A biotechnology company in Research Triangle Park has secured funding to advance two of its gene therapy programs toward the clinic.
The Foundation Fighting Blindness is providing $1.7 million in project-based funding to Opus Genetics, a biotech trying to address causes of blindness through the development of gene therapy treatments that target inherited retinal diseases.
The funding includes a $1 million award to support Opus in conducting a preclinical safety study for a gene therapy vector designed to target a specific inherited retinal disease that affects more than 6,000 people in the U.S. The company expects that this safety study is the last preclinical study required before the gene therapy candidate enters clinical trials. This program could be in clinical trials in the second half of 2025, CEO Ben Yerxa said in an email.
Additionally, Opus secured about $700,000 in project-based and operational funding that will support the preclinical development of a novel viral vector that treats specific gene mutations that cause a rare eye disease that affects the retina. This program is earlier in development, with the initial funding providing the company with resources to test the vector in an established animal model and to conduct early safety assessments.
The Foundation Fighting Blindness has supported Opus since its launch. The company raised $19 million in seed funding prior to its launch in 2021, with the Retinal Degeneration Fund, the venture arm of the Foundation Fighting Blindness, leading the initial round. Opus last year filed paperwork with the U.S. Securities and Exchange Commission showing it has raised another $4.5 million.
Opus has already advanced one gene therapy candidate into clinical trials.
The company in September began dosing the first cohort of patients in a Phase 1/2 trial evaluating the company's lead program, OPGx-LCA5. These were three patients in their 20s with Leber congenital amaurosis, a rare inherited eye disorder that causes severe vision loss within the first few months of a patient's life.
Testing in this first cohort was mostly around the goal of evaluating the safety of the gene therapy, but in March, the company announced some early positive results regarding the gene therapy's efficacy. Multiple subjects in this initial cohort experienced vision improvements.
One person improved from seeing only large blurry objects to seeing mountains for the first time in their life. Another person improved from having hand motion vision — meaning they could see the motion of a person's hands waving in front of them, but could not, for instance, make out the number of fingers on each hand — to being able to read the largest letters on an eye exam chart.
Following these early positive results, Yerxa previously told Triangle Business Journal that the company would advance the program with another cohort of patients at the next highest dose. The company also plans to talk with the U.S. Food and Drug Administration to expand the study population to include subjects 13 years or older.
Yerxa, in an email, said the company will not have an update available for this program until later this summer.
