Stargardt disease type 1 (STGD1) is a rare genetic eye condition that causes progressive vision loss, often beginning in childhood or adolescence. It is the most common form of inherited macular degeneration and can lead to legal blindness. STGD1 is caused by mutations in the ABCA4 gene, which normally helps clear waste from the photoreceptor cells in the retina. When ABCA4 gene doesn't function properly, toxic substances like A2E accumulate and damage the retinal pigment epithelium (RPE), leading to vision loss. There are currently no approved treatments for STGD1. HG005 is an investigational gene therapy designed to deliver a healthy copy of the ABCA4 gene to the retina. Because the gene is too large to fit into a single AAV (adeno-associated virus) vector, HG005 used two AAV vectors that work together in retinal cells to produce the full-length, functional ABCA4 protein. The goal of HG005 is to restore normal waste removal, protect retinal cells from further damage, and slow or stop vision loss.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
6
Once subretinal injection; The duration of the study is about 52 weeks for each subject, including a 4 weeks screening period, enrollment visit, treatment visit, and 56 weeks follow-up period.
Eye & ENT Hospital of Fudan University
Shanghai, China
RECRUITINGIncidence and severity of systemic adverse events
Number of adverse events (AEs), serious adverse events (SAEs), and dose-limiting toxicities (DLTs)
Time frame: 26 & 52 weeks
Change of study eye versus control eye in Low Luminance Visual Acuity(LLVA)
Time frame: 52 weeks
Change of study eye versus control eye in Fundus Autofluorescence (FAF)
Time frame: 52 weeks
Change of study eye versus control eye in Optical Coherence Tomography (OCT)
Time frame: 52 weeks
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