Opus genetics launches clinical trial for MERTK-related retinitis pigmentosa

Opus Genetics, a clinical-stage biopharmaceutical company, recently announced that they have launched an early phase clinical trial for OPGx-MERTK, an investigational gene therapy in development for MERTK-related RP.

“Launching our clinical trial for a MERTK gene therapy is a defining moment for patients and for the field of inherited retinal disease…For patients living with MERTK-related retinitis pigmentosa, this trial represents the first real opportunity to potentially change the course of a disease that has historically led to inevitable vision loss.” – George Magrath, M.D., Chief Executive Officer of Opus Genetics

What happens in MERTK-related RP?

MERTK-related RP affects an estimated 60,000 people globally, resulting in progressive vision loss starting peripherally due to the loss of light-sensing photoreceptor cells in the retina. There are two types of photoreceptors, cones and rods, which differ in structure, function and location. Rods are located in the periphery of the retina and are responsible for night vision and peripheral awareness. Cones are located in the centre of the retina and give you sharp vision and colour detection in good lighting. To learn more, please watch this short animation video on Inherited Retinal Disease.

These rod and cone cells shed the tips of their outer segments every day. The retinal pigment epithelium (RPE), a layer of cells that support the photoreceptors, must engulf and digest these outer segments to prevent them accumulating in the subretinal space. This process is known as phagocytosis. The gene MERTK is a key trigger of this process and is therefore required for photoreceptor survival. When MERTK is absent or non-functional, the shed segments accumulate causing oxidative stress and inflammation which disrupts support from the RPE. This leads to progressive photoreceptor death starting with rods in the periphery, as they don’t receive the required support from the RPE that keeps the cells healthy.

What is OPGx-MERTK?

OPGx-MERTK is an adeno-associated viral vector (AAV) gene therapy. This means that a small virus, that doesn’t cause illness in humans, is used as a delivery system to package and transport the gene therapy into cells to alter the genetic material. Opus Genetics look to deliver a healthy copy of the MERTK gene into retinal cells, allowing for normal ‘clean-up’ processes in the RPE to take place, reducing the accumulation of shed photoreceptor segments and preventing photoreceptor degeneration.

The trial:

This early phase clinical trial will take place in Abu Dhabi and will evaluate the safety, tolerability and preliminary efficacy of a single subretinal injection of OPGx-MERTK.  The program looks to commence activities in 2026 according to the latest press release from Opus Genetics, however no further information is available currently about recruitment activities or trial dates.

If you wish to read more about Opus Genetics, please visit their website here. Opus Genetics have also recently dosed the first patients in their OPGx-BEST1 phase 1/2 clinical trial, which you can read about here.