Table 1.

Characteristics of the Participants at Baseline.^*

Participant	Sex	Age	Zygosity (Second Allele)†	Best Corrected FST Sensitivity		Red-Light Visual Acuity‡
				Study Eye	Control Eye	Study Eye	Control Eye
		yr		logMAR		log cd-sec/m 2
Cohort 1
C1P1	Female	50	CH (c.1445T→A)	3.5	3.5	−2.0	Limited data
C1P2	Male	42	CH (c.5587–1G→C)	3.9	3.9	Limited data	Limited data
Cohort 2
C2P1	Female	54	Homozygous	2.7	2.3	−1.7	−1.8
C2P2	Male	20	CH (c.508A→T)	1.4	1.4	−2.1	−2.4
C2P3	Female	19	CH (c.7344_7345delTT)	0.6	0.5	−3.2	−3.2
C2P4	Female	63	CH (c.5668G→T)	0.9	0.6	−3.9	−3.8
C2P5	Female	17§	CH (c.3123dupT)	3.9	3.9	−0.5	−0.5
Cohort 3
C3P1	Female	28	CH (c.4723A→T)	2.3	1.6	−1.5	−1.9
C3P2	Female	38	CH (c.5668G→T)	1.0	0.9	−3.7	−3.7
C3P3	Female	36	CH (c.4661_4663delAAG)	3.9	3.9	−0.6	−0.6
C3P4	Male	35	CH (c.1666delA)	2.0	0.7	−1.6	−2.2
C3P5	Female	59	CH (c.5788_5792delAAAGA)	2.9	2.3	−1.3	−1.8
Cohort 4
C4P1	Male	14	Homozygous	3.9	3.9	−1.3	−1.2
C4P2	Male	9	CH (c.4723A→T)	1.2	1.2	−2.6	−3.0

^*Cohort 1 comprised adults who received a low dose (6×10¹¹ vector genomes [vg] per milliliter) of EDIT-101 gene therapy in the worse (study) eye; cohort 2, adults who received an intermediate dose (1×10¹² vg per milliliter); cohort 3, adults who received a high dose (3×10¹² vg per milliliter); and cohort 4, children who received an intermediate dose. The contralateral (control) eye was untreated. Participant identifiers are defined according to cohort (C) number and participant (P) number. FST denotes full-field stimulus testing.

^†The second allele is provide for participants with compound heterozygosity (CH).

^‡A best corrected visual acuity of 3.9 log₁₀ of the minimum angle of resolution (logMAR) is equivalent to light perception; 3.5 logMAR, to black–white discrimination; and 3.2 logMAR, to white field projection.

^§The participant turned 18 years of age after consent was initially provided and provided consent as an adult before receiving treatment.