Ocugen, a Malvern, Pennsylvania-based biotechnology company developing gene therapies for blindness diseases, has granted Roots Pharmaceutical exclusive rights to its investigational retinitis pigmentosa (RP) treatment across the Middle East and North Africa (MENA). The company announced on October 14, 2026 that it signed the licensing agreement for OCU400, a modifier gene therapy, with Roots Pharmaceutical and its strategic partner, Al-Dhow International Holding.
“By partnering with Roots Pharmaceutical, an established leader with strong reach across the Middle East and North Africa, we are expanding our ability to bring our potential one-time gene therapy to patients in a region where retinitis pigmentosa is highly prevalent and the unmet medical need is significant,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer and Co-founder of Ocugen.
Inside the Deal
Under the agreement, Ocugen will receive an upfront license fee and potential initial development milestone payments totaling up to $4 million. It could also earn up to $255 million in sales milestones, plus royalties of 22% on OCU400 net sales generated by Roots Pharmaceutical.
Ocugen will manufacture the commercial supply of OCU400 under a separate supply agreement.
“This agreement represents another important step in our strategy to build strong regional partnerships and expand global access to our potentially transformational gene therapies,” Musunuri added.
Why the MENA Region
Ocugen considers RP a leading cause of inherited vision loss worldwide. The company estimates that RP is 10 to 20 times more prevalent in the MENA region than in the U.S. and Europe. RP causes progressive vision loss and can lead to blindness.
Roots Pharmaceutical brings more than two decades of experience commercializing medicines in the region, according to the release. Ocugen points to its regional capabilities and healthcare relationships as key to bringing OCU400 to patients there.
“This partnership reflects Roots’ commitment to bringing breakthrough gene therapies to the Middle East and North Africa, leading and shaping the treatment paradigm for our patients, which is our main objective,” said Dr. Islam Zayed, Chief Executive Officer and Co-Founder of Roots Pharmaceutical. “OCU400 has the potential to change the outlook, and the lives, of patients living with retinitis pigmentosa.”
What’s Next for OCU400
OCU400 is being evaluated in liMeliGhT, a Phase 3 clinical trial. Ocugen expects topline data in the first quarter of 2027. The company then plans to file for approval in the U.S. and Europe, through a Biologics License Application (BLA) and a Marketing Authorization Application (MAA), respectively.
Roots Pharmaceutical is already looking beyond OCU400. “We look forward to working closely with Ocugen to build the diagnosis-to-treatment pathway that gene therapy requires across our markets, and to exploring further collaboration across Ocugen’s ophthalmology pipeline,” Zayed said.
About Ocugen
Ocugen’s modifier gene therapy platform takes a gene-agnostic approach. Rather than targeting a single gene mutation, its therapies are designed to address underlying disease biology by restoring balance across multiple gene networks. Beyond RP, the company is developing programs for Stargardt disease and geographic atrophy, an advanced form of dry age-related macular degeneration.
About Roots Pharmaceutical
Founded in Kuwait in 2006, Roots Pharmaceutical commercializes therapies, medical devices, and an aesthetics line. It has a direct commercial presence mainly in Gulf Cooperation Council (GCC) countries and key North African countries. Its portfolio spans neurology, hematology, oncology, rare diseases, dermatology, and cardiovascular care, and the company works with more than 30 global partners.
