safety and effects of a single intravitreal injection of vmco-010 optogenetic therapy in subjects with stargardt disease

Conditions: Stargardt Disease

Summary: the purpose of the study is to evaluate the safety and effects of a single intravitreal injection of virally-carried multi-characteristic opsin (vmco-010) in subjects with stargardt disease

this multicenter open label study will evaluate single dose level of vmco-010 in up to 6 subjects with stargardt's disease. subjects with documented clinical diagnosis of stargardt disease (classic fleck phenotype and/or well-demarcated sub-foveal area of significantly reduced autofluorescence as imaged by faf), or genetic diagnosis with pathogenic variants in abca4, elovl4, or prom 1. all subjects will continue to be assessed for 48 weeks following treatment with vmco-010.

Eligibility criteria:
inclusion criteria: 1. ≥16 years of age 2. able to comprehend and give informed consent. 3. able to comply with testing and all protocol tests. 4. documented clinical diagnosis of stargardt disease (classic fleck phenotype and/or well-demarcated sub-foveal area of significantly reduced autofluorescence as imaged by faf), or genetic diagnosis with pathogenic variants in abca4, elovl4, or prom1 5. in the study eye: etdrs bcva in range of 1.3 logmar (approximate snellen equivalent: 20/400) to 1.9 logmar (snellen equivalent: 20/1600), and etdrs bcva no better than 20/200 in the fellow eye. 6. presence of retinal inner nuclear and nerve fiber layers on optical coherence tomography (oct) testing in the study eye at screening exclusion criteria: 1. presence of any concurrent ocular disease that would affect study outcomes (e.g., severe cataracts; subjects can be enrolled 3 months after successful cataract surgery). 2. received any of the following treatments: gene therapy, stem cell therapy, surgical implantation of prosthetic retinal chips (such as argus-ii) or sub-retinal injections. 3. has taken non-approved items (supplement containing vitamin a or beta-carotene, liver-based products, or prescription oral retinoid medications) over the past 30 days 4. participation in an interventional study of a vitamin a derivative ≤ 3 months prior to screening 5. presence of significant cardiovascular or cerebrovascular disease, including stroke within 12 months of entry. 6. resting heart rate outside specified limits upon repeated measurement. 7. history of uncontrolled diabetes, hepatitis, pancreatitis, cirrhosis, liver failure, uncontrolled thyroid disease or hypervitaminosis a. 8. any intraocular surgery or thermal laser within 3 months of trial entry or any prior thermal laser in the macular region. 9. any major surgical procedure within one month of trial entry or anticipated during the trial. 10. clinically significant abnormal lab results at screening 11. known serious allergies to the fluorescein dye used in angiography or intraocular pressure measurement, povidone iodine, or to the components of the vmco-010 formulation 12. in the investigator's opinion, any severe acute or chronic medical condition, psychiatric condition, physical examination finding or laboratory abnormality 13. pre-existing conditions in the study eye such as glaucoma, diseases affecting the optic nerve causing significant visual field loss, history of uveitis, corneal or lenticular opacities). 14. presence of any complicating systemic diseases such as malignancies whose treatment could affect central nervous system function.. 15. subjects who are positive for syphilis, hepatitis b, c, and human immunodeficiency virus (hiv) will be excluded.. 16. presence of narrow iridocorneal angles contraindicating pupillary dilation in the study eye. 17. presence of disorders of the ocular media in the study eye which could interfere with visual acuity and other ocular assessments, including oct, during the study period. 18. presence of macular hole in the study eye, evident by ophthalmoscopy and/or by oct examinations 19. current evidence of retinal detachment in the study eye assessed by the investigator that significantly affects central vision. 20. current use of hydroxychloroquine, chloroquine, or any related retina-toxic compounds. 21. active ocular inflammation or recurrent history of idiopathic or autoimmune associated uveitis.

Keywords: eye diseases retinal degeneration retinal dystrophy eye diseases, hereditary