AviadoBio Ltd. and UgeneX Therapeutics have entered into an exclusive option and license agreement for UGX-202, a novel gene therapy aimed at treating retinitis pigmentosa and other retinal diseases.

Information on the Target

UGX-202 is an innovative, one-time intravitreally delivered gene therapy that utilizes optogenetics to restore vision in individuals suffering from retinal degeneration, specifically retinitis pigmentosa (RP). Currently undergoing investigation in an investigator-initiated study, UGX-202 addresses a substantial unmet need, with over 1.5 million people affected by RP globally. This therapy not only has the potential to treat RP but could also be applicable to various other inherited retinal disorders and macular degeneration.

The mechanism of UGX-202 involves an adeno-associated virus (AAV)-based gene therapy designed to deliver a light-sensitive protein to retinal cells. As many retinal conditions lead to the deterioration of photoreceptors, which are vital for detecting light and transmitting visual signals to the brain, optogenetics aims to rejuvenate surviving retinal cells, enabling them to take over the function of the compromised photoreceptors. This process presents a promising avenue to regain vision regardless of the genetic causes underlying these diseases.

Industry Overview in the Target's Specific Country

The incidence of retinal diseases, such as retinitis pigmentosa, is a growing concern in the global healthcare landscape, with a particular emphasis on innovative therapies. In the United Kingdom, where significant advancements are being made