Opus Genetics completes dosing in first cohort of Leber congenital amaurosis trial
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Key takeaways:
- The first cohort has finished dosing in the phase 1/2 clinical trial of OPGx-LCA5.
- The gene therapy, under investigation for Leber congenital amaurosis, demonstrated signs of biological activity through 90 days.
Dosing is complete in the first cohort of the phase 1/2 clinical trial of OPGx-LCA5 for the treatment of Leber congenital amaurosis, according to a press release from Opus Genetics.
An adeno-associated virus 8 vector is used to deliver a functional LCA5 gene to the outer retina via subretinal administration in patients with Leber congenital amaurosis resulting from biallelic mutations in the LCA5 gene.
https://opusgtx.com/companynews/opus-genetics-announces-completion-of-dosing-in-first-cohort-of-phase-1-2-trial-of-gene-therapy-opgx-lca5-in-patients-with-rare-inherited-retinal-disease-lca5/
Published March 26, 2024. Accessed March 27, 2024.
The company plans to advance the gene therapy to the next highest dose following positive safety and efficacy data from the first cohort of three adult patients. The next cohort is expected to be initiated later this year, and future plans include expanding the study population to subjects aged 13 years and older.
“In the first cohort, OPGx-LCA5 has been well tolerated and demonstrated clear signs of biological activity through 90 days, warranting continued evaluation in the next highest dose,” Tomas S. Aleman, MD, the study’s principal investigator, said in the release. “Moreover, early anecdotal and [virtual reality] challenge test feedback is encouraging and indicates that some of the patients, who have been nearly totally blind all their lives, are now able to see and identify objects for the first time.”
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