A Landmark Partnership in Gene Therapy
AviadoBio has obtained an exclusive option for a gene therapy targeting rare eye diseases in a deal that could be worth up to $413 million for Chinese biotech UgeneX Therapeutics.
The partnership centers on UGX-202, an AAV-based therapy developed by UgeneX and designed for intravitreous, one-time administration to deliver a light-sensitive protein into retinal cells. The candidate is currently in clinical trials for retinitis pigmentosa (RP), with a second study in another unspecified retinal indication expected to begin by the end of the year.
Terms of the Agreement
Under the agreement, UK-based AviadoBio has secured an option to obtain exclusive worldwide rights, excluding China, to develop and commercialize UGX-202 for RP and other indications. If the option is exercised, UgeneX will receive upfront payments, R&D milestones, and sales-based payments totaling up to $413 million, along with royalties on future sales.
Expanding Clinical Pipelines
AviadoBio CEO Lisa Deschamps said the collaboration strengthens the company’s therapy pipeline:
“We are excited to expand our portfolio of clinical-stage assets that bridge the eye and brain. Our work in targeted delivery, potent genetic payloads, and rapid clinical translation now extends to RP and other retinal diseases through this partnership with UgeneX,” she said.
AviadoBio’s lead program, AVB-101, is an AAV-based, one-time therapy for frontotemporal dementia (FTD) with GRN mutations, currently in a Phase 1/2 clinical trial. The program previously attracted Astellas Pharma, which paid $50 million for an option to license the asset.
A Move into the Global Market
UgeneX President Wu Kai described the deal as a major step forward for the company’s entry into the global therapy market, showcasing the innovation and R&D capacity of its growing pipeline.
The agreement marks a significant move in the retinal therapy space, an emerging frontier aimed at restoring or improving vision in patients with retinal degenerative diseases such as retinitis pigmentosa.
Strategic Value & Market Dynamics
From a strategic standpoint, the deal underscores how biotechs are increasingly looking globally — including China — for novel medicine assets. AviadoBio’s willingness to commit up to $413 million sends a signal that therapies remain an attractive area for high-risk, high-reward investment. Meanwhile, it’s ability to partner outside China suggests Chinese biotech innovation is gaining international traction.
On the market side, the therapy field is crowded but increasingly differentiated — optogenetics is one of the newest approaches within retinal disease. If UGX-202 succeeds, it could establish AviadoBio as a major player in ocular therapies (in addition to its neuro-degenerative focus).
How the Gene Therapy Works
UGX-202 is built on AAV delivery technology, a well-established approach that uses adeno-associated viruses as carriers to introduce corrective genetic material into targeted cells. In this case, the therapy delivers a gene encoding a light-sensitive opsin protein that helps retinal cells regain responsiveness to light — a process that mimics natural vision function. This one-time therapy is designed for intravitreal injection, making it less invasive than subretinal procedures used in some other ocular therapy programs.
Researchers at note that the candidate has demonstrated strong safety and early efficacy signals in preclinical models, showing potential to restore partial light perception and improve visual function. If proven successful in human trials, the therapy could represent one of the first intravitreal optogenetic therapies to enter commercial use.
Conclusion
This landmark therapy option agreement between AviadoBio sets a new benchmark in rare-eye disease treatment collaborations. By advancing UGX-202, the companies are not only targeting a large unmet need in retinitis pigmentosa and other retinal disorders but also redefining how therapy assets are developed and commercialised globally. The next several years will be critical in validating the approach, and if successful, could pave the way for broader applications of therapy in the eye and beyond.
