The CASGEVY^®
Treatment Journey

People living with TDT pictured may or may not have received CASGEVY.

Josh and Luke, living with TDT, and their mom, Alison

People living with TDT pictured may or may not have received CASGEVY.

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Before you begin your CASGEVY journey, it’s important to understand the treatment process and what to expect along the way.

There are 6 steps in the CASGEVY treatment journey. Watch Cora’s CASGEVY story from start to finish, and click through each step below to learn more.

The length and timing of certain steps may vary per person. The entire CASGEVY treatment journey could take up to a year. Location of the steps on this page is based on the clinical study. Your healthcare team will help map out a treatment plan that works for you and your support network.

Your red blood cell transfusions will continue throughout the treatment journey until you have received CASGEVY as determined by your healthcare provider.

Click through each step to learn more.

Deciding on CASGEVY Preparing for Cell Collection Collecting Your Blood Stem Cells Making Your CASGEVY Chemotherapy, Receiving CASGEVY, and Recovery After Treatment

Deciding on CASGEVY

Arrows in different directions representing the choices and options when considering CASGEVY® (exagamglogene autotemcel)

Timing will vary by person

Eligibility is determined with your healthcare team at your ATC

If you are interested in learning more about gene therapy, talk with your healthcare provider and ask if CASGEVY might be an option for you. You will then need to schedule an appointment at an Authorized Treatment Center (ATC). The healthcare providers at ATCs are the only ones who can confirm whether CASGEVY is right for you and can help you begin the treatment process.

Chemotherapy is needed before receiving CASGEVY. It’s an important part of the process but can also affect your ability to have children. Talk to your healthcare team to discuss the fertility preservation options that may be available to you. Fertility preservation is a procedure that saves sperm, eggs, or reproductive tissue for possible use at a later date.

Preparing for Cell Collection

You will continue receiving transfusions

CASGEVY is a treatment that is made using your own blood stem cells. These are cells that can develop into red blood cells.

Before your healthcare team collects your blood stem cells, they'll want to make sure your body is ready. This includes continuing to get red blood cell transfusions (RBCTs) until your hemoglobin reaches a specific level that will prepare you for cell collection. Your healthcare provider will make adjustments as needed to get your body to that specific hemoglobin level before the next step can take place.

You may need to stop taking certain medications for at least 8 weeks during this step. Your healthcare provider will inform you if any updates need to be made to your medications.

Collecting Your Blood Stem Cells

Up to 7 days* (based on the clinical study)

Location of this step is determined by the healthcare provider.

Blood stem cells are the cells used to make CASGEVY specifically for you. To collect your cells, you will need to travel to your ATC.

There are 2 parts to the collection process:

1) Moving your blood stem cells into the blood stream, also called mobilization. Your healthcare team will give you medicines that help your blood stem cells move from your bone marrow to your blood so they can be collected. In the clinical study, mobilization happened in 2 stages:

For the first 4 days, people received a medicine called granulocyte-colony stimulating factor (G-CSF)
For the next 3 days, people received a medicine called plerixafor (in addition to G-CSF, which continued for 1-2 additional days)

2) Blood stem cell collection, which may be called apheresis. A healthcare provider or nurse will use an intravenous, or IV, line to hook you up to a blood stem cell collection machine. This machine separates blood stem cells from your blood and then returns your blood to your body.

In the clinical trial, on average,^† people required 1 cycle of blood stem cell collection. If more than 1 collection is needed, each collection will be separated by a minimum of 14 days and you will be able to return home during that time. This gives your body time to make new blood stem cells.

During this step, extra cells called “back-up cells” are also collected and stored at the ATC. These are not sent out for editing and are stored frozen at the center in case there are any issues in the treatment process. If CASGEVY can’t be given to you for any reason or does not work in your body, your healthcare provider will put these rescue cells back into your body. If you are given rescue cells, you will not have any treatment benefit from CASGEVY.

*This may happen more than once.

^†This was measured as a median amount, which is the middle value of a group of numbers; half of the numbers are less than the median and half are higher.

Making Your CASGEVY

5-6 months

You'll be at home while CASGEVY is being made at a lab

After your blood stem cells are collected, they will be carefully packaged and shipped to a lab. This is where CASGEVY will be made specifically for you using your own blood stem cells. You will return home until your treatment is ready.

It may take up to 6 months from the time your cells are collected to manufacture and test CASGEVY before it is sent back to your healthcare provider. People who want to preserve their fertility should discuss options with their healthcare provider before treatment. You’ll receive red blood cell transfusions during this time to maintain your hemoglobin above a certain level, and your healthcare provider may have you stop taking certain medications before you receive chemotherapy.

While waiting for your treatment, you should start preparing for your upcoming stay at your ATC. Talk with your loved ones and discuss how they can help cover responsibilities you may have at home.

Chemotherapy, Receiving CASGEVY, and Recovery

Icon representing person sitting in an infusion chair receiving CASGEVY® (exagamglogene autotemcel) at the ATC

Approximately 7 weeks (based on the clinical study)

At your ATC

Once your CASGEVY is ready, you will travel to your infusion Authorized Treatment Center (ATC) to undergo chemotherapy and receive your CASGEVY. The first step will be to receive a type of chemotherapy. In the clinical study, this took 4 days. The chemotherapy will cause your blood cells to fall to a very low level, so your healthcare providers will continue to monitor how you're feeling and allow you time to rest. Talk to your healthcare provider about other side effects of chemotherapy.

Next, you’ll receive your CASGEVY as a one-time infusion through an IV into the vein.

After the CASGEVY infusion, you will stay at the ATC so your healthcare provider can closely monitor your recovery. Your healthcare provider will decide when you can go home.

After receiving chemotherapy and CASGEVY, your immune system will need time to recover. Consider bringing personal items like photos, special blankets, or other keepsakes from home that are meaningful to you, as you may have limited interaction with loved ones.

After Treatment

Once your healthcare team determines you have recovered, they will let you know when you can go home. You will work with your healthcare team to come up with a long-term follow-up plan. This will include appointments with your healthcare provider and at the ATC to continue to monitor your health.