Clinical Research Directory

Inclusion Criteria: * Age ≥18 years (adult participants) or 12-17 years (adolescent participants) at the time of consent/assent. * Provide written informed consent and/or assent prior to any study procedures. * Willing to adhere to the clinical protocol and follow directions of the Investigator regarding post-surgery restrictions. * Are a good candidate for surgery, per the Investigator's judgment. * Have LCA with autosomal-recessive RDH12 mutation(s), confirmed by a Clinical Laboratory Improvement Amendments (CLIA)-certified laboratory. Historic testing, up to 15 years prior to date of consent, may be considered. * Clinical diagnosis of LCA with RDH12 mutation(s), in the judgment of the Investigator. * BCVA 20/200 (1.0 logarithm of the minimum angle of resolution \[logMAR\]) or worse for the sentinel adult in each cohort; BCVA 20/40 (0.5 logMAR) or worse for all subsequent participants in each cohort. Exclusion Criteria: * Women of childbearing potential (WOCBP) who are pregnant, lactating, and/or unwilling to use effective contraception from Screening through 1 year after IMP administration. * Men who are unwilling to use effective contraception from Screening through 180 days after IMP administration. * Have an ocular infection, a pre-existing eye condition, or a complicating systemic disease that could preclude the planned surgery or any future ocular surgery. This includes individuals who are immunocompromised and/or on continuous systemic immunosuppressive therapy. * Have a past or current condition that may preclude participation in the study, interfere with outcome measure testing or test results, or otherwise make the potential participant unsuitable for the study. * Have previously received gene therapy of any kind. * In either eye, have undergone intraocular surgery within 90 days prior to planned IMP administration or have active inflammation at Screening resulting from prior ocular surgery. * Have used any investigational device or investigational drug within 90 days (or 5 half-lives of the drug, whichever is longer) prior to planned IMP administration or intend to participate in another drug or device study during the same period as the current study. * Have received or plan to receive a vaccination within 6 weeks prior to or 6 weeks after IMP administration. Note: For the influenza vaccine, the exclusionary period is shorter: 2 weeks prior to and 5 weeks after IMP administration (i.e., during steroid treatment). * Have received anticoagulant therapy within 2 weeks prior to planned IMP administration. * Currently use medications that are potentially neuroprotective/beneficial or retinotoxic. * Are incapable of performing visual function testing (e.g., FST), with or without assistance, for reason other than poor vision. * Have any contraindication to a course of oral steroids, in the opinion of the Investigator. * Have a known history of hypersensitivity to constituents or excipients in the pharmaceutical formulation of the IMP. * Have a known or active infection of human immunodeficiency virus (HIV) or hepatitis B or C virus. * Have a known or active infection of herpes simplex virus with ocular manifestations. * Are an employee of the Sponsor or a relative of the Investigator or investigative site staff.