By Michael Dabaie


Ocugen Inc. said the U.S. Food and Drug Administration accepted its Investigational New Drug application to initiate a clinical trial of OCU400 in retinitis pigmentosa.

The biopharmaceutical company focused on gene therapies to cure blindness diseases, said the trial, to start in the first quarter of 2022, will enroll patients with mutations in NR2E3 or RHO genes.

Ocugen shares were up 4% to $6.28 in premarket trade.

"We're collaborating with leading centers in eye care and have been vital partners to getting our trial launched and receive patients. With this final decision by the FDA, we are embarking on a new pathway of care through this innovative gene therapy," said Chief Executive Shankar Musunuri.

OCU400 was granted four orphan drug disease designations from the FDA for treating four different gene mutation-associated retinal degenerative diseases between 2019 and 2020. The European Medicines Agency granted Ocugen broad orphan medicinal product designation in 2021 for OCU400 for the treatment of both retinitis pigmentosa and Leber congenital amaurosis.


Write to Michael Dabaie at


(END) Dow Jones Newswires

December 09, 2021 08:22 ET (13:22 GMT)

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