Beijing – based Chinagene Tech, a specialist in hereditary ophthalmopathy diagnosis and gene therapy, has announced obtaining Investigational New Drug (IND) approval from the US FDA for its ophthalmology gene therapy ZVS101e. The therapy is designed to treat Bietti’s crystalline dystrophy (BCD), a rare genetic eye disease. ZVS101e, a Category 1 drug, also received clinical trial approval in China last month.
Global Incidence and Impact of BCD
BCD has a global incidence rate of 1 in 67,000, making it more common in East Asia, where the incidence rate is 1 in 24,000. Most patients with BCD develop night blindness and vision loss in their 20s to 40s, and become legally blind by their 50s to 60s. The progressive nature of BCD underscores the need for effective treatments to address this debilitating condition.
ZVS101e’s Development and Clinical Trials
ZVS101e was awarded orphan drug designation (ODD) status in the US in August 2021, highlighting its potential to address an unmet medical need. The therapy has concluded the first exploratory investigator – initiated trial in BCD worldwide, demonstrating a good safety and efficacy profile. This milestone positions ZVS101e as a promising candidate for further clinical development and potential approval for the treatment of BCD.-Fineline Info & Tech
