Our Approach

Creating an engine for addressing inherited retinal diseases

It’s estimated that over 180,000 patients are waiting for treatments for their individual genetic conditions. Addressing this problem will require a coordinated, sustainable development and manufacturing approach.

Opus’ targets

More than 260 genes are known to cause inherited retinal diseases, which affect hundreds of thousands of patients. Almost all of these IRDs lack effective therapies to halt disease progression or rescue visual function.

Opus Genetics’ mission is to pave a proven, derisked, efficient path to the clinic for these urgently needed new therapies. Our first three programs address mutations in the LCA5, RDH12 and NMNAT1 genes that cause Leber’s congenital amaurosis. We continue to develop novel gene therapies for many of the other 260+ unaddressed retinal disease indications.

Over 260 genes are known to cause inherited retinal diseases

gene map

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’ 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, small-scale for small populations and a small target (eye)

Although considered rare, vision-threatening inherited retinal diseases affect over 2 million people worldwide.  Over 260 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 will 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.

—Ash Jayagopal, CSO, Opus Genetics