AviadoBio Enters License and Option Agreement with UgeneX for Next Generation Optogenetics Gene Therapy

Rare Daily Staff

AviadoBio said it entered into an exclusive option and license agreement with Shanghai-based UgeneX Therapeutics for the development and commercialization of UGX-202, an experimental AAV-based gene therapy in clinical development for patients with retinitis pigmentosa and a second, undisclosed indication entering the clinic by year’s end.

Retinitis pigmentosa (RP) is a group of progressive inherited retinal diseases (IRDs) characterized by the primary degeneration of rod photoreceptors, followed by the loss of cone photoreceptors. It is a leading cause of visual disability and blindness, affecting more than 1.5 million people worldwide. Progressive symptoms include night blindness, a reduction in peripheral and central vision—commonly called “tunnel vision”—and reduced ability to see details and colors. The progression of RP can vary widely between individuals, though many with the disease are legally blind by 40 to 50 years of age.

UGX-202 is an AAV gene therapy that uses 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 lost photoreceptors, re-establishing vision regardless 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 $413 million, as well as royalties on net sales.

“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 of 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.”

Photo: Lisa Deschamps, CEO of AviadoBio