People living with SCD pictured may or may not have received CASGEVY.
When deciding if CASGEVY™ may be right for you, it's important to get answers to the questions you have about CASGEVY and gene therapy.
The answers below can help you understand more about CASGEVY and if it might be an option for you.
CASGEVY is a one-time therapy used to treat people 12 years and older with sickle cell disease (SCD) who have frequent vaso-occlusive crises or VOCs.
CASGEVY is made specifically for each patient, using the patient’s own edited blood stem cells, and increases the production of a special type of hemoglobin called hemoglobin F (fetal hemoglobin or HbF). Having more HbF increases overall hemoglobin levels and has been shown to improve the production and function of red blood cells. To learn more about CASGEVY, download the overview brochure here.
The CASGEVY treatment journey could take up to 1 year. The only one who can decide if the treatment is worth it is you, in partnership with your support network and healthcare provider. To see CASGEVY study results, read here.
CASGEVY uses a gene-editing technology (CRISPR/Cas9) to change one of the genes in the DNA of your blood stem cells, so your body can start making its own working red blood cells. Learn more about how CASGEVY works by clicking here.
You can talk with your healthcare provider to find out if CASGEVY is the right treatment option for you. Learn more about how to start the conversation about CASGEVY with your healthcare provider here.
In the clinical study, 93.5% of people (29 out of 31) were free from severe VOCs for at least 12 months in a row after CASGEVY infusion. Learn more about the clinical study as well as safety and side effects here.
Gene therapy is a technique that adds, removes, or edits the genes inside cells. It may help manage or treat a genetic disease.
The gene editing done for CASGEVY uses a special tool called CRISPR/Cas9 (Clustered Regularly Interspaced Short Palindromic Repeats), which can be used to precisely find and edit a gene in the DNA. Once someone goes through gene editing, their genes are edited for good.
CRISPR/Cas9 combines guiding and editing tools, to find genes and do precise gene editing. With CASGEVY, it is used to edit the BCL11A gene, which is one of the genes that controls how much fetal hemoglobin your body makes.
CASGEVY does not use any viruses, including the HIV virus, to edit your cells.
The length of the entire process will vary from person to person and can take up to 1 year to complete. For more details about the journey, including steps, tips, and helpful resources, click here.
There are 6 steps in the CASGEVY treatment journey. The length and timing of certain steps may vary per person. The entire CASGEVY treatment journey could take up to a year. Location of these steps is based on the clinical study. Your healthcare team will help map out a treatment plan that works for you and your support network. As an overview, the steps include deciding on CASGEVY, getting ready for cell collection, collecting your blood stem cells, making your CASGEVY, receiving CASGEVY, and after treatment. Get more details about each step of the CASGEVY treatment journey here.
In order to prepare your body to receive CASGEVY, you will need to undergo chemotherapy. The chemotherapy will last for 4 days, but you may continue to experience symptoms after the therapy has been completed. Get more details about the CASGEVY journey here. Talk to your healthcare provider about the side effects of chemotherapy.
After receiving chemotherapy, it may not be possible for you to become pregnant or father a child. You should discuss options for fertility preservation with your healthcare provider before treatment.
Once you’ve completed CASGEVY treatment, you and your healthcare provider will determine how frequently you should meet based on your schedule, needs, and how you respond to your treatment. Learn more about each step of the journey, including post-treatment monitoring, here.
While CASGEVY does change, or edit, DNA in your body's blood stem cells, it does not change whether or not you'll pass sickle cell disease on to your children.
You will need to take time off from some personal commitments, as well as work or school, for some parts of the CASGEVY journey. However, you and your care team can work to determine the best times for you to start treatment. Learn about all the steps and the time they will take here.
CASGEVY is only available at Authorized Treatment Centers (ATCs). The healthcare teams at these centers treat people who are living with sickle cell disease with CASGEVY. After your healthcare provider prescribes CASGEVY, you can find an ATC near you.
You, your healthcare provider, and your care team will work together to determine how to prepare for each step, and what the timing will be. Get more details about the CASGEVY journey here.
