In children with spinal muscular atrophy (SMA), the SMN1 gene is missing or not working properly. ZOLGENSMA® (onasemnogene abeparvovec-xioi) replaces the function of the missing or nonworking SMN1 gene with a new, working SMN gene. ZOLGENSMA is designed to help the body continuously produce SMN protein with a single infusion.
To help you understand how this is possible, let’s look at how ZOLGENSMA works.
In children with spinal muscular atrophy (SMA), the SMN1 gene is missing or not working properly. ZOLGENSMA® (onasemnogene abeparvovec-xioi) replaces the function of the missing or nonworking SMN1 gene with a new, working SMN gene. ZOLGENSMA is designed to help the body continuously produce SMN protein with a single infusion.
To help you understand how this is possible, let’s look at how ZOLGENSMA works.
Scientific innovation has led to some pretty incredible breakthroughs in modern medicine. And today, we’re going 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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The SMN1 gene is the body’s main source of SMN protein, and without it, motor neuron cells may not get the SMN protein they need to survive.
In SMA, the body uses some SMN protein produced by the SMN2 backup gene. However, 90% of the SMN protein that the SMN2 backup gene makes is not fully functional. Usually, the more copies of the SMN2 gene a person has, the less severe his or her SMA is.
Motor neuron cells control muscle function. When motor neuron cells don’t get enough SMN protein, they stop working. Once they are lost, they cannot be brought back. This means everyday activities like eating, breathing, or sitting up become harder to do, and can be lost permanently.
ZOLGENSMA is made up of a new, working SMN gene that is placed inside a delivery vehicle called a vector. The vector helps deliver the working SMN gene to motor neuron cells throughout the body.
The vector that delivers the SMN gene is made from a virus called adeno-associated virus 9, or 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. Vectors are used because they can travel throughout the body and deliver the new, working gene to the cells throughout the child's body. ZOLGENSMA does not change or become a part of the child’s DNA.
By replacing the function of the SMN1 gene, ZOLGENSMA restores SMN protein production 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. Remember, the sooner treatment is received, the sooner the progression of SMA can be stopped.
This simple summary is designed to help you talk about ZOLGENSMA with others. Share it with your loved ones or take it on the go to your child's next doctor’s appointment. How ZOLGENSMA Works helps make the science of gene therapy easy to understand.
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What is the most important information I should know about
ZOLGENSMA?
What is the most important information I should know about ZOLGENSMA?
What should I watch for before and after infusion with ZOLGENSMA?
What do I need to know about vaccinations and ZOLGENSMA?
Do I need to take precautions with the patient’s bodily waste?
What are the possible or likely side effects of ZOLGENSMA?
What is ZOLGENSMA?
ZOLGENSMA is a prescription gene therapy used to treat children less than 2 years old with spinal muscular atrophy (SMA). ZOLGENSMA is given as a one-time infusion into a vein. ZOLGENSMA was not evaluated in patients with advanced SMA.
The safety information provided here is not comprehensive. Talk to the patient’s doctor about any side effects that bother the patient or that don’t go away.
You are encouraged to report suspected side effects by contacting the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch, or Novartis Gene Therapies, Inc. at 833-828-3947.
Please see the Full Prescribing Information.
