First dose administered in Phase 1/2 clinical trial for gene therapy candidate OCU400 for inherited retinal degeneration

Ocugen Inc. announced that the first patient has been dosed in the Phase 1/2 clinical trial (NCT05203939) of OCU400, a modifier gene therapy candidate for the treatment of retinitis pigmentosa (RP) resulting from mutations in the nuclear receptor subfamily 2 group E member 3 (NR2E3) and Rhodopsin (RHO) genes.

According to Shankar Musunuri, PhD, MBA, CEO, chairman of the board and co-founder of Ocugen, the milestone has been reason for celebration at the company.

“Every day, our teams are working toward developing a therapeutic for people who have no options when facing inherited retinal diseases,” Musunuri said in a statement. “The first phase of the study is a safety evaluation of the product, eventually progressing into an efficacy study in patients. Today’s announcement signifies a first and monumentally critical step forward in achieving our mission to cure blindness diseases.”

RP is a group of rare, genetic disorders that involve a breakdown and loss of cells in the retina (the light-sensitive tissue that lines the back of the eye).

Common symptoms include difficulty seeing at night and a progressive loss of side (peripheral) vision. It is generally estimated that RP affects roughly 1 in 4000 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.

Ocugen’s modifier gene therapy 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, in turn, multiple retinal diseases—with a single product. Traditional gene therapy, which transfers a functional version of a non-functional gene into target cells, addresses only one individual gene mutation at a time.

According to David Birch, PhD, scientific director at the Rose-Silverthorne Retinal Degenerations Laboratory, the premise is that disease progression can be halted at whatever stage patients are currently at, potentially preventing further vision loss.

“This Phase 1/2 clinical trial targets people who have RP resulting from mutations in the NR2E3 and RHO genes,” Birch said. “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.”