Chigenovo Submits NDA for ZVS101e, a Gene Therapy for Bietti Crystalline Dystrophy, to China’s CDE

This is followingVitalgen's VGR-R01,The second product for market approval applicationAAV Gene Therapy for Bietti Crystalline Dystrophy (BCD).

ZVS101e is a drug developed for the treatment ofAAV Gene Therapy for Bietti Crystalline Dystrophy (BCD).

BCD consists ofCaused by CYP4V2 mutations,ZVS101e adopts a gene replacement therapy strategy, constructingrAAV2/8-hCYP4V2 vector, delivered into retinal pigment epithelial cells via subretinal ultra-microinjection, enabling the cells to regain normalCYP4V2The gene can sustainably express CYP4V2 protease with physiological activity.

ZVS101e is BeijingThe gene therapy product, jointly developed by Professor Yang Liping’s research group from the Department of Ophthalmology at Peking University Third Hospital and CHIGENOVO, completed its intellectual property transfer in March 2021.

It is worth noting that,ZVS101e atIn December 2022, it received approval for Phase I/II clinical trials in China and the United States, respectively, becoming the world’s first BCD gene therapy product to enter clinical development.

As of now, ZVS101e has completed a total of 4clinical trials, including 2 investigator-initiated trials (IITs) and 2 registrations, with a total enrollment of nearly 100 patients.

ZVS101eValidated through multiple clinical trials,90%The vision of the above patients improved, with an average improvement of12.9individualETDRSLetters, among which50%Significant Improvement in Patients15more letters, indicating significant improvement in visual acuity; adverse events were1-2Grade, self-healing without treatment or cured after hormone therapy.

Currently,ZVS101e has been included by the CDEInclusion in the List of Breakthrough Therapy Designations means that ZVS101e has simultaneously qualified for “Priority Review,” significantly shortening its review and approval timeline and accelerating its market launch.

Furthermore,ZVS101e alsoGranted Orphan Drug Designation and Regenerative Medicine Advanced Therapy (RMAT) designation by the FDA, as well as Priority Medicines (PRIME) designation by the EMA。

In fact, there are already two products targetingBCD's AAV gene therapy has submitted a marketing application to the China National Medical Products Administration.

• On June 23, 2026, the CDE accepted the application submitted by Taichang Biologics, a subsidiary of Vitalgen, forVGR-R01 Marketing Authorization Application.

• August 25, 2026,CDE accepted the applicationCHIGENOVO's filingPiperacillin InjectionMarketing Authorization Application.

If these two gene therapies are successfully approved for market launch in the future, they will enter into direct competition.

According to reports, the number of BCD patients in China is estimated atBetween 60,000 and 140,000, with relatively few overseas patients. CurrentlyBCDThere is no effective treatment available worldwide; Vitalgen'sVGR-R01CHIGENOVO'sZVS101e is poised to provide new treatment options for patients with BCD.

However, judging from the current state of the global market, gene therapy remains in an awkward position of being “acclaimed by critics but not by consumers,” with the exception of Novartis’s SMA gene therapyApart from Zolgensma, sales of other commercial AAV gene therapy products have all fallen short of expectations.

The gene therapy market in China may be more pessimistic; Belief Medicine's gene therapy for hemophilia BXinjiuning asChina's First AAV Gene Therapy Product: No Commercial Patients One Year After Launch, Takeda AlsoRecently terminated the commercialization collaboration with Belief Medicine regarding Xinjiuning.

Compared toXinjiuning and similar productsSystemic gene therapy, with ophthalmic gene therapy priced lower, whileBCD, as a currently untreatable indication with lower prices andUrgentDisease Treatment NeedsThis may drive the scale-up of ophthalmic gene therapies.