Your LYFGENIA™ FAQs answered

Sickle cell disease is caused by a [[gene]] mutation that produces abnormal hemoglobin. Repeated blockages and damage to the blood vessels over time can prevent your organs and tissues from getting the oxygen they need and can cause [[vaso-occlusive events (VOEs)]]

Yes. LYFGENIA is a [[gene addition therapy]]. It uses a viral vector to add genetic material directly to your stem cells, helping your body produce functional [[red blood cells]].

No. LYFGENIA is a [[gene]] therapy treatment that uses your body’s own stem cells.

LYFGENIA works at the genetic level to help your body make functional red blood cells, so that you can potentially decrease or stop experiencing [[vaso-occlusive events (VOEs)]].

No. LYFGENIA cannot cause HIV infection because parts of the HIV virus responsible for causing the infection have been removed from the vector that LYFGENIA uses.

During the decision-making process, you’ll likely have questions. We’ve created a list to keep handy at your next doctor visit.

Patients treated with LYFGENIA have developed blood cancers. Treatment with LYFGENIA may increase your risk of developing blood cancer. Blood cancer can develop many years after treatment with LYFGENIA. Blood cancer can be life-threatening and/or cause death. Because of the risk of blood cancer, you should talk to your doctor about the benefits and risks of LYFGENIA, and about your treatment options. Your doctor may evaluate if you have risk factors that increase your chances of developing blood cancer after LYFGENIA.

Because of the risk of cancer, it is important for you to be monitored at least every 6 months for a minimum of 15 years after LYFGENIA. Monitoring will include blood tests that measure your blood cell counts and evaluation of the blood cells where the gene product is present with specialized tests. If these tests are abnormal, additional testing may be recommended by your doctor, which might include more frequent blood tests and a bone marrow evaluation, which can tell your doctor if a blood cancer is developing.

Blood cancer may cause no symptoms, or symptoms can be general. You or your caregiver should call your healthcare provider right away for any of these signs or symptoms:

• Abnormal bruising or bleeding (including nosebleed)
• Blood in urine, stool, or vomit
• Coughing up blood
• Severe headache
• Unusual stomach or back pain
• Fever (100.4°F/38°C or higher)
• Swollen glands
• Abnormal tiredness

If you are diagnosed with a cancer, have your treating physician contact bluebird bio at 1-833-999-6378.

You may experience side effects associated with other medicines administered as part of the LYFGENIA treatment regimen. Talk to your physician regarding those possible side effects. Your healthcare providers may give you other medicines to treat your side effects. It is important that you or your caregiver tell your healthcare providers that you have received LYFGENIA.

Like many treatments, LYFGENIA has potential side effects. The most common ones are low blood pressure and hot flush on the day of treatment and blood cancer and a longer time for platelets to recover following treatment. Because of the risks, you should talk to your doctor about the benefits and risks of LYFGENIA, and about your treatment options.

Unfortunately, there are no available data about LYFGENIA and pregnancy, but there are risks for pregnant individuals and there are risks associated with conditioning on pregnancy and fertility. Make sure to consult your doctor if you’re pregnant or considering getting pregnant.

The LYFGENIA clinical trial (Study 1) protocol evolved over time as a result of improvements to the cell collection and LYFGENIA creation process. There were three parts to Study 1 in the LYFGENIA clinical trials—Study 1-A, Study 1-B, and Study 1-C. Protocol changes happened between Study 1-A and 1-B and between Study 1-B and Study 1-C. The cell collection and LYFGENIA creation process that occurred in Study 1-C is the same process that takes place in the FDA-approved version of LYFGENIA. Individuals treated with an earlier version of LYFGENIA developed blood cancers.