AviadoBio Expands Gene Therapy Portfolio with $413M Chinese Deal

England-based AviadoBio has secured an option to license a promising gene therapy for rare eye diseases from Chinese biotech UgeneX Therapeutics. The deal, potentially worth up to $413 million, marks a significant expansion of AviadoBio's gene therapy pipeline and highlights growing cross-border collaborations in the biotechnology sector.

UGX-202: A Novel Approach to Retinitis Pigmentosa

The centerpiece of the agreement is UGX-202, an AAV-based gene therapy developed by UgeneX for the treatment of retinitis pigmentosa (RP). This innovative therapy utilizes optogenetics, delivering a light-sensitive protein to retinal cells through a one-time, intravitreal injection. UGX-202 is currently undergoing clinical trials, with a study in RP already in progress and plans for a second trial in an undisclosed indication set to begin by year's end.

AviadoBio CEO Lisa Deschamps expressed enthusiasm for the potential of optogenetics in treating retinal diseases, stating, "Optogenetics is an exciting field with clinical precedent and clear opportunity for next-generation approaches, like UGX-202, to improve vision for people with retinal disease."

Deal Structure and Global Implications

Under the terms of the agreement, AviadoBio has secured an option for the exclusive worldwide license to develop and commercialize UGX-202 outside of China. Should AviadoBio exercise this option, UgeneX stands to receive upfront payments, R&D and sales milestones totaling up to $413 million, as well as sales-based royalties upon commercialization.

This collaboration represents a significant step for UgeneX in entering the global market. UgeneX President Wu Kai commented on the deal's importance, saying it "demonstrates the R&D capabilities and global potential of our pipeline."

For AviadoBio, the agreement expands its portfolio beyond neurodegenerative conditions of the brain to include ocular gene therapies. Deschamps highlighted the company's expertise in this area, noting, "Our team has deep experience in ocular gene therapy development, and we look forward to partnering with the UgeneX team and building a pipeline within UGX-202 together."

Strategic Expansion of AviadoBio's Gene Therapy Focus

This latest move builds upon AviadoBio's existing gene therapy pipeline, which is led by AVB-101, a phase 1/2 candidate for frontotemporal dementia with GRN gene mutations. The company's approach to targeted delivery and potent genetic payloads has already attracted attention from major players in the pharmaceutical industry, with Astellas securing a $50 million option to license AVB-101 last year.

The addition of UGX-202 to AviadoBio's portfolio demonstrates the company's commitment to advancing gene therapies across multiple therapeutic areas. As the field of gene therapy continues to evolve, collaborations such as this between AviadoBio and UgeneX may become increasingly common, potentially accelerating the development of innovative treatments for patients with rare and challenging diseases.