OCU400
Gene Therapy Target: NR2E3
Most recent annual filing: 2026-03-04
Nuclear hormone receptor (NHR)-based modifier gene therapy platform using AAV5 vector delivery to retinal tissue; targets NR2E3 and RORA to address multiple inherited retinal diseases including retinitis pigmentosa, Stargardt disease, and dry AMD/geographic atrophy with a gene-agnostic approach
Targets: NR2E3, RORA
Next-generation inhalation-based mucosal vaccine platform based on a novel ChAd vector; designed to generate local mucosal immunity in the nasopharyngeal region for respiratory infectious diseases including COVID-19 and influenza
Targets: SARS-CoV-2 Spike protein, influenza
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