It marks the first clinical trial for the Pennsylvanian biotech’s modifier gene therapy platform. The platform aims to target nuclear hormone receptors (NHRs) that regulate multiple functions within the retina, giving it the potential to address many different gene mutations (and thus multiple disease) with a single product. (Traditional gene therapies only address one individual gene mutation at a time).
RP is a group of rare, genetic disorders that involve a breakdown and loss of cells in the retina. Common symptoms include difficulty seeing at night and a progressive loss of peripheral vision. It is estimated that RP affects roughly 1 in 4,000 people – approximately two million people – globally. There is currently no approved therapy intended to stop the progression of RP based on all of the genetic mutations that cause the disease.
The first phase of the study is a safety evaluation of the product, eventually progressing into an efficacy study in patients.
“Our premise is that disease progression can be halted at whatever stage patients are currently at, potentially preventing further vision loss,” said David Birch, PhD, Scientific Director at the Rose-Silverthorne Retinal Degenerations Laboratory.
“This Phase 1/2 clinical trial targets people who have RP resulting from mutations in the NR2E3 and RHO genes. Based on the safety and efficacy outcomes, this study may be expanded to include additional genetic mutations in a Phase 3 study designed to demonstrate broad therapeutic applications of OCU400 in people with RP and Leber congenital amaurosis.
"If approved, we believe OCU400 may ultimately impact the lives of people facing retinitis pigmentosa and other retinal diseases rooted in the mutations of more than 175 genes.”