Your LYFGENIA™ FAQs answered

LYFGENIA is a genegeneA sequence of DNA responsible for controlling inherited traits therapy indicated for individuals with sickle cell disease who are 12 years or older and have a history of vaso-occlusive events (VOEs)vaso-occlusive events (VOEs)In a LYFGENIA clinical study, VOEs were defined as any of the following events requiring evaluation at a medical facility: an episode of acute pain with no medically determined cause other than vaso-occlusion, lasting more than 2 hours, acute chest syndrome, acute hepatic sequestration, or acute splenic sequestration.

Yes, LYFGENIA was approved by the FDA in December 2023.

LYFGENIA is for individuals with sickle cell disease who are 12 years or older.

Before treatment, people living with sickle cell disease and their healthcare providers will work together to help consider whether LYFGENIA is right for them and if they’re eligible for LYFGENIA.

Almost all individuals treated with LYFGENIA no longer experience vaso-occlusive events (VOEs)vaso-occlusive events (VOEs)In a LYFGENIA clinical study, VOEs were defined as any of the following events requiring evaluation at a medical facility: an episode of acute pain with no medically determined cause other than vaso-occlusion, lasting more than 2 hours, acute chest syndrome, acute hepatic sequestration, or acute splenic sequestration. 88% of individuals (28/32) did not experience any vaso-occlusive events (VOEs). 94% of individuals (30/32) did not experience any severe vaso-occlusive events (sVOEs)severe vaso-occlusive events (sVOEs)In a LYFGENIA clinical study, sVOEs were defined as vaso-occlusive events (VOEs) requiring a hospitalization or multiple visits to an emergency department/urgent care over 72 hours and receiving IV medications at each visit, or priapism requiring any level of medical attention.

If you’re considering LYFGENIA, you should start by talking to a doctor. Looking for more resources about LYFGENIA? You can find them with my bluebird support by contacting a Patient NavigatorPatient NavigatorA patient support specialist at my bluebird support who is knowledgeable about LYFGENIA and is equipped with resources to help navigate questions, including concerns about healthcare insurance or treatment planning.

LYFGENIA is a one-time infusioninfusionThe process by which LYFGENIA is administered into your veins that can only be administered at a Qualified Treatment Center (QTC)Qualified Treatment Center (QTC)A hospital that has been carefully selected based on expertise in areas such as transplants or cell and gene therapy, with staff trained to administer LYFGENIA after a hematologist provides a referral.

Your journey may look different depending on your circumstances, unique to you and your care team at the Qualified Treatment Center (QTC)Qualified Treatment Center (QTC)A hospital that has been carefully selected based on expertise in areas such as transplants or cell and gene therapy, with staff trained to administer LYFGENIA.

LYFGENIA is only available at a Qualified Treatment Center (QTC)Qualified Treatment Center (QTC)A hospital that has been carefully selected based on expertise in areas such as transplants or cell and gene therapy, with staff trained to administer LYFGENIA. A QTC is a specialized hospital qualified to administer a bluebird bio genegeneA sequence of DNA responsible for controlling inherited traits therapy. Each LYFGENIA QTC has been carefully selected based on their expertise in areas such as transplant, cell, and gene therapy.

If you’re interested in a Qualified Treatment Center (QTC)Qualified Treatment Center (QTC)A hospital that has been carefully selected based on expertise in areas such as transplants or cell and gene therapy, with staff trained to administer LYFGENIA, take a look at our QTC locator tool. This is where you can find the latest list. We’re also activating more centers, so please continue to check for updates.

Chemotherapy is given to condition or make room for stem cellsstem cellsAn immature cell that has the potential to develop into any blood cell type, including white blood cells, red blood cells, and platelets in your bone marrow for genegeneA sequence of DNA responsible for controlling inherited traits therapy or stem cell transplant. Some form of chemotherapy is required for all transplant options—whether it is gene therapy or donor stem cell transplant.

It’s possible. Because chemotherapy is used during the conditioningconditioningThe process of making room in your bone marrow for gene therapy or a stem cell transplant through chemotherapy. Conditioning is required for all transplant options—whether it’s gene therapy or a donor stem cell transplant process, genegeneA sequence of DNA responsible for controlling inherited traits therapy comes with a risk of infertility. It is important to learn about these risks and discuss them with your doctor, and, if needed, discuss any options for fertility preservation, such as freezing eggs or sperm.

Your Patient NavigatorPatient NavigatorA patient support specialist at my bluebird support who is knowledgeable about LYFGENIA and is equipped with resources to help navigate questions, including concerns about healthcare insurance or treatment planning at my bluebird support can help you explore your coverage options and what out-of-pocket costs you may be responsible for, including copays.

Patient Navigators can also help identify potential sources of support for eligible and uninsured individuals.

Connect with a Patient Navigator:

Send an email: mybluebirdsupport@bluebirdbio.com

Call: 1-833-888-NEST (6378)

my bluebird support can help support you when exploring your benefits. This includes coverage options. my bluebird support can also work closely with your health insurance provider and your doctor’s office staff by offering guidance and answers to coverage questions.