Malachi, a child with SMA, laughing as his father, Torrence, lifts him up and smiles at him.

ZOLGENSMA is an essential, one-time treatment option

Malachi has 2 copies of SMN2 gene
Treated at ~4 months old

The only gene replacement therapy and only one-dose SMA treatment

ZOLGENSMA^® (onasemnogene abeparvovec-xioi) is the only treatment that addresses the genetic cause of spinal muscular atrophy (SMA) with just one dose, and is a type of medicine called gene replacement therapy. ZOLGENSMA works by increasing the amount of SMN protein in the body to keep the muscles working as they should. Patients will receive a corticosteroid before and after infusion with ZOLGENSMA and will undergo blood tests and monitoring.

More about SMA

SMA is a progressive and rare genetic disease that affects the motor neuron cells in the spinal cord and impacts the muscles used for breathing, eating, crawling, and walking.

ZOLGENSMA is a gene replacement therapy for SMA that replaces the missing or nonworking SMN1 gene with a new, working SMN gene. The new SMN gene helps the body’s cells—particularly the motor neuron cells—to keep producing SMN protein.

Cause of SMA

Missing or nonworking SMN1 gene

SMA is caused by a missing or nonworking survival motor neuron 1 (SMN1) gene. This is the primary gene responsible for producing the survival motor neuron (SMN) protein, which is critical to the health of motor neurons—the nerves that control our muscles. When the SMN1 gene is missing or is not working properly, the body cannot make enough SMN protein. Without enough SMN protein, some motor neuron cells throughout the body lose their ability to function and, as a result, die. When this happens, people with SMA may experience muscle weakness throughout the body, which can affect essential activities, such as walking, eating, and in some cases, breathing.

Graphic showing the results of the unaffected and affected children with SMA.

The difference between the SMN1 gene and SMN2 backup gene

How ZOLGENSMA works

In SMA, the SMN1 gene is missing or not working properly. The SMN1 gene makes about 90% of the SMN protein your child needs.

ZOLGENSMA is the only gene replacement therapy for SMA that replaces the missing or nonworking SMN1 gene by delivering a new, working SMN gene to the body’s cells with a single infusion. ZOLGENSMA is designed to keep continuously working in the body.

With ZOLGENSMA, a new, working SMN gene is placed inside a delivery vehicle called a vector.

The vector that delivers the SMN gene is made from a virus called AAV9. This type of virus is not known to make people sick. To make the vector, the DNA of the virus is removed so that the new SMN gene can be put inside.

Graphic of the new, working SMN gene. AAV9 houses new, working SMN gene. This is called a vector.

ZOLGENSMA travels throughout the body and delivers the new, working gene to motor neuron cells.

Graphic of the vector with new, working SMN gene inside the motor neuron traveling through an infant's body

By replacing the SMN1 gene, ZOLGENSMA enables the production of SMN protein and helps preserve essential muscle function.

Motor neuron cells that would have died without treatment can survive and be maintained, stopping the progression of SMA.

Graphic of motor neuron with new, working SMN gene preserving essential muscle function

Making a decision for a child with SMA?

Remember, only ZOLGENSMA is designed to work with a one-time dose.

See how ZOLGENSMA works

ZOLGENSMA is the only gene replacement therapy that replaces the SMN1 gene to stop the progression of SMA.

See the transcript

Scientific innovation has led to some pretty incredible breakthroughs in modern medicine. And today, we’re going to learn about one of those amazing breakthroughs—a gene therapy called ZOLGENSMA^® (onasemnogene abeparvovec-xioi) that stops the progression of spinal muscular atrophy, or SMA, with a one-time infusion. Don’t worry, you don’t have to be a scientist to understand it. Let me show you how it works. Children with SMA are born with a missing or nonworking SMN1 gene. ZOLGENSMA is an infusion that delivers SMN genes where they’re needed, in a child’s body, to help keep the muscles working as they should. Now that you know the basics, let’s dive in a little deeper to see how ZOLGENSMA works in the body and how it helps children with SMA. A dedicated team of scientists at Novartis Gene Therapies approached SMA treatment in a bold way—by targeting the genetic root cause of SMA with a one-time infusion. Why is targeting the genetic root cause so important? Well, the SMN1 gene provides instructions for motor neuron cells to make SMN protein. Without enough SMN protein, the motor neuron cells stop working—and that's a big problem! These motor neuron cells are responsible for all types of muscle movement that we rely on daily for eating, breathing, sitting up, walking, or, when you hear a great song, singing. If there isn’t enough SMN protein being made, motor neurons stop working. Then, these basic functions we rely on become harder to do and can be lost permanently. As you can imagine, the sooner treatment is received, the sooner the progression of SMA can be stopped. This is where the amazing science behind ZOLGENSMA comes in. ZOLGENSMA is designed to stop the progression of SMA. The main components of ZOLGENSMA are a gene and a vector. The gene is a new, fully functioning SMN gene that's just waiting to get to work. The vector is made from a type of virus called AAV9, which is not known to make people sick. The viral DNA is removed and replaced with the new SMN gene, which is then delivered to the right places. Think of the vector as a delivery truck—with its cargo, in this case the new SMN gene, it’s ready to make its delivery to the motor neuron cells. This process happens throughout the body with many new SMN genes getting delivered to their destination. That’s pretty cool, right? Even cooler is the fact that more than two-thousand children have received ZOLGENSMA. Thanks for watching. And if you have any questions about ZOLGENSMA, make sure you talk to your doctor.

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