Researchers seek new treatment for blinding diseases AMD and Stargardt

NEI has funded a project to develop new treatment for the dry form of age-related macular degeneration (AMD) and Stargardt disease.

The project, led by Christopher Cioffi, Rensselaer Polytechnic Institute, and Konstantin Petrukhin, Columbia University, will conduct preclinical testing of a treatment that targets the build-up in the retina of toxic yellow-brown pigment granules called lipofuscin.

"We are leveraging our drug development expertise to advance the development of a novel class of bispecific visual cycle modulators," said Petrukhin. "We hope that our development candidate, which combines optimal safety and efficacy, will emerge as a new and improved therapy for macular degeneration, benefiting diverse patient populations in need of enhanced treatment options."

AMD is the leading cause of blindness among older Americans, and dry AMD is the most common form. Stargardt disease is a rare genetic eye disease in which vision loss typically begins in childhood. Both conditions involve the build-up of lipofuscin in an area of the retina crucial for vision called the macula. Pyridinium bisretinoids are components of lipofuscin that are cytotoxic, or kill cells. Cioffi and Petruhkin aim to inhibit the synthesis of bisretinoids in order to treat dry AMD.