SAD of IVT VP-001 in PRPF31 Mutation-Associated Retinal Dystrophy Subjects

Inclusion Criteria:

1. Male or female sex; ≥ 18 years of age at Baseline (Visit 2).

2. Have a molecular (genetic) diagnosis of PRPF31 mutation.

3. Have a clinical diagnosis of PRPF31 mutation-associated retinal dystrophy, that is,RP11. The following conditions are allowed for inclusion if due to RP11, if in theopinion of the investigator they will not interfere with study evaluations or haveresolved: macular edema (intraretinal, sub-retinal or other fluid) requiring regulartreatment at a frequency of less than every 6 weeks; macular edema must be stablefor at least 3 months prior to Screening (Visit 1). The investigator must consultwith the study Medical Monitor.

4. If ≥ 18 years of age, understand the language of the informed consent and arewilling and able to provide written informed consent prior to any study procedures.Are willing to comply with the instructions and attend all scheduled study visits.

5. Have light perception (LP) or better vision in the study eye. 7. Participants ofchildbearing potential and male participants must not be pregnant or lactating andmust be sexually inactive by abstinence, which is consistent with the preferred andusual lifestyle of the participant or agree to use adequate birth control throughoutstudy duration. Adequate birth control is defined as hormonal - oral, implantable,injectable, or transdermal contraceptives; mechanical - spermicide in conjunctionwith a barrier such as a condom or diaphragm; intrauterine device (IUD); or surgicalsterilization of partner. For nonsexually active participants, abstinence may beregarded as an adequate method of birth control. Participants of childbearingpotential include all participants who have experienced menarche and have notundergone successful surgical sterilization (bilateral tubal ligation, hysterectomy,or bilateral oophorectomy) or are not post-menopausal (12 months after last menses).

Exclusion Criteria:

1. Have any uncontrolled systemic disease that, in the opinion of the Investigator,would preclude participation in the study that include but are not limited toinfection, uncontrolled elevated blood pressure, cardiovascular disease, or glycemiccontrol issues, or any other medical condition that may put the participant at riskdue to study procedures.

2. Mutations in genes that cause autosomal dominant RP, X-linked RP, or presence ofbiallelic mutations in autosomal recessive RP/retinal dystrophy genes other thanPRPF31 mutations.

3. Have used anti-vascular endothelial growth factor (VEGF) agents within 2 months orcorticosteroid injections within the last 3 months.

4. Have had Ozurdex® implants placed within 3 months or Retisert® or Iluvien® implantsplaced within 3 years prior to Baseline (Visit 2).

5. Within 3 months prior to Baseline (Visit 2), have undergone any vitreoretinalsurgery (scleral buckle, pars plana vitrectomy, retrieval of a dropped nucleus orintraocular lens, radial optic neurotomy, sheathotomy, cyclodestructive proceduresor multiple filtration surgeries [2 or more]) or any other ocular surgery.

6. Have ocular media opacity or poor pupillary dilation prohibiting quality ophthalmicevaluation or photography, as assessed by the investigator.

7. Have used any investigational drug or device within 90 days or 5 estimatedhalf-lives of Baseline (Visit 2), whichever is longer, or plan to participate inanother study of drug or device during the study period. Participation inobservational trials is allowable based on investigator discretion and consultationwith the Medical Monitor. It is assumed that the observational trial evaluationswould not interfere with participation in this study.

8. Have received any prior cell or gene therapy for a retinal condition.

9. Have a recent history (<6 months) or current excessive recreational drug or alcoholuse, in the opinion of the investigator.

10. Any retinal pathology other than RP11 that in the investigator's opinion couldaffect study results.

11. Participants should not have any conditions, in the investigator's opinion, that mayput the participant at increased risk, confound study data, or interferesignificantly with the participant's study participation.