Complement-targeted gene therapies for treatment of GA, other ocular diseases licensed by Kriya Therapeutics

Kriya Therapeutics Inc. recently took the wraps off an exclusive agreement with the Medical University of South Carolina (MUSC) Foundation for Research Development to license next generation complement-targeted gene therapies for the treatment of geographic atrophy and other ocular diseases.

Geographic atrophy, also known as atrophic age-related macular degeneration (AMD), is an advanced form of AMD, a progressive retinal disease affecting millions of adults worldwide. Patients with geographic atrophy experience irreversible loss of vision with significant impact on quality of life due to the chronic and progressive nature of the disease. There are currently no FDA-approved treatments available for geographic atrophy.

In a news release, Theresa Heah, MD, MBA, president and chief medical officer of Kriya’s ophthalmology division, the partnership highlights the broad potential of Kriya’s technology and R&D platforms to drive innovation in diseases with established biology, and furthers our mission of developing transformative gene therapies for diseases with high unmet need.

“We believe that complement hyperactivity is a clinically validated target implicated in the pathogenesis of retinal degeneration in geographic atrophy, and has the potential to address other ocular diseases,” she said in a statement. “We are excited to develop gene therapies that precisely target this pathway, in collaboration with academic partners who have been at the forefront of characterizing the biology underlying these diseases.”

According to the company, the complement system plays a crucial role in the body’s innate immune system by enhancing its ability to clear pathogens and damaged cells, and regulating inflammatory immune responses through complement control proteins. Dysregulation and hyperactivity of the complement system is associated with the onset and progression of serious inflammatory diseases, including geographic atrophy and other ocular conditions. Through this agreement, Kriya is advancing gene therapies that are designed to durably express engineered molecules that selectively reduce complement hyperactivity at the site of pathology following one-time administration.

“Geographic atrophy due to age-related macular degeneration is a devastating disease with a profound impact on patients, as there are no approved treatments for the irreversible loss of central vision that often occur,” said Peter K. Kaiser, MD, a professor of ophthalmology, Cole Eye Institute, Cleveland Clinic. “Recent advances in complement inhibitor therapies have provided important additional evidence that targeting complement holds great promise in treating geographic atrophy. I am excited by the potential of restoring balance to the complement system with a one-time gene therapy that can deliver a meaningful long-term solution and a major advancement in the field of retinal disease.”

Bärbel Rohrer, PhD, professor of ophthalmology at the Medical University of South Carolina, and co-inventor of the technology, noted that by targeting the inhibition of complement proteins directly involved in complement activation, the approach has the potential to deliver a selective and profound biological effect.

“After having confirmed the potential of this gene therapy strategy in mouse models of age-related macular degeneration, we are very excited to take the next step towards a clinical application for patients with geographic atrophy by partnering with Kriya, a leader in the development of novel gene therapies,” Rohrer concluded.