Chinese Ophthalmic AAV Gene Therapy UGX202 Secures Over $400M Overseas Licensing Deal Ahead of IPO

Recently, SuzhouUgeneX (referred to as "UgeneX") and AviadoBio LtdAviadoBio Reaches Strategic Agreement to Advance Optogenetics Pipeline UThe Exclusive Option for Overseas Development and Commercialization of GX202 GrantedAviadoBio。

Can attractAviadoBio's favor,UgeneX andUGX202, What's the Charm?

UgeneX is a young Biotech, founded in July 2021, but with a deep background. According to reports,UgeneX has received cumulative investments exceeding 100 million yuan from its establishment to the present. The company's team has over 15 years of practical experience in the research and development of innovative therapies in ophthalmology and neurobiology.

Currently,UgeneX has built three core platforms – optogenetics platform, neuroregeneration platform, and AAV vector development platform, based on these three...The large platform has created multiple BIC/FIC ophthalmology pipelines, with indications coveringRetinitis Pigmentosa, Dry Age-Related Macular Degeneration, and Other Retinal Diseases.

Image Source:UgeneX Official Website

UGX202YesUgeneX's leading project has advanced to the clinical research stage.Section, currently evaluating and exploringSafety and Efficacy in Patients with Advanced Retinitis Pigmentosa.

UGX202 developed by UgeneXAAV virus as a vector, carrying innovative photosensitive proteins with a wide spectrum and high sensitivity, specifically infecting the retina.Ganglion cells and confer photoreceptive functions, reconstructing the visual pathway in patients with mid-to-late stage photoreceptor degenerative diseases.

AviadoBio is a UK-based biotech company,Dedicated to transforming groundbreaking science and precision delivery into revolutionary medicines that improve the lives of patients with neurological disorders.

Currently, AviadoBio's R&D pipeline includes only one product, AVB-101 (AAV-PGRN) is in the clinical stage and is currently conducting a trial targeting GRN mutationsFrontotemporal Dementia（FTD-GRN）Phase 1/2 study in patients. Notably, Astellas acquired the license for AVB-101 from AviadoBio Ltd for $50 million in October 2024. Other pipelines are still in preclinical development.

For AviadoBio, the introductionUGX202 can add a clinical asset to its pipeline portfolio while expanding its focus from the neurology field to ophthalmology.

Further Reading:First Optogenetic Gene Therapy Submits BLA

In addition, the success of optogenetic gene therapy has been validated in clinical development,The first optogenetic gene therapy has submitted a BLA.——July 2025, U.S. Biotechnology CompanyNanoscope Announces Start of Rolling Submission to FDA for itsThe Gene Therapy MCO-010 Based on OptogeneticsBiologics License Application (BLA) for the treatment of severe vision loss caused by retinitis pigmentosa (RP), regardless of genetic mutation.

If optogenetic gene therapy receives FDA approval for marketing, optogenetic gene therapy will become the next breakthrough technology. Its cross-gene mutation characteristic will change the current limitation of ophthalmic gene therapy being restricted to specific gene mutations, and it may become the next big trend.

More importantly, optogenetic gene therapy is not limited to ophthalmic applications. In global development, optogenetic gene therapy also covers a variety of other disease areas, including neurology, cardiology, metabolic obesity, and more. Additionally, recent research has explored the application of optogenetics in drug screening.

References:

1.UgeneX《UgeneX Grants AviadoBio Exclusive Option for Overseas Rights of Optogenetics Pipeline UGX202

2.UgeneX "UgeneX Optogenetics Pipeline UGX202 Completes First Patient Dosing"

3.UgeneX Official Website:https://www.u-genex.com/sy

4.https://aviadobio.com/pipeline/

5.https://www.fiercebiotech.com/biotech/aviadobio-secures-option-rare-eye-disease-gene-therapy-could-bring-chinese-biotech-413m

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