AAV Gene Therapies

We are accelerating the delivery of AAV gene therapies for central nervous system (CNS) disorders using next-generation science

The promise of gene therapy lies in its elegance: the ability to treat genetic diseases simply by replacing a defective gene with a working copy. It takes world-class thinkers, state-of-the-art science, and time. We have the thinkers and the science—time is the one component that our patients don’t have. So our mission is to responsibly accelerate delivery of these medicines.

Our next-generation science uses the leading platform for in vivo treatment of neurodegenerative diseases—vectors known as adeno-associated viruses (AAVs)—to deliver a functional copy of the gene to cells in both the central nervous system and the periphery. The gene delivered encodes for proteins that are secreted. The uptake of secreted protein in neighboring cells is referred to as cross-correction. This provides a potential expansion of benefits to cells beyond those directly transduced by gene therapy. There are potential risks associated with all gene therapies, including AAV-based therapies.

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About AAV Vectors and
AAV-based Therapies

Adeno-associated viruses (AAVs) are the leading platform for gene delivery used in the treatment of a variety of neurodegenerative diseases.

  • Wild-type AAV is not known to cause any human disease
  • Therapeutic recombinant AAVs (rAAVs) are made by replacing all AAV protein coding sequences with a gene expression cassette
  • The cassette contains the functional therapeutic gene, as well as the regulatory elements necessary for that gene to be expressed once it’s delivered to the target cell nucleus

Schematic of AAV gene replacement

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See what’s on the horizon

Learn about the therapies we’re working on.

Review Our Pipeline

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Information for patients and caregivers

Access helpful content for those directly affected by monogenic CNS disorders.

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