Milestone of BCD gene therapy: Vitalgen VGR-R01 IND application accepted


On August 9, 2022, Shanghai Vitalgen BioPharma Co., Ltd. (hereinafter referred to as “Vitalgen”) announced that the IND application of its self-developed VGR-R01 injection (rAAV2/8-CYP4V2) was accepted by Center for Drug Evaluation (CDE), National Medical Products Administration. VGR-R01 is a gene therapy product independently developed by Vitalgen for patients with Bietti crystalline dystrophy (BCD) caused by CYP4V2 gene mutation. It is also the first therapeutic drug for BCD with IND application. Its mechanism of action is gene replacement.

Figure 1. IND application of VGR-R01 injection accepted

“BCD is an inherited retinal disease relatively common in China but rare in Western countries.” said scientific founder of Vitalgen Researcher Li Wei, “In view of this, for BCD, a rare disease that has received little attention, we have a sense of mission to provide medicine for Chinese patients. During the four years of cooperation with the team lead by Professor Wei Wenbin of Beijing Tongren Hospital Affiliated to Capital Medical University, we continuously accumulated preclinical experience in BCD, and consolidated the foundation for clinical translation. The IND application of VGR-R01 is an important milestone for patients with BCD; it is also a gratifying and important moment for practitioners in the pharmaceutical industry. We look forward to using gene therapy to bring cure opportunities to more patients.”

About Bietti crystalline dystrophy
Bietti crystalline dystrophy (BCD), also known as Bietti crystalline retinopathy, is an autosomal recessive, progressive retinal degenerative disease. The BCD mutant gene is CYP4V2 located at 4q35, encoding a protease involved in lipid metabolism, and is caused by homozygous or compound heterozygous mutations. Most patients with BCD develop symptoms such as night blindness and decreased visual acuity in their 20s and 40s, and develop legal blindness in their 50s and 60s. Although it is a serious blinding genetic eye disease, compared with some widely concerned “star genetic diseases”, there are few basic and clinical studies on BCD. Eighty years after Professor Bietti first observed clinically and named the disease, BCD remains incurable.

About VGR-R01
VGR-R01 is a gene therapy product for patients with BCD caused by CYP4V2 gene mutation. CYP4V2 protein is a member of the P450 enzyme family. It is highly expressed in retinal pigment epithelium (RPE), with fatty acid hydroxylase activity, and is related to lipid metabolism. Mechanism of action of VGR-R01 is gene replacement. It mediates the expression of CYP4V2 protein in RPE cells by replenishing the correct copy of the CYP4V2 gene. VGR-R01 aims to prevent or improve the structural and/or functional damage of RPE cells, photoreceptor cells and choroid by correcting the fatty acid metabolism disorder in the retina of patients, so as to correct visual impairment, protect residual visual function, or delay vision deterioration.

Figure 2. Mechanism of action of VGR-R01

Early clinical study of VGR-R01
“An Early Clinical Study of VGR-R01 in the Treatment of Bietti Crystalline Dystrophy (BCD)” is ongoing at Beijing Tongren Hospital Affiliated to Capital Medical University. This study has been approved by the Clinical Study Ethics Committee of Beijing Tongren Hospital (Ethics Approval Number: TREC2022-KY010).

Principal Investigator

Professor Wei Wenbin, Professor Zhao Xiuli


Mr. Zhang 13552757069, Dr. Wang (WeChat 13683178767)

Major inclusion criteria

1. Male or female with age ≥ 18 years old and < 80 years old.

2. Patients clinically diagnosed with BCD, with molecular diagnosis confirming CYP4V2 biallelic mutation;

3. Patients with best corrected visual acuity of the target eye ≤0.1;

4. Patients who must agree to use reliable contraceptions until at least one year after VGR-R01 injection;

5. Patients without serious systemic diseases.