Table S1. Study Inclusion and exclusion criteria

Inclusion Criteria:
MERTK-associated retinitis pigmentosa
VA: 20/100 or less in worse eye
Ability to perform tests of visual and retinal function
Good general health based on a pre-treatment medical evaluation
Ability to comply with research procedures
Exclusion Criteria:
Pre-existing eye conditions that would preclude the planned surgery or interfere with the interpretation of study endpoints or surgical complications (for example, choroidal neovascularization, glaucoma)
Complicating systemic diseases (such as medical conditions causing immunosuppression) that would preclude the gene transfer, ocular surgery or known sensitivity or allergy to medications planned for use in the peri-operative period
Use of anti-platelet agents that may alter coagulation within 7 days prior to study agent administration
Use of immunosuppressive medications
Pregnancy or breastfeeding
Individuals (males and females) of childbearing potential who are unwilling to use effective contraception for 1 year following agent administration and barrier contraception for 3 months following agent administration
Any other condition that would prevent a subject from completing follow-up examinations during the course of the study and that, in the opinion of the investigator, makes the subject unsuitable for the study
Current, or recent (within the past 30 days, or 10 half lives of the drug) participation, in any other research protocol involving investigational agents or therapies
Recent (within past 6 months) receipt of an investigational biologic therapeutic agent.

Table S2. Schedule of study visits

Timepoint / Baseline / Day
B1 / B2 / 0 / 1 / 2 / 3 / 4 / 5 / 6 / 7 / 8 / 9 / 10
Informed consent/assent / ■
History / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■
Physical exam / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■
Electrocardiogram, chest X-ray / ■
Pregnancy test / ■ / ■
Vital signs / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■
Hematology, chemistry, urinalysis / ■ / ■ / ■ / ■ / ■ / ■
AAV antibody and antigen specific reactivity (ASR) measurement / ■ / ■
Peripheral blood PCR / ■ / ■ / ■ / ■ / ■
Ophthalmic examination* / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■
Visual function* / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■
Fundus photography* / ■ / ■ / ■
Adverse event recording / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■
Timepoint / Day / Year
30 / 60 / 90 / 180 / 270 / 365 / 1.5 / 2 / 3 / 4-15
History / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■
Physical examination / ■ / ■ / ■
Vital signs / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■
Hematology, chemistry, urinalysis / ■ / ■ / ■ / ■ / ■
AAV antibody and antigen specific reactivity (ASR) measurement / ■ / ■
Ophthalmic examination* / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■
Visual function* and Lens examination for changes using LOCS classification / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■
Fundus photography* / ■ / ■ / ■ / ■ / ■
Adverse event recording / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■ / ■
Long-term follow-up / ■

*these examinations will be performed on both eyes.

Table S3. Window of acceptable timeframe for study evaluations
Acceptable timeframe (days)
Timepoint / +/- 2 / +/-5 / +/-30
Days 30, 60 / ■
Days 90, 180, 270, 365 / ■
Years 1.5, 2-15 / ■

Table S4 Fold Increase in Circulating anti-AAV2 Titer

Case # / Day 9/10 / Day 90 / Day 365
1 / 27 / n/a / 16
2 / 1 / 2 / 0
3 / 8 / 6 / n/a
4 / 0 / 1 / 1
5 / 1 / 1 / 1
6 / 1 / n/a / n/a

n/a= sample not available