Ocugen signs term sheet for korean licensing of OCU400 gene therapy

Ocugen has announced the signing of a binding term sheet to negotiate and enter into a licensing agreement with a “well-established leader in the pharmaceutical and healthcare sector in Korea” for exclusive Korean rights to OCU400.

OCU400 is Ocugen’s novel modifier gene therapy for retinitis pigmentosa (RP). The OCU400 (AAV5-hNR2E3) gene therapy platform delivers the nuclear hormone receptor NR2E3 to the retina and retinal pigment epithelium (RPE) to preserve and potentially restore retinal structure and function.

Under the license agreement, Ocugen will receive upfront license fees and near-term development milestones up to $11 million. Ocugen will be entitled to sales milestones of $1 million for every $15 million of net sales in Korea, in addition to a royalty of 25% on net sales of OCU400 generated by the unnamed partner. Manufacturing of OCU400 will be conducted by Ocugen.

According to a press release from the company, there are an estimated 15,000 individuals in the Republic of Korea with RP.

Shankar Musunuri, PhD, MBA, chairman, CEO, and co-founder of Ocugen, commented on the agreement in a press release from the company, saying, “This regional licensing agreement is aligned with our business development strategy to partner with well-established companies in their respective countries and regions—leveraging their networks and know-how to treat as many RP patients as possible.”

Ocugen noted that additional details of the deal will be available when the definitive agreement between the parties is executed, which is expected to happen within the next 60 days.

Earlier this year, Ocugen announced a positive 2-year safety and efficacy update for the phase 1/2 OCU400 clinical trial for the treatment of early to advanced RP in pediatric and adult populations. According to the company, OCU400 demonstrated meaningful improvement of 2-line gain (10 letters on ETDRS chart) in low-luminance visual acuity (LLVA) in treated eyes when compared to untreated fellow eyes.

Ocugen is currently advancing OCU400 through phase 3 clinical development (liMeliGhT) with a target Biologics License Application filing in mid-2026.

References:

1. Ocugen, Inc. announces signing of binding term sheet for the license of OCU400 modifier gene therapy for retinitis pigmentosa in Korea. Published June 5, 2025. Accessed June 5, 2025. https://www.globenewswire.com/news-release/2025/06/05/3094247/0/en/Ocugen-Inc-Announces-Signing-of-Binding-Term-Sheet-for-the-License-of-OCU400-Modifier-Gene-Therapy-for-Retinitis-Pigmentosa-in-Korea.html

2. Crago S. Ocugen announces positive 2-year safety and efficacy update of Phase 1/2 OCU400 for treatment of retinitis pigmentosa. Published January 15, 2025. Accessed June 5, 2025. https://www.ophthalmologytimes.com/view/ocugen-announces-positive-2-year-safety-and-efficacy-update-of-phase-1-2-ocu400-for-treatment-of-retinitis-pigmentosa