Why gene therapy?
Genetic mutations prevent production and/or appropriate function of the protein encoded by the gene. Opus Genetics is developing gene therapies to deliver a normal copy of the disease-causing gene, beginning with mutations in genes that cause different forms of Leber congenital amaurosis, a rare form of inherited retinal disease (IRD).
There are several advantages of ocular gene therapy, including:
- small dose required;
- ability to precisely place the dose in the desired location subretinally;
- high levels of immune privilege in the back of the eye; and
- ability to observe the safety and efficacy readily and non-invasively with various imaging techniques and functional diagnostic approaches.
Opus’ first three programs are being developed to address retinal diseases caused by mutations in the LCA5, RDH12 and NMNAT1 genes. In the near future, we will leverage our approach to expand our pipeline to address additional genetic targets across the spectrum of 280+ inherited retinal diseases. » Our approach