Adverum Biotechnologies Inc.

Company Overview

Adverum is a clinical-stage gene therapy company targeting unmet medical needs in ocular and rare diseases. The company has developed a technique for developing viral vectors that are engineered for improved success of transfection. It has used that technology to develop a pipeline of treatments for acute macular degeneration and diabetic retinopathy for clinical use. Adverum develops gene therapy product candidates proposed to provide durable efficacy by inducing sustained expression of a therapeutic protein. Core capabilities include novel vector discovery, nonclinical and clinical development and pre-commercial planning. The company has assay development and current Good Manufacturing Practices (GMP) quality control, in-house manufacturing expertise, specifically in scalable process development,

Designed to deliver long-term durable levels of aflibercept associated with a robust, sustained treatment response, ADVM-022 (Ixo-vec) is the lead product candidate. It reduces the treatment burden and fluctuations in macular fluid associated with frequent anti-vascular endothelial growth factor (VEGF) injections. After an in-office IVT injection, ADVM-022 transduces retinal cells using a proprietary vector capsid, AAV.7m8, that carries an aflibercept coding sequence and is controlled by a proprietary expression cassette. The goal of this product is to enhance patients' quality of life and their actual vision. The business operates into a single reportable segment.

Financial Highlights (FY 2024)

Company's Business Segments

• Gene Therapy : The company develop treatments for serious ocular and rare diseases, particularly wet age-related macular degeneration (wet AMD), with a focus on single-administration, intravitreal (IVT) therapies.