A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)

Main Inclusion Criteria Relating to Study Initiation:

• Male or female, ≥ 8 years of age at Screening with a clinical diagnosis of LCA10 and a molecular diagnosis of homozygosity or compound heterozygosity for the c.2991+1655A>G mutation, based on genotyping analysis at Screening. A historic genotyping report from a certified laboratory is acceptable with Sponsor approval.

• BCVA better or equal to Logarithm of the Minimum Angle of Resolution (LogMAR) +3.0 (Hand Motion), and equal to or worse than LogMAR +0.4 in the treatment eye.

• Detectable outer nuclear layer (ONL) in the area of the macula.

• An electroretinogram (ERG) result consistent with LCA. A historic ERG result may be acceptable for eligibility.

Main Exclusion Criteria Relating to Study Initiation:

• Presence of any significant ocular or non-ocular disease/disorder (including medication and laboratory test abnormalities).

• Prior receipt of intraocular surgery, periocular surgery, or IVT injection within 1 month prior to study start or planned intraocular surgery or procedure during the course of the study.Subjects who received an intraocular or periocular surgery between 1 to 3 months prior Screening, may only be considered for inclusion if there are no clinically significant complications of surgery present, and following approval by the Medical Monitor.

• History or presence of ocular herpetic diseases.

• Presence of any active ocular infection in the either eye.

• Presence of lens opacities/cataracts in the treatment eye.

• Current treatment or treatment within the past 12 months with therapies known to influence the immune system.

• History of glaucoma, or an IOP greater than 24 mmHg, at is not controlled with medication.

• History of amblyopia

• Use of any investigational drug or device within 90 days or 5 half-lives of Day 1, whichever is longer, or plans to participate in another study of a drug or device during the PQ-110-003 study period.

• Any prior receipt of genetic or stem-cell therapy.

• Known hypersensitivity to antisense oligonucleotides or any constituents of the injection.

• Pregnant and breastfeeding subjects.

Main Inclusion Criteria Relating to Treatment Initiation Contralateral Eye:

• BCVA equal to or better than LP (logMAR +4), using the best BCVA reading at Month 12 and based on ETDRS or BRVT.

• Detectable outer nuclear layer (ONL) in the area of the macula.

• Clear ocular media and adequate pupillary dilation to permit good quality retinal imaging.

Main Exclusion Criteria Relating to Treatment Initiation Contralateral Eye:

• Presence of any significant ocular or non-ocular disease/disorder (including medication and laboratory test abnormalities).

• History or presence of ocular herpetic diseases.

• Presence of any active ocular infection in either eye.

• Presence of any lens opacities which are clinically significant, would adequately prevent clinical and photographic evaluation of the retina.

• A planned IVT injection or intraocular or periocular surgery/procedure (including refractive surgery) during the course of the study.

• A history of glaucoma or an IOP greater than 24 mmHg that is not controlled with medication.

• History of amblyopia.

• Plans to participate in another study of a drug or device during the study period.

• Pregnant and breastfeeding subjects.