AviadoBio & UgeneX Therapeutics Announce Exclusive Option-License Agreement to Develop Next Generation Optogenetics Gene Therapy to Restore Vision in Patients With Retinal Disease


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 in clinical development for patients with retinitis pigmentosa (RP), with a second undisclosed indication entering the clinic by year-end.

UGX-202 is an AAV gene therapy that employs visual optogenetics to deliver a light-sensitive protein to cells in the retina. In many retinal diseases, patients experience a loss of photoreceptors – cells responsible for detecting light and converting it into visual signals for the brain. Optogenetics reprograms preserved cells in the retina to take on the function of the lost photoreceptors, thus re-establishing vision irrespective of the underlying genetic cause. The UGX-202 capsid, regulatory sequences, and opsin transgene have all been optimized for potential best-in-class performance.

Under the terms of the agreement, AviadoBio will have the option to receive a worldwide exclusive license outside of Greater China for the development and commercialization rights to UGX-202 in RP and other indications. If the option is exercised, UgeneX will be eligible to receive upfront payments, research and development milestones, and sales milestone payments of up to USD $413 million, as well as royalties on net sales.

“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. 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,” said Lisa Deschamps, CEO, AviadoBio. “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.”

“We are delighted to partner with AviadoBio to jointly promote the clinical application and global collaboration of ophthalmic gene therapy,” said Dr. Wu Kai, President of UgeneX. “This partnership is a significant milestone for our company toward entering the global market and also demonstrates the R&D capabilities and global potential of our pipeline.”