AviadoBio and UgeneX Therapeutics Announce Agreement to Advance Gene Therapy for Retinitis Pigmentosa
AviadoBio and UgeneX Therapeutics announced an exclusive option and license agreement for the development and commercialization of UGX-202, an investigational AAV-based gene therapy currently in clinical development for retinitis pigmentosa (RP). The agreement also includes a second undisclosed indication expected to enter the clinic by year-end.
UGX-202 is an adeno-associated virus (AAV) gene therapy designed to restore vision through visual optogenetics. The therapy delivers a light-sensitive protein to retinal cells, effectively reprogramming preserved cells in the retina to take on the function of lost photoreceptors.
In retinal diseases such as RP, the progressive loss of photoreceptors leads to severe visual impairment and blindness. By enabling remaining retinal cells to respond to light, optogenetics bypasses the need for functioning photoreceptors and has the potential to restore vision regardless of the underlying genetic cause.
The UGX-202 platform incorporates optimized capsid design, regulatory elements, and a proprietary opsin transgene, all engineered for enhanced safety, efficacy, and cellular targeting. These refinements aim to position UGX-202 as a best-in-class therapeutic candidate in the emerging field of optogenetic vision restoration.
Under the terms of the agreement, AviadoBio will have the exclusive option to license worldwide development and commercialization rights to UGX-202 outside of Greater China. Should AviadoBio exercise the option, UgeneX would be eligible to receive up to $413 million in upfront, research, development, and sales milestone payments, in addition to tiered royalties on global 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,” 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.”