Adverum Biotechnologies

Adeno-Associated Virus Delivery, Gene Therapy

Also additional Clin1 wet AMD; undisclosed (partner: GenSight) Clin2 retinitis pigmentosa; undisclosed (partner: Lexeo) Clin1 Friedreich's ataxia; ADVM-062 Clin0 blue cone monochromatism; 4 undisclosed programs RD/Clin0 geographic atrophy, retinitis pigmentosa

Adverum Biotechnologies is creating one-time gene therapies for patients with ocular and rare diseases. These gene therapies provide durable expression of therapeutic proteins that are lacking in diseased patients. To identify effective vectors to deliver the gene therapies, Adverum is using multi-step directed evolution that screens millions of different Adeno-associated viruses’ (AAVs) capsid genes for novel properties such as cell type specificity. Following novel vector discovery, selected optimized AAVs can be produced using Adverum’s highly scalable manufacturing process. Adverum is advancing a pipeline of gene therapies for ocular diseases such as ADVM-022 for age-related macular degeneration which has entered clinical trials.