JWK006

JWK006 is an investigational adeno-associated virus (AAV)-based gene therapy developed for the treatment of Stargardt disease type 1 (STGD1). STGD1 is an autosomal recessive retinal dystrophy caused by mutations in the *ABCA4* gene, which encodes a transmembrane flippase protein essential for the visual cycle. Deficiency in ABCA4 leads to the accumulation of toxic bisretinoids (lipofuscin) in the retinal pigment epithelium (RPE), resulting in progressive photoreceptor degeneration and central vision loss. JWK006 is designed to deliver a functional copy of the *ABCA4* gene to the retina via subretinal injection, aiming to restore protein expression and halt the progression of the disease. The program is currently being evaluated in Phase I/II clinical trials in China.

Other names

JWK006 injectionJWK-006 injectionJWK 006 injectionJWK006注射液