Opus reports positive pediatric data for gene therapy in rare eye disorder

Opus Genetics announced positive three-month data from the pediatric cohort of its ongoing phase 1/2 clinical trial evaluating its investigational gene augmentation therapy for the ultra-rare eye disorder, Leber congenital amaurosis type 5 (LCA5).

Three pediatric participants aged 16-17 with severe baseline vision impairment received a single subretinal injection of OPGx-LCA5. All three participants achieved measurable improvements in visual acuity, retinal sensitivity, and real-world navigation tasks within three months, including: A group average of a 0.3 logMAR improvement in visual acuity which is greater than was observed in the adult cohort; greater than one log unit improvement in cone sensitivity to both red and blue light; all participants identified more objects through three-months compared to baseline.

 Leber congenital amaurosis is a group of inherited retinal diseases characterized by severe impaired vision or blindness at birth. OPGx-LCA5 is designed to address a form of LCA that is due to biallelic mutations in the LCA5 gene, which encodes the lebercilin protein. LCA5-associated inherited retinal disease is an early-onset severe inherited retinal dystrophy. Studies in patients with this mutation have reported evidence for the dissociation of retinal architecture and visual function in this disease, suggesting an opportunity for therapeutic intervention through gene augmentation. OPGx-LCA5 uses an adeno-associated virus 8 (AAV8) vector to precisely deliver a functional LCA5 gene to the outer retina.