An Clinical Study Evaluating the Safety, Tolerability, and efficAcy of HG005 in StaRgardT Disease

Inclusion criteria

• Patient ≥ 6 and ≤17 years at the time of signing informed consent, with clinical diagnosis of Stargardt disease;
• At least one ABCA4 allele on each chromosome;
• Both eyes must have well-defined macular atrophic lesions consistent with the diagnosis of Stargardt macular dystrophy.
• Meet visual acuity criteria based on ETDRS letter chart
• Subject must agree to contraception during the study.
• Acceptable hematology, clinical chemistry, urine laboratory, and protocol required eye examination.

Exclusion criteria

• Presence of active intraocular inflammation or uveitis history in either eye;
• Presence of ocular or periocular infection history in either eye within 2 weeks prior to selection;
• History or presence of corneal dystrophy in the study eye;
• History of HIV or hepatitis A, B, or C infection;
• Previous treatment with any gene therapy or cell therapy (e.g., stem cell transplantation);
• Additional intraocular surgery in study eye 3 months prior to baseline visit;
• Participation in an oral therapeutic STGD clinical trial within 3 months (or within 5 half-lives after last dose) prior to Screening
• Any concomitant treatment that, in the opinion of the investigator, might interfere with the surgical procedure or healing process of the eye
• Any other conditions that would not allow the potential subject to complete follow-up examinations during the study and would, in the opinion of the investigator, make the potential subject unsuitable for the study.