AviadoBio secures option to rare eye disease gene therapy that could bring Chinese biotech $413M

England's AviadoBio is looking to China as a potential source of additional gene therapies, securing an option to a $413 million biobucks deal that could deliver a clinical-stage rare eye disease candidate.

The asset that has caught AviadoBio’s eye is UGX-202, an AAV gene therapy developed by China’s UgeneX Therapeutics. The one-time, intravitreally delivered candidate is designed to deliver a light-sensitive protein to cells in the retina.

UGX-202 is already undergoing a clinical trial for retinitis pigmentosa (RP), and plans are in place to start a second study in an undisclosed indication by the end of the year, according to the Oct. 9 release.

Under this morning’s agreement, U.K.-based AviadoBio has secured the option to later pick up the exclusive worldwide license outside of China to develop and commercialize UGX-202 in RP and other indications.

Should AviadoBio take up its option, UgeneX will be in line to receive upfront payments, R&D and sales milestones that could reach a combined total of $413 million. The Chinese biotech is also eligible for sales-based royalties should UGX-202 make it to market.

AviadoBio’s current pipeline is led by AVB-101, a one-dose AAV-based gene therapy being evaluated in a phase 1/2 study in patients with frontotemporal dementia with GRN gene mutations. The therapy has already caught the attention of Astellas, which last year paid $50 million for the option to license the drug.

“We are thrilled to expand our portfolio of clinic-stage assets that span neurodegenerative conditions from the eye to the brain, united by our core expertise in targeted delivery, potent genetic payloads, and rapid translation to now include RP and other retinal diseases,” AviadoBio CEO Lisa Deschamps said in this morning’s release.

“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,” Deschamps added. “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.”

In a statement, UgeneX President Wu Kai commented that the deal with AviadoBo marks a “significant milestone for our company toward entering the global market and also demonstrates the R&D capabilities and global potential of our pipeline.”