Opus Genetics develops AAV-based gene therapies targeting inherited retinal diseases and describes itself as patient-first and clinical-stage. Its portfolio includes a derisked LCA5 lead program currently in a Phase 1/2 clinical trial. The company is advancing multiple preclinical candidates, including programs targeting rhodopsin-mediated autosomal dominant retinitis pigmentosa (RHO-adRP) and MERTK-related retinitis pigmentosa. Opus plans IND-enabling studies for a newly designed AAV vector to replace mutated MERTK in retinal pigmented epithelial cells and anticipates the RHO vector safety study will be the last preclinical study before entering clinical trials. Opus is based in Research Triangle Park, N.C., and is backed by the Foundation Fighting Blindness’ venture arm, the RD Fund. The company’s current financing update reflects project-based, non-dilutive support to advance preclinical and IND-enabling work. Opus Genetics is a patient-focused gene therapy company developing AAV-based therapies for orphan inherited retinal diseases. The company was formed to advance preclinical work from scientific cofounders Jean Bennett, Junwei Sun and Eric Pierce, with lead programs licensed from the University of Pennsylvania. Its lead programs, OPGx-001 (LCA5) and OPGx-002 (RDH12/LCA13), target severe forms of Leber congenital amaurosis and recent preclinical data have demonstrated potential to restore retinal structure and function. Opus expects to file an IND for OPGx-001 in early 2022 and to enter the clinic in mid-2022. The company says it is building novel orphan manufacturing scale and efficiencies and plans to expand its pipeline with additional programs. Opus is based in Raleigh, N.C., and launched with seed funding to advance its preclinical programs.

Total raised

$21M

Funding rounds

2

Latest round

Seed

Latest activity

Sep 2021