What are AAV Vectors?

What is an adeno-associated virus (AAV)?

Approaches for inherited conditions stemming from a single genetic mutation, including hemophilia B, have predominantly focused on the delivery of a functional, or working, gene using a viral vector.

Gene therapy for hemophilia B uses a certain type of vector called an adeno-associated virus, or AAV. The AAV, naturally existing in the world at large, is deactivated, so it won’t cause any illness while it performs its new task to deliver a therapy. And AAV vectors don’t change your DNA, they just help deliver the working gene to a targeted part of the body.

Number 1

In AAV gene therapy or gene transfer, a working gene is inserted into an AAV vector.

Working gene being inserted into AAV vector
Number 2

AAV vectors protect and deliver the working gene to its destination through a one-time infusion.

Gene inside of a AAV vector
Number 1 Working gene being inserted into AAV vector

In AAV gene therapy or gene transfer, a working gene is inserted into an AAV vector.

Number 2 Gene inside of a AAV vector

AAV vectors protect and deliver the working gene to its destination through a one-time infusion.

AAV gene therapy is being investigated for a number of reasons, to understand:

How long does the
working gene work?

Is the working gene
protected until it arrives
at its destination?

Does the working
gene cause any
adverse events?

Designing AAV vectors to fit particular needs

The success of gene therapy is influenced by the design of the vector and some other factors.

The design of an AAV vector affects the delivery of the working gene into the cells and how well the AAV gene therapy will work.

An ideal AAV vector for gene therapy

Allows one-time administration with long‑lasting effects

One-time IV administration

Delivers the working gene to the correct part of the body

Working gene is delivered to the correct part of the body

Enables treatment of the largest possible population

Hemophilia B population image

Targeted for little impact on other cells

Target

Allows one-time
administration with
long‑lasting effects

One-time IV administration

Delivers the working
gene to the correct
part of the body

Working gene is delivered to the correct part of the body

Enables treatment of the
largest possible population

Hemophilia B population image

Targeted for little impact on
other cells

Target

AAV Vectors have different natural targets

AAV vectors have several characteristics that make them well-suited to the delivery of gene therapies compared with other vectors. Different subtypes of AAV are called serotypes. Different serotypes target different parts of the body.

Select an AAV vector to see which parts of the body it targets

AAV1 body image Brain Icon Icon of Lungs Heart Icon Liver Icon Testes Icon Skeletal Muscle Icon
AAV1
AAV2
AAV3
AAV4
AAV5
AAV6
AAV7
AAV8
AAV9

KEY

Brain Icon

Brain

Icon of Lungs

Lungs

Heart Icon

Heart

Liver Icon

Liver

Testes Icon

Testes

Skeletal Muscle Icon

Skeletal Muscle

which of the following are true?

Please choose an option.

They're all true!

AAV-based gene therapy has been studied for decades, involves targeting specific parts of your body, and does not change your DNA.

Learn why gene therapy is a fit with hemophilia B blue-arrow

Why is AAV gene therapy a
promising option for hemophilia B?

Learn more blue-arrow