Sichuan University West China Hospital Completes First-in-Human Dosing in Global First AAV Gene Therapy Trial for Stargardt Disease Type 1 with JWK006 Injection
Jinweike Biotechnology
AAV Gene Therapy New Drug Developer
Clinical Study on Gene Therapy for Stargardt Disease Type 1
Recently, the team led by Professor Lu Fang from the Ophthalmology Department of West China Hospital, Sichuan University, successfully completed the first patient dosing in the clinical study titled "Safety and Efficacy of Single Unilateral Intravitreal Administration of JWK006 Injection in Type 1 Stargardt Disease Patients." This is also the world's first AAV gene therapy clinical trial for Type 1 Stargardt disease. The study is a single-arm, single-dose, dose-escalation trial evaluating the safety and efficacy of the gene therapy drug JWK006 Injection after administration. It has received approval from the Biomedical Ethics Review Committee of West China Hospital, Sichuan University.
Professor Lu Fang from the Ophthalmology Department of West China Hospital, Sichuan University, stated: "The entire surgical process went very smoothly. Our team made thorough preoperative preparations, and no ocular or systemic adverse reactions were observed after the administration of the drug. The pathogenic gene ABCA4 for Type 1 Stargardt disease exceeds the capacity limitations of the commonly used AAV gene vector in ophthalmology. In this clinical research project involving JWK006 injection, we utilized a dual AAV gene therapy technology independently designed and developed by our team, marking a breakthrough attempt in the field of gene therapy for hereditary retinal diseases. We will closely monitor the patient's post-treatment condition and share relevant information in a timely manner. We are also continuing with the screening of subjects and strictly following the clinical trial protocol for enrollment. We believe more patients will join our clinical trial."
About Stargardt Disease Type 1
Stargardt Disease Type 1, also known as Fundus Flavimaculatus, is the most common macular dystrophy in adolescents and can lead to severe, irreversible bilateral vision loss. Its prevalence is approximately 1/10,000 to 1/8,000, and the disease-causing gene is the ABCA4 gene. Mutations in this gene impede the transport and recycling of retinoids, causing their accumulation and ultimately leading to the formation of toxic retinoid dimers (A2E), which result in degeneration of the retinal pigment epithelium and photoreceptor cells, causing vision loss or even blindness in patients. The ABCA4 gene sequence is 6.8kb in size, and conventional adeno-associated virus (AAV) vectors cannot accommodate the ABCA4 gene sequence due to their limited capacity (<4.7kb). As an irreversible rare eye disease, there is currently no effective treatment for Stargardt Disease Type 1. Current research focuses on gene therapy, small molecule treatments (drugs, dietary supplements), cell replacement therapy (RPE cell transplantation), and gene editing, but there are currently no effective and safe treatments available for clinical use.
About JWK006 Injection
JWK006 Injection is an AAV gene therapy product jointly developed by the team of Academician Wei Yuzhong and Researcher Yang Yang from the Ophthalmology Department of West China Hospital, Sichuan University, the National Key Laboratory of Biotherapy, and Chengdu Jinweike Biotechnology Co., Ltd. The preliminary research findings were published as a cover article in the journal *Human Gene Therapy*. JWK006 Injection utilizes a self-developed dual-AAV vector technology to efficiently restore the expression of full-length ABCA4 protein in retinal cells, thereby improving patients' retinal structure and function. Currently, this clinical trial is recruiting patients nationwide in China. Patients and their families are welcome to consult at the Ophthalmology Department of West China Hospital (Contact: Teacher Wu, Phone: 18284506280).
Manuscript: Liu Fanfei Proofreading: Lu Fang
Editor: Liu Yilin Responsible Editor: Ma Ke
E.N.D
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Statement: The content is sourced fromOphthalmology Department, West China Hospital, Sichuan University. This article aims for knowledge sharing. All content is for reference only and does not constitute any advice.