Our Approach

Creating an engine for addressing inherited retinal diseases

It’s estimated that over 300,000 patients are waiting for treatments for their individual genetic conditions, and more than 280 genes are known to cause inherited retinal diseases.

OPGx-RHO is in preclinical development to support the initiation of a first-in-human trial in 2026.

Opus Genetics’ mission is to pave a proven, derisked, efficient path to the clinic for these urgently needed new therapies. Our IRD programs address mutations in genes that cause different forms of bestrophinopathy, Leber congenital amaurosis (LCA) and retinitis pigmentosa (RP), and are based on world-class science from gene therapy pioneers at the University of Pennsylvania (including the lab of Jean Bennett, MD, PhD), Harvard Medical School, and the University of Florida.

 

260+ genes are associated with Retinal disease

Opus’ unique approach

  1. Validated science, well characterized material by preeminent leaders in the field, and safe and effective retinal transduction by AAV
  2. Insight & experience of our founders’ and teams’ careers-long research and development in inherited retinal diseases brings best practices for clinical development (e.g., design, timing, dosing, endpoints)
  3. Infrastructure and processes that can be levered for multiple programs with at least one IND per year
  4. Manufacturing that is high-quality, pharmaceutical grade tailored for rare disease populations

“Although considered rare, vision-threatening inherited retinal diseases affect over 2 million people worldwide. Over 280 genes have been identified which are responsible for these conditions, but since 2017 only one gene therapy has been approved for a single class of IRD.  We intend to address the need for therapies by developing a tailored manufacturing approach to support a sustainable pipeline validated by world-class scientists, to significantly expand the number of gene therapies available for retinal diseases.”

—Ben Yerxa, PhD, President, Opus Genetics