Ocugen Announces Key Licensing Agreement for OCU400 in South Korea: A Novel AAV Gene Therapy for RP

MALVERN, Pa. – Ocugen, Inc., a pioneering biotechnology leader in gene therapies for blindness diseases leveraging AAV (adeno-associated virus) technology, announced today a binding term sheet for a licensing agreement with a prominent Korean pharmaceutical and healthcare company. This agreement grants exclusive Korean rights for OCU400—Ocugen’s innovative modifier gene therapy for retinitis pigmentosa (RP).

Understanding OCU400

OCU400 is an investigational gene therapy developed by Ocugen, Inc. It uses an AAV to deliver a modified version of the nuclear hormone receptor gene, NR2E3, to the retina. This gene therapy aims to address a broad range of inherited retinal diseases (IRDs) by restoring retinal homeostasis and potentially rescuing photoreceptors from degeneration.

Agreement Details

Under the terms, Ocugen stands to receive upfront license fees and near-term development milestones totaling up to $11 million. Additionally, the company will earn $1 million in sales milestones for every $15 million in net sales of OCU400 in Korea, along with a 25% royalty on those net sales. Ocugen will also be responsible for manufacturing the commercial supply of OCU400, an AAV-based therapy.

With an estimated 15,000 individuals affected by RP in South Korea, this partnership offers a significant opportunity for the Korean licensee to become a leader in AAV gene therapy within the region while helping thousands of patients.

Strategic Vision

Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen, stated, “This regional licensing agreement aligns with a business development strategy to partner with well-established companies in their respective countries and regions—leveraging networks and know-how to treat as many RP patients as possible.” He added that this regional approach allows Ocugen to retain rights to larger geographies, maximizing patient reach and generating shareholder returns.

Ocugen is currently advancing OCU400 through Phase 3 clinical development, with a target Biologics License Application filing planned for mid-2026 for this promising AAV gene therapy.