Advancing Novel Gene Therapies

We are advancing a pipeline of gene therapy product candidates targeting the treatment of ocular diseases.

ADVM-022 Overview

ADVM-022 utilizes a propriety vector capsid, AAV.7m8, carrying an aflibercept coding sequence under the control of a proprietary expression cassette. ADVM-022 (AAV.7m8-aflibercept) is administered as a single, in-office intravitreal (IVT) injection, designed to deliver long-term efficacy, reduce the burden of frequent anti-VEGF injections, and improve vision outcomes for patients with wet age-related macular degeneration (wet AMD).


ADVM-022 for Wet AMD

In recognition of the need for new treatment options for wet AMD, the U.S. Food and Drug Administration (FDA) granted Fast Track designation to ADVM-022 for the treatment of wet AMD.

OPTIC Trial of ADVM-022 in Wet AMD

The OPTIC trial is designed as a multi-center, open-label, dose-escalation trial evaluating the safety and tolerability of a single intravitreal (IVT) injection of ADVM-022. In our OPTIC trial, we enrolled difficult-to-treat wet AMD patients who required frequent anti-VEGF injections to maintain their vision.

Long-term data from the OPTIC trial (n=30) of ADVM-022 for wet AMD have demonstrated durable efficacy following a single, in-office IVT injection. Safety and efficacy data presented at the Association for Research in Vision and Ophthalmology (ARVO) in April 2021 showed 60% of patients were injection-free beyond one year and patients had an 85% reduction in annualized injection frequency following a single low dose. All ADVM-022-related ocular adverse events (AE) were mild (80%) to moderate (20%). No clinical or fluorescein evidence of posterior inflammation and no hypotony, vasculitis, retinitis, choroiditis, vascular occlusions, or endophthalmitis were observed.

Patients completing two years in OPTIC are being enrolled into an extension trial to be followed for up to five years. For more information about the clinical trial, please visit

For updates on the OPTIC trial, please visit the press releases section of our website.

About Wet AMD

Age-related macular degeneration (AMD) is a progressive disease affecting the macula, the region of the retina at the back of the eye responsible for central vision. In patients with neovascular or wet AMD, an aggressive form of AMD, abnormal blood vessels grow underneath and into the retina. These abnormal blood vessels leak fluid and blood into and beneath the retina, causing vision loss.

Wet AMD is a leading cause of vision loss in patients over 60 years of age, with a prevalence of approximately 1.2 million individuals in the U.S. and 3 million worldwide1. The incidence of new cases of wet AMD in the U.S. is approximately 150,000 to 200,000 annually, and this number is expected to grow significantly as the country’s population ages2,3.

Although VEGF is known to play a central role in the growth of new blood vessels in wet AMD, the underlying molecular causes of the disease are not completely known. Current standard-of-care therapies for wet AMD are proteins that bind VEGF and neutralize its activity. These therapies can be burdensome, as patients generally require chronic IVT injection of anti-VEGF protein every 4-12 weeks. Compliance with this regimen can be difficult for patients and their caregivers, leading to compliance deficiencies and loss of vision from underdosing.

Resources for wAMD


  1. Arch Ophthalmol. 2004;122(4):564-572. doi:10.1001/archopht.122.4.564.
  2. Brown GC, Brown MM, Sharma S, et al. The Burden of Age-Related Macular Degeneration: A Value-Based Medicine Analysis. Transactions of the American Ophthalmological Society. 2005.
  3. California Retina Consultants. Advances in Wet AMD. Available at: