Kriya Therapeutics Licenses Emerging Foundation-Funded Dry AMD Gene Therapy
Eye On the Cure Research News
The Foundation Fighting Blindness is funding Bärb Rohrer, PhD, Medical University of South Carolina, to evaluate safety and efficacy of the gene therapy in a large animal model
Kriya Therapeutics, a developer of pioneering gene therapies, has licensed an emerging gene therapy for people with dry age-related macular degeneration (AMD) from the Medical University of South Carolina (MUSC). Developed by MUSC’s Bärbel Rohrer, the gene therapy is designed to inhibit the retinal damage caused by the overactive complement system, part of the innate immune system, in people with dry AMD. Dr. Rohrer’s novel approach targets complement inhibition, where damage is most likely to occur in the retina. Through its Translational Research Acceleration Program (TRAP), the Foundation Fighting Blindness is funding Dr. Rohrer to evaluate the treatment in a large animal model, an important step toward launching a clinical trial.
Dry AMD is a key focus for the Foundation, because there are currently no therapies approved by the US Food & Drug Administration (FDA) for the condition, which is a leading cause of blindness in people over 55 in developed countries. The Foundation currently provides $3 million in funding for seven AMD projects.
“We are delighted to see Kriya’s investment in the innovative work of Dr. Rohrer to advance a promising treatment for dry AMD toward a clinical trial. The condition is a critical unmet need,” says Claire Gelfman, PhD, chief scientific officer at the Foundation Fighting Blindness. “Kriya’s investment also helps fulfill the goal of our TRAP program to move emerging treatments out of labs and into clinical trials.”
The Foundation currently provides $4.8 million for six TRAP projects.
The hallmark of dry AMD is the accumulation of deposits known as drusen underneath the retina. Comprised of lipids and proteins, drusen can lead to degeneration of the macula, the central region of the retina which provides central vision, color and daytime vision, and the ability to read and recognize faces.
In 2005, Foundation-funded researchers identified variations in genes associated with an overactive complement system and increased AMD risk. While the complement system protects the body from harmful viruses and bacteria, it can be damaging if it isn’t regulated correctly. Proteins from the complement system have been found in drusen.
Virtually all people with AMD start off with the dry form. People can lose central vision if the dry form progresses to cell loss in the macula, an advanced condition known as geographic atrophy (GA). There is currently no treatment for dry AMD or GA.
People can also develop wet AMD, which is characterized by the growth of leaky blood vessels underneath the retina. There are a few FDA-approved treatments available to block blood vessel growth to save and restore vision for people with wet AMD.
For more information about AMD, visit the AMD section of the Foundation’s website.