AviadoBio Licenses Optogenetics Gene Therapy UGX-202 for Retinal Diseases

Oct 09 , 2025
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LONDON and SHANGHAI – October 9, 2025 – AviadoBio Ltd. 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. UGX-202 is currently in clinical development for retinitis pigmentosa (RP), with a second, undisclosed indication expected to enter the clinic by the end of the year.

Optogenetics Mechanism Aims to Restore Vision

UGX-202 is a novel, one-time treatment that employs visual optogenetics, delivered via an AAV gene therapy, to restore vision in people with retinal degeneration. The therapy works by delivering a light-sensitive protein to preserved cells in the retina. In diseases like RP, the photoreceptors responsible for detecting light are lost. Optogenetics effectively reprograms the remaining retinal cells to assume the function of the lost photoreceptors, aiming to re-establish vision irrespective of the underlying genetic cause. The therapy’s capsid, regulatory sequences, and opsin transgene have all been optimized for potential best-in-class performance.

Lisa Deschamps, CEO of AviadoBio, stated that optogenetics is an exciting field with a clear opportunity for next-generation approaches, like UGX-202, to improve vision for people with retinal disease. UGX-202 has the potential to treat RP as well as multiple other inherited retinal diseases and macular degeneration.

Deal Terms and Market Need

Under the terms of the agreement, AviadoBio holds the option for an exclusive worldwide license outside of Greater China for UGX-202. If the option is exercised, UgeneX stands to receive upfront payments, research and development milestones, and sales milestone payments of up to USD $413 million, plus royalties on net sales. Dr. Wu Kai, President of UgeneX, called the partnership a significant milestone for entering the global market.

Retinitis pigmentosa (RP) is a group of progressive inherited retinal diseases and a leading cause of visual disability and blindness, affecting over 1.5 million people worldwide. The condition is characterized by the degeneration of photoreceptors, leading to progressive symptoms like night blindness, “tunnel vision,” and eventual legal blindness in many patients by ages 40 to 50.

Source:

https://aviadobio.com/aviadobio-and-ugenex-therapeutics-announce-exclusive-option-license-agreement/

About PackGene

PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.

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