By Isabel Di Rosa
Scheie Vision Annual Report 2021
In October 2021, Scheie Eye Institute researchers Jean Bennett, MD, PhD and Mr. Junwei Sun launched a new biotech startup called Opus Genetics, which aims to discover new treatments for rare blinding conditions. Dr. Bennett and colleagues previously developed Luxturna, the novel gene therapy that has treated vision loss in patients with a form of Leber congenital amaurosis (LCA) caused by mutations in the RPE65 gene. Dr. Bennett and Mr. Sun have teamed up with Dr. Eric Pierce, Professor of Ophthalmology at Harvard Medical School and Massachusetts Eye and Ear Infirmary, to launch this new company.
Opus Genetics’ $19 million in seed funding is provided by the Foundation Fighting Blindness’ Retinal Degeneration Fund, as well as the Manning Family Foundation and Bios Partners. Other founding members of Opus include Dr. Ben Yerxa, Dr. Rusty Kelley, Mr. Jason D. Menzo, and Mr. Peter Ginsberg, all of whom have senior positions at the Foundation Fighting Blindness.
Building upon Dr. Bennett and Mr. Sun’s prior research and the expertise of the other founders, Opus Genetics has an initial goal of investigating potential gene therapies to treat variations of LCA. The gene therapies (namely, OPGx-001 and OPGx-002) would work by targeting genetic mutations in the LCA5 and LCA13 genes. If successful, these therapies could restore sight in patients with these forms of congenital blindness.
Opus Genetics coins itself as a patient-first company. “We are selecting some of the most neglected forms of inherited blindness that big pharma traditionally would not touch because they are extraordinarily rare,” Dr. Bennett explained. “These are devastating diseases, and we want to help the patients and their families. The patients are an integral part of Opus Genetics - in fact, they helped found this company.”
Large pharmaceutical companies are less likely to research rare diseases because of the low profit margin; treatments for common diseases can generate much greater revenue. Opus Genetics will help fill the gap in research on LCA, and hopefully result in targeted treatment options.