Press release
Ocugen, Inc. Announces First Patient Dosed in Phase 1/2 Clinical Trial for Gene Therapy Candidate OCU400 to Treat Inherited Retinal Degeneration
Announcement marks first clinical trial in humans of Ocugen’s modifier gene therapy platform MALVERN, Pa., April 01, 2022 (GLOBE NEWSWIRE) -- Ocugen, Inc.
About this update from Ocugen, Inc.
[{"type":"text","content":"Announcement marks first clinical trial in humans of Ocugen’s modifier gene therapy platform\nMALVERN, Pa., April 01, 2022 (GLOBE NEWSWIRE) -- Ocugen, Inc. (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene therapies, biologicals and vaccines, announced that the first patient has been dosed in the Phase 1/2 clinical trial 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. This first patient dosing marks the beginning of the dose-escalating, observer-blind, Phase 1/2 safety and efficacy study. This Phase 1/2 study is currently enrolling. More information can be found on ClinicalTrials.gov, under identifier number NCT05203939. “Everyone at Ocugen is excited about this important milestone. Every day, our teams are working toward developing a therapeutic for people who have no options when facing inherited retinal diseases,” said Dr. Shankar Musunuri, Chairman of the Board, Chief Executive Officer, and Co-Founder of Ocugen. “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 4,000 people – approximately two million people – globally.1 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. “Our premise is th...