MAD of IVT VP-001 in PRPF31 Mutation-Associated Retinal Dystrophy Subjects (Wallaby)

Inclusion criteria

• ✓. Male or female sex; ≥12 years of age at Baseline (Visit 2).
• ✓. Have a molecular (genetic) diagnosis of PRPF31 mutation.
• ✓. 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 the opinion of the investigator they will not interfere with study evaluations or have resolved: macular edema (intraretinal, sub-retinal or other fluid) requiring regular treatment at a frequency of less than every 6 weeks; macular edema must be stable for at least 3 months prior to Screening (Visit 1). The investigator must consult with the study Medical Monitor.
• ✓. If ≥18 years of age, understand the language of the informed consent and are willing and able to provide written informed consent prior to any study procedures. If a minor (12 to \<18 years of age), a parent or legal guardian willing and able to provide written permission for the minor's participation prior to performing any study related procedures and pediatric participant able to provide age appropriate assent for study participation.
• ✓. If ≥18 years of age, are willing to comply with the instructions and attend all scheduled study visits. If a minor (12 to \<18 years of age), able to complete all study assessments, comply with the protocol, and has a parent or caregiver willing and able to follow study instructions and attend study visits with the participant as required, in the opinion of the Investigator.
• ✓. Meets ≥1 of the following for visual function in the study eye:
• ✓. V4e visual field \>1000 deg2, per kinetic perimetry
• ✓. \<Mean microperimetry threshold: \>5 decibel (dB) to \<15dB

Exclusion criteria