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October 9, 2025, AviadoBio andUgeneX (UgeneX Therapeutics) announced that the two parties have reached an exclusive option and license agreement for the development and commercialization of the optogenetics pipeline UGX-202. UGX-202 is an adeno-associated virus (AAV) gene therapy currently in clinical development for the treatment of patients with retinitis pigmentosa (RP). A second undisclosed indication is expected to enter the clinical stage by the end of the year.According to the terms of the agreement, AviadoBio has the right to opt for a global exclusive license for the development and commercialization rights of UGX-202 in RP and other indications outside the Greater China region. If this option is exercised, UgeneX will be eligible to receiveUp to 413 million US dollars in upfront payments, R&D milestone payments, and sales milestone payments, as well as royalties on net sales.In many retinal diseases, patients' photoreceptors (cells responsible for detecting light and converting it into visual signals) gradually degenerate. Optogenetics can reprogram the remaining cells in the retina to replace the function of lost photoreceptors, restoring vision regardless of the type of underlying genetic mutation.UGX202 is UgeneX's next-generation optogenetics investigational product, utilizing a self-developed AAV virus as a vector to carry an innovative broad-spectrum and highly sensitive photosensitive protein. It specifically infects retinal ganglion cells and confers light-sensing capabilities, reconstructing the visual pathway for patients with mid-to-late stage photoreceptor degenerative diseases. As a "universal" innovative gene therapy that transcends genetic mutations, UGX202 overcomes the limitations of rare disease patient numbers and is applicable to multiple patient populations, including those with retinitis pigmentosa, Stargardt disease, choroideremia, and advanced dry age-related macular degeneration.The capsid, regulatory sequences, and opsin transgene of UGX-202 have all been optimized, with the potential to achieve best-in-class clinical performance.
Dr. Kai Wu, President of UgeneXRepresentation："We are delighted to collaborate with AviadoBio to advance the clinical application of ophthalmic gene therapy and promote global cooperation."UgeneX has been committed to developing innovative gene therapies for ophthalmology, improving the quality of life for patients with visual impairments. This collaboration not onlyReflects the commitment of both parties toHigh Confidence in the Future Clinical Potential and Market Prospects of UGX202, and at the same timeIs an important milestone for our company to step into the international market, fullyReflectTo usThe team's international perspective and R&D capabilities, as well as the global value of the pipeline。”
AviadoBio's current R&D pipeline is centered around AVB-101, a one-time administration AAV gene therapy being evaluated in a Phase 1/2 clinical study for the treatment of frontotemporal dementia patients carrying GRN gene mutations. The therapy has drawn attention from Astellas, which last year paid $50 million to secure an option license for the drug.
AviadoBio CEO Lisa Deschamps stated"We are excited to expand our clinical-stage drug portfolio covering neurodegenerative diseases from the eye to the brain, leveraging our core expertise in targeted delivery, potent gene payloads, and rapid translation. This now includes retinitis pigmentosa (RP) and other retinal diseases. Optogenetics is an exciting field with clinical precedent, offering clear opportunities for next-generation approaches like UGX-202 to improve vision in patients with retinal diseases. Our team has extensive experience in ocular gene therapy development, and we look forward to collaborating with the UgeneX team to advance the UGX-202 development pipeline."AAV gene therapy, with its advantages of precise delivery and long-lasting efficacy, has become the core growth engine in the field of gene therapy, holding immense potential for future development.According to incomplete statistics, in the past five years, China's AAV gene therapy sector has experienced a "surge," with over 50 companies entering the field. The first domestically produced AAV gene therapy, BBM-H901, has been approved for marketing in China. Additionally, 50 AAV gene drugs have received IND approval, eight of which have entered Phase III clinical trials.The collaboration between UgeneX and AviadoBio represents not only the overseas advancement of a single pipeline but also a microcosm of China's biopharmaceutical industry transitioning from "importing" to "exporting." As UGX202 progresses clinically in global markets,Is expected toChina-produced AAV gene therapy establishes global recognition.Reference: Company Announcement
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