Built by and for Patients

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 260+ inherited retinal diseases.  » Our approach

Patient Resources

We understand there is great interest within the patient community to advance the science, and we are committed to transparently driving transformative retinal gene therapy treatments to patients.

Opus is the first company conceived and launched by the Foundation Fighting Blindness’ venture arm, the Retinal Degeneration Fund, to further the Foundation’s mission of advancing treatments and cures for blinding retinal diseases. As such, Opus is uniquely positioned to bring experts, resources and patients together to efficiently advance ocular gene therapies for patients that to date have not been developed. We intend to have a dialogue with and inform the patient community of our progress as we advance our programs.

For more information and to get updates from Opus on our clinical progress, complete the form below.

Patient Resources

“While potential treatments for these ultra-rare conditions have existed for years, families have been stuck in a holding pattern waiting on someone to deliver a feasible business model to bring them to market. We’re thrilled to be a part of the launch of Opus Genetics to establish a patient-first priority and build capabilities to bring life-altering treatments for the people who need them most.”

– Paul Manning, Manning Family Foundation