Zhongyin Biotech's Recombinant AAV Gene Therapy ZVS101e Receives FDA RMAT Designation for Bietti Crystalline Dystrophy

Jul 20, 2024 07:19 CST Updated 07:20

CHIGENOVO

Clinical Genetic Diagnosis, Prevention, and Gene Therapy Drug Development for Hereditary Eye Diseases

July 20, 2024

eMedClub News

Recently, CHIGENOVO Co., Ltd. announced that its self-developed ZVS101e injection has received the Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. FDA, intended forTreatment of Bietti Crystalline Dystrophy (BCD) Caused by CYP4V2 Gene Mutation。

The press release noted that this is another milestone event achieved by CHIGENOVO, following the inclusion of ZVS101e Injection in the CDE Breakthrough Therapy Drug Program on June 28, 2024. The acquisition of RMAT designation further confirms the significant potential of ZVS101e Injection in treating and slowing the progression of BCD.

BCD is a special type of retinitis pigmentosa, with typical changes characterized by yellowish-white crystalline substances deposited on the retina. Clinically, it manifests as progressive vision loss and visual field constriction. Patients typically become blind around the age of 40, and currently, there is no effective treatment available. In China, there are approximately 60,000 to 100,000 BCD patients, making it one of the significant causes of irreversible bilateral blindness in the working-age population and a key focus of blindness prevention efforts. BCD is caused by mutations in the CYP4V2 gene, which follows an autosomal recessive inheritance pattern. These mutations result in the loss of function of the encoded protein, making it suitable for gene replacement therapy.

ZVS101e is a recombinant AAV vector containing the human CYP4V2 gene. After subretinal administration of ZVS101e injection, it can efficiently infect retinal cells and specifically express the CYP4V2 protein in the cells, compensating for the loss of protein function caused by gene mutations, thereby effectively treating retinal function in BCD patients.This alternative therapeutic drug will be applicable to all BCD patients carrying CYP4V2 gene mutations.。

Source: CHIGENOVO Official WeChat Account

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