V2 protein at a proper level.

Through correcting fatty acid metabolism in patients’ retina, VGR-R01 prevents and improves the structural and/or functional damage to RPE cells, photoreceptor cells and choroidal cells. VGR-R01 aims to correct visual impairment, protect residual visual function, or delay deterioration of visual acuity.

Figure 2. VGR-R01 working mechanism diagram

Early Clinical Study of VGR-R01

Beijing Tongren Hospital of Capital Medical University is conducting an early clinical study of VGR-R01 for the treatment of BCD. This study has been approved by the Clinical Trial Ethics Committee of Beijing Tongren Hospital (Ethics Approval No. TREC2022-KY010).

About Vitalgen

Founded in March 2020 in Shanghai, China, Shanghai Vitalgen Biopharma is dedicated to turning cutting-edge gene and cell therapy technologies into clinically accessible therapies for the benefit of more patients. Vitalgen has the autonomous intellectual property rights of ViVec® AAV vector screening platform, ViLNP® lipid nanoparticle delivery technology platform, ViCas® CRISPR gene editing technology platform, and ViHiYi® AAV high-yield technology platform. Using techniques including gene substitution, gene regulation, and gene editing, Vitalgen developed therapeutic treatments for central nervous system diseases, metabolic and hematologic diseases, and tumors. The company also has a diversified pipeline of potential First-in-class products. The company has several pipelines in clinical development, primary on rare pediatric diseases, hemophilia, and ophthalmic diseases. The available clinical data shows that the gene drugs have proven to be safe and effective.

At present, Vitalgen has received multiple rounds of investment from well-known funds such as IDG Capital, Loyal Valley Capital and Hillhouse Capital. It has built a 2,500 square meters gene therapy R&D operation center and a 3,000 square meters gene therapy pilot production facility in Zhangjiang Hi-Tech Park and Lingang New Area respectively. It’s GMP line is also under construction.

About uBriGene

uBriGene Biosciences Inc. has always been committed to the development and application of gene therapy technologies, as well as providing comprehensive CDMO solutions for the gene therapy industry. uBriGene has established GMP production bases of over 10,000 square meters in Suzhou and Jinan, China. It also has established CMC R&D centers in North America (both the USA and Canada). The CMC R&D centers abroad will expand uBriGene’s GMPs production capability, as well as provide more potential and forward perspective for technological innovation and foundational R&D. Currently, uBriGene has dozens of production lines, including prokaryotic fermentation scales of 10L-50L-200L and eukaryotic cell culture scales of 50L-200L-500L-2000L, as well as advanced purification processes with AAV virus yields as high as 1E14VG/L. uBriGene is fully committed to supporting gene therapy companies.

uBriGene’s mission is to make lives healthier and make the world better.

As a CDMO company and one of the core players in the new drug R&D industry chain, uBriGene will continue to work hard in the CGT field to help new gene therapeutic companies accelerate their R&D, manufacturing, and clinical processes in the context of the increasingly clear Marketing Authorization Holder (MAH) system and specialized division of labor. The company continues to contribute to the development of the biopharmaceutical industry by assisting new drug companies in accelerating R&D and manufacturing, IND applications, clinical trials, and other aspects.

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