Press Release
AviadoBio and UgeneX Therapeutics Announce Exclusive Option-License Agreement to Develop Next Generation Optogenetics Gene Therapy to Restore Vision in Patients with Retinal Disease
• UGX-202 is a novel, one-time intravitreally–delivered gene therapy that employs optogenetics with the aim to restore vision in people with retinal degeneration
• UGX-202 is currently being studied in patients with retinitis pigmentosa (RP) in an investigator-initiated study
• More than 1.5 million people living with RP worldwide represent a significant unmet need for new treatments
• UGX-202 has the potential to treat RP as well as multiple other inherited retinal diseases and macular degeneration
LONDON and SHANGHAI – October 9, 2025 – AviadoBio Ltd. (“AviadoBio”), and UgeneX Therapeutics, today announced an exclusive option and license agreement for the development and commercialization of UGX-202, an investigational, AAV-based gene therapy in clinical development for patients with retinitis pigmentosa (RP), with a second undisclosed indication entering the clinic by year-end.
UGX-202 is an AAV gene therapy that employs visual optogenetics to deliver a light-sensitive protein to cells in the retina. In many retinal diseases, patients experience a loss of photoreceptors – cells responsible for detecting light and converting it into visual signals for the brain. Optogenetics reprograms preserved cells in the retina to take on the function of the lost photoreceptors, thus re-establishing vision irrespective of the underlying genetic cause. The UGX-202 capsid, regulatory sequences, and opsin transgene have all been optimized for potential best-in-class performance.
Under the terms of the agreement, AviadoBio will have the option to receive a worldwide exclusive license outside of Greater China for the development and commercialization rights to UGX-202 in RP and other indications. If the option is exercised, UgeneX will be eligible to receive upfront payments, research and development milestones, and sales milestone payments of up to USD $413 million, as well as royalties on net sales.
“We are thrilled to expand our portfolio of clinic-stage assets that span neurodegenerative conditions from the eye to the brain, united by our core expertise in targeted delivery, potent genetic payloads, and rapid translation to now include RP and other retinal diseases. Optogenetics is an exciting field with clinical precedent and clear opportunity for next-generation approaches, like UGX-202, to improve vision for people with retinal disease,” said Lisa Deschamps, CEO, AviadoBio. “Our team has deep experience in ocular gene therapy development, and we look forward to partnering with the UgeneX team and building a pipeline within UGX-202 together.”
“We are delighted to partner with AviadoBio to jointly promote the clinical application and global collaboration of ophthalmic gene therapy,” said Dr. Wu Kai, President of UgeneX. “This partnership is a significant milestone for our company toward entering the global market and also demonstrates the R&D capabilities and global potential of our pipeline.”
About Retinitis Pigmentosa (RP)
Retinitis pigmentosa (RP) is a group of progressive inherited retinal diseases (IRDs) characterized by the primary degeneration of rod photoreceptors, followed by the loss of cone photoreceptors. It is a leading cause of visual disability and blindness, affecting over 1.5 million people worldwide.1 RP is typically diagnosed in children and young adults. However, the average age of diagnosis for patients with RP is approximately 35.1 years.2 Progressive symptoms include night blindness, reduction in peripheral and central vision, commonly called “tunnel vision,” and reduced ability to see details and colors.3 The progression of RP can vary widely between individuals, though many with RP are legally blind by 40-50 years of age.4
About UgeneX Therapeutics
UgeneX Therapeutics Co., Ltd. is a company founded in July 2021, focused on developing novel gene therapy products for ophthalmic diseases. Backed by a team of over 15 years of experience in ophthalmology and neurobiology, the company has established an innovative ophthalmic gene therapy technology platform and an internationally leading pipeline. To date, it has secured investments exceeding 100 million RMB.
References
1. Verbakel S et al. Progress in Retinal and Eye Research 2018;66:157-186;
About AviadoBio
At AviadoBio, we are relentlessly chasing cures by translating groundbreaking science and precision delivery into life-changing medicines for people living with neurological conditions. With our deep understanding of the brain and suite of proprietary gene therapy platforms and delivery technologies, AviadoBio is working to overcome the challenges of delivering the right drug to the right place. Its innovative, neuroanatomy-led approach is designed to maximize the therapeutic potential of gene therapy to halt or potentially reverse neurodegenerative diseases. AviadoBio was founded on pioneering research from King’s College London and the UK Dementia Research Institute and has a leadership team with extensive gene therapy development, delivery, and commercialization experience, which uniquely positions the company for success in bringing transformative medicines to patients.
AviadoBio’s investors include New Enterprise Associates (NEA), Monograph Capital, F-Prime Capital, Johnson & Johnson, through its corporate venture capital organization, Johnson & Johnson Innovation – JJDC, Inc., SV Health Investor’s Dementia Discovery Fund (DDF), Advent Life Sciences, EQT Life Sciences (Dementia Fund), LifeArc Ventures, and Astellas Pharma.
For more information, please visit www.aviadobio.com and follow us on X @AviadoBio and LinkedIn at AviadoBio.
Contact
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fspeer@aviadobio.com
+1-312-543-2881