UgeneX Therapeutics Enters $413M Global Licensing Deal for AAV-Based Optogenetic Therapy UGX-202 Targeting Retinal Degenerative Diseases

October 9,SuzhouUgeneX Therapeutics Co., Ltd. (referred to as "UgeneX") andSuzhou UgeneX Therapeutics Co., Ltd. and the UK's AviadoBio jointly announced that the two parties have signed an exclusive option and license agreement to co-develop and commercialize a novel gene therapy named UGX-202, which aims to restore vision for patients with retinal degenerative diseases through optogenetics technology.

UGX-202 is an innovative, single intravitreal injection AAV (adeno-associated virus) vector gene therapy drug that utilizes cutting-edge optogenetics technology to deliver light-sensitive proteins to retinal ganglion cells, "reprogramming" them into cells with photoreceptive capabilities. This replaces the lost rod/cone cells due to disease and rebuilds the visual pathway. The unique feature of this therapy is that its effectiveness does not depend on the specific type of pathogenic genetic mutation in patients, offering hope to individuals suffering from various inherited retinal diseases.

Currently, UGX-202 is being used in an investigator-initiated clinical trial to treat patients with Retinitis Pigmentosa (RP), a progressive hereditary eye disease. There are over 1.5 million patients worldwide, and there is currently no effective cure, making the clinical need urgent.In addition to RP, another indication for this therapy is also planned to enter the clinical research stage by the end of this year.

According toUgeneXIntroduction: UGX-202 has undergone systematic optimization in terms of viral vector design, regulatory sequences, and photosensitive protein genes, demonstrating the potential to become a best-in-class therapy. It also holds promise for expanding into the treatment of other hereditary retinal diseases and major blinding eye conditions such as age-related macular degeneration.

According to the terms of the agreement, AviadoBio will obtain the exclusive option for the development and commercialization of UGX-202 in global markets outside of Greater China. Upon exercising this option,UgeneXEntitled to receive cumulative payments of up to $413 million (approximately RMB 3 billion), including upfront payment, R&D and sales milestone payments, as well as future sales royalties.

This collaboration combines the R&D capabilities of China's local innovative enterprises with the global development experience of international biotechnology companies, marking that China's gene therapy achievements are accelerating onto the world stage, and also bringing new hope to millions of retinal disease patients worldwide.

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