A gene therapy for wet AMD

Adverum presents first preclinical data on dosing contralateral eye with ADVM-022

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MENLO PARK, Calif.—Adverum Biotechnologies Inc., a clinical-stage gene therapy company targeting unmet medical needs in ocular and rare diseases, presented preclinical data on April 30 for the first time on sequentially dosing a contralateral eye with ADVM-022 gene therapy in cases of wet age-related macular degeneration (wet AMD). The data were presented in a poster session at the Association for Research in Vision and Ophthalmology (ARVO) 2019 Annual Meeting in Vancouver, British Columbia and reportedly demonstrate that ADVM-022 can be administered safely to the second eye two months after injection in the first eye.
“We are excited to show that the second eye injection with ADVM-022 can be performed safely at the time of peak immune response to this therapy injected into the first eye. It is encouraging to see that levels of expression in the second eye are still within the aflibercept therapeutic levels,” said Dr. Mehdi Gasmi, president and chief scientific officer of Adverum Biotechnologies. “The findings from this study highlight ADVM-022’s potential for the treatment of patients with pre-existing vector immunity or bi-lateral disease.”
Highlights from the poster included:
  • Sequential intravitreal injections of ADVM-022 at 2x1012 vg in one eye followed by the contralateral eye two months later induced sustained expression of aflibercept in ocular compartments of the second eye, including vitreous, aqueous humor, retina, and choroid for up seven months.
  • Sequential injections of ADVM-022 were well tolerated, as assessed by ophthalmic examinations and histopathology. Minimal perivascular infiltrates and mild inflammation were observed, comparable between first and second eyes. Average retinal thickness and volume assessed by optical coherence tomography was unchanged in both eyes for the duration of the study.
  • When assessing immunological response, the development of immunity following ADVM-022 in one eye does not block transduction following sequential dosing in the contralateral eye in non-human primates.
Adverum’s gene therapy candidate for wet AMD, ADVM-022, utilizes a proprietary vector capsid (AAV.7m8) carrying an aflibercept coding sequence under the control of a proprietary expression cassette and is administered as a single intravitreal administration. ADVM-022 is designed to provide sustained therapeutic levels of aflibercept, minimize the burden of frequent anti-VEGF injections, and improve real-world vision outcomes for patients with wet AMD.
Age-related macular degeneration is a progressive disease affecting the retinal cells in the macula, the region of the eye responsible for central vision. Disease progression results in the death of retinal cells and the gradual loss of vision. Wet AMD is an advanced form of AMD, affecting approximately 10 percent of patients living with AMD. In patients with wet 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. In most patients, wAMD develops in both eyes at different times.
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 United States and 3 million worldwide. The standard-of-care therapy for wet AMD is anti-VEGF intravitreal injections. These are effective, but typically require long-term eye injections every four to eight weeks in order to fully maintain vision gains. Compliance with this regimen can be difficult for patients, caregivers and healthcare systems, leading to suboptimal dosing and loss of vision from undertreatment.

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