Adverum Biotechnologies is preparing to start the final phase (Phase 3) of testing for Ixo-vec, a new gene therapy for people with age-related macular degeneration (AMD), a common eye disease that affects the central area of the retina and causes vision loss. AMD can be “dry” or “wet”. The dry type is more common and vision loss is usually gradual. The wet type is more severe, and vision loss can occur quickly. Ixo-vec is designed to treat wet AMD.

What Is Ixo-vec?

Ixo-vec is a one-time treatment given through a shot into the gel-like center of the eye. This type of injection is commonly done in a doctor’s office. Ixo-vec is designed to stop harmful proteins, called VEGFs, from creating leaky blood vessels that damage the retina and cause vision loss. 

Unlike current treatments that require regular shots every one to three months for the rest of a patient’s life, Ixo-vec may work for many years with just one injection. This could greatly reduce the time and stress involved with treatment.

Strong Results and Fewer Injections Needed

In the 52-week LUNA study with 60 patients:

• Ixo-vec cut the need for extra treatments by:
  • 88% for the low-dose group and 
  • 92% for the high-dose group. 
• Over 50% of the patients in the low-dose group and nearly 70% in the high-dose group didn’t need any extra treatments. 

In the four-year OPTIC study with 30 patients, the treatment burden was reduced by 86%, with about half of the patients avoiding extra injections. Ixo-vec was generally safe, and any eye inflammation was managed with standard steroid drops.

The Phase 3 Studies

Based on these encouraging results, the company will run two large studies: one called ARTEMIS, with about 284 patients in the U.S., and another, not yet named, that will include patients in the U.S., Canada, Europe, Latin America, and the Asia Pacific. 

ARTEMIS, the U.S.-based study with locations in Arizona, California, and Florida, is currently recruiting participants. 

*Foundation Fighting Blindness. (2025, February 28). Adverum Advancing Wet AMD Gene Therapy into Phase 3 Clinical Trials. Retrieved from https://www.fightingblindness.org/news/adverum-advancing-wet-amd-gene-therapy-into-phase-3-clinical-trials-1894