Gene Therapy Pipeline Update: Adverum Biotechnologies Doses First Patient for Wet AMD

Adverum Biotechnologies, a clinical-stage gene therapy company developing drugs to teated rare ocular diseases, announced that the first patient was dosed in the third cohort (n=9) of the ongoing OPTIC phase 1 clinical trial (NCT03748784) for ADVM-022 for the treatment of neovascular or wet age-related macular degeneration (wet AMD).

What is ADVM-022: ADVM-022 utilizes a propriety vector capsid, AAV.7m8, carrying an aflibercept coding sequence under the control of a proprietary expression cassette. ADVM-022 is administered as a one-time intravitreal injection, designed to deliver long-term efficacy, reduce the burden of frequent anti-VEGF injections, optimize patient compliance, and to improve vision outcomes for wet AMD and diabetic retinopathy patients.

What is wet AMD: Around 10-15% of the cases of macular degeneration are classified under wet AMD. In this type of macular degeneration, abnormal blood vessels grow under the retina and macula. These new blood vessels may then bleed and leak fluid, causing the macula to bulge or lift up from its normally flat position, thus distorting or destroying central vision. Under these circumstances, vision loss may be rapid and severe.

What is OPTIC Phase 1 clinical trial: It is an open-label, multicenter, dose-escalation study designed to evaluate 2 dose levels in up to 30 subjects (15 per dose) with active choroidal neovascularization (CNV) secondary to AMD. The primary endpoint for this study is the safety and tolerability of ADVM-022. All subjects will continue to be assessed for 104 weeks following treatment with ADVM-022.

What other gene therapies are being developed for ophthalmic diseases: Currently, close to 60 gene therapies are in development for the treatment of patients with ophthalmic diseases.

How many candidates are in the gene therapy pipeline for the treatment of AMD: According to a recent market report by Roots Analysis, AMD is the leading indication amongst ophthalmic diseases, for which gene therapies are being developed.

To get more insights about the market, check out the detailed report here.