Chinagene Tech has had ZVS101e, a recombinant adeno-associated virus (AAV) gene augmentation therapy delivering a functional CYP4V2 copy, accepted for priority review by its China homeland's Centre for Drug Evaluation (CDE) for Bietti's crystalline dystrophy (BCD) in patients with biallelic CYP4V2 mutations.

A randomised, controlled Phase III trial (31 treated with a single subretinal injection of 7.5×10¹⁰ vg/eye, 32 untreated controls) met its primary endpoint at week 24, with 35% of treated patients gaining at least 15 Best Corrected Visual Acuity (BCVA) letters versus 3% of controls (p=0.001), and a mean BCVA improvement of 11.5 letters versus 1.5 letters (between-group difference 10 letters, p<0.001). Secondary endpoints including Multiluminance Mobility Test and full-field stimulus threshold favoured treatment, with no serious adverse events in the active arm.

BCD is a recessive retinal dystrophy causing night blindness, progressive vision loss, and typically blindness by age 40, with an estimated 60,000 to 140,000 patients in China and no approved disease-modifying therapy globally. ZVS101e holds US orphan drug designation (2021) and RMAT status (2024, the first Chinese ocular gene therapy to receive it), plus China NMPA Breakthrough Therapy designation (2024), giving it a clear regulatory fast-track profile.