Opus reports vision improvements for patients in LCA5 gene therapy clinical trial

Key Takeaways

• Opus Genetics has announced vision improvements for the first three adult patients in its Phase 1/2 gene therapy clinical trial for Leber congenital amaurosis 5 (LCA5).
• Some of the patients, who had been almost totally blind since birth, can now see and identify objects for the first time. 

Opus Genetics has announced vision improvements for the first three adult patients in its Phase 1/2 gene therapy clinical trial for Leber congenital amaurosis 5 (LCA5), a rare, severe, inherited retinal disease. Some of the patients, who had been almost totally blind since birth, can now see and identify objects for the first time. The company has also reported positive safety data for the trial thus far. 

Although LCA5 patients have severe vision loss at birth, they have surviving retinal structure which researchers believe can be harnessed for improved vision using gene therapy. The new gene therapy, known as OPGx-001, uses a human-engineered adeno-associated virus (AAV) to deliver healthy copies of the LCA5 gene to the retinas of patients, augmenting the mutated copies causing vision loss. The therapy is administered through a one-time injection underneath the retina. 

Opus plans to administer the next highest dose of its LCA5 gene therapy to the next cohort of adult patients in mid-2024. The company also has plans sometime in the future to dose patients as young as 13 years of age. 

For more information on this trial, visit https://clinicaltrials.gov/study/NCT05616793 or contact Sarah Tuller at 860-888-2718 or STuller@OpusGtx.com. 

Edited by Miriam Kaplan, PhD

Source: Eye on the Cure Research News, Foundation Fighting Blindness, March 26, 2024; see source article