AAV Vectors for Improved CNS Targeting

Targets Neuronal Cells to Develop Gene Therapies and Treat Neurological Diseases

These engineered AAV vectors are designed to target neuronal cell types to aid in the development of gene therapies, which can treat a variety of neurological diseases. In 2020, the market for CNS targeting technologies was worth $6 billion and is expected to reach $46.5 billion by 2030. Neurological diseases can result in life-threatening complications or lead to serious symptoms and/or death. Effective treatments remain limited. Gene therapies have the potential to treat individuals suffering or at risk for suffering from various neurological diseases and conditions.

Researchers at the University of Florida developed improved adeno-associated virus (AAV) vectors to target the central nervous system (CNS) for treatment of neuronal diseases and disorders.

Application

AAV capsid modification that targets the central nervous system and other neuronal cell types to treat diseases that affect neurological tissue and/or function

Advantages

• Enables AAV capsids to target to neuronal cell types and be less prone to neutralization by antibodies
• The AAV capsids may be used in a variety of applications, including but not limited to compositions and therapeutic methods for treatment of neurological diseases affecting the central nervous system

Technology

University of Florida researchers have generated AAV capsids of the serotypes AAV1, AAV9 and AAVrh.10 containing a specific CNS-targeting protein insertion and have shown them to be effective at targeting different neuronal cell types, increasing transduction efficiency as much as 25 fold in certain circumstances. The insertion of the CNS targeting peptide also eliminates an immunogenic epitope, making the vectors less likely to be neutralized by the immune system.