AviadoBio, UgeneX Partner on UGX-202 Gene Therapy for RP

AviadoBio and UgeneX Therapeutics have announced an exclusive option and license agreement for the development and commercialization of UGX-202, an investigational gene therapy currently in clinical development for retinitis pigmentosa (RP). The agreement also includes a second, undisclosed indication expected to enter clinical trials by the end of the year.

UGX-202: Optogenetic Approach to Vision Restoration

UGX-202 is an adeno-associated virus (AAV)-based gene therapy that utilizes a visual optogenetics approach. It delivers a light-sensitive protein to retinal cells, enabling the reprogramming of surviving retinal cells to perform the function of degenerated photoreceptors.

In retinal diseases such as RP, the progressive degeneration of photoreceptors leads to significant visual impairment and eventual blindness. Unlike conventional gene therapies that target specific mutations, UGX-202 aims to restore visual function regardless of genetic cause, offering broad applicability.

The platform includes a suite of optimized components, engineered capsids, regulatory elements, and a proprietary opsin transgene, designed for improved safety, efficacy, and cellular targeting, with the goal of establishing UGX-202 as a best-in-class candidate in the optogenetics space.

Strategic Terms and Global Rights

Under the agreement, AviadoBio will hold an exclusive option to license global development and commercialization rights to UGX-202 outside of Greater China. Should the option be exercised, UgeneX stands to receive up to $413 million in a combination of upfront payments, research funding, development and commercial milestone payments, as well as tiered royalties on global net sales.

Executive Perspectives

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