Clinical Research Directory

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.