Gene therapy is a potentially curative therapy. This means that it could act as a cure, but it is too new to say for sure. It has been shown to result in a significant decline in pain episodes, but we need to learn more about long-term impacts and side effects. It is also not clear whether this is a “one-and-done” treatment. The FDA currently recommends 15 years of patient follow up after treatment.

Gene therapy became available in 2024.

Gene therapy is approved for people ages 12 and up. Sickle cell disease SS and S-beta-zero-thalassemia are eligible. The FDA indicates that sickle cell disease SC is not included.

Gene therapy requires you to have chemotherapy. This means it could result in:

• Infertility or secondary cancer
• Temporary weakening of the immune system so that you cannot fight off any infections
• Temporary hair loss

Only a few centers across the USA offer gene therapy for sickle cell disease. Sites must have both medical expertise and willingness to accept a complex cost structure.

Gene therapy is expensive, and FDA-approved high-cost medications can come with barriers. Exagamglogene autotemcel is estimated to cost $2.2 million, and lovotibeglogene autotemcel is estimated to cost $3.1 million. Initiatives like the Centers for Medicare and Medicaid Services’ Cell and Gene Therapy Access Model may help reduce costs for individuals on Medicaid.

As far as we know, yes. It is designed to be able to help raise fetal hemoglobin (HbF), which should work for all different kinds of sickle cell disease. However, the amount of experience with the different kinds has not been nearly the same – we know the most for SS and S Beta zero thalassemia types.

Exagamglogene autotemcel is the first approved use of gene editing. However, gene addition therapy has been used to treat other conditions, including:

• Retinal degeneration
• Spinal muscular atrophy
• Beta-thalassemia
• X-linked Adrenoleukodystrophy
• Hemophilia A & B
• Bladder cancer
• Acute-lymphoblastic leukemia

To learn more about the gene therapies used to treat these conditions, click here.
For a longer (but not complete) list of conditions that have been treated using gene therapy, click here.

For many people, the benefits of this new treatment outweigh the risks. Your doctors will help you determine whether this is a good option for you.

• How long will this take?
• What is the time commitment?
• Where is the nearest treatment center?
• What are my other options?

There are several resources available. The below sources are considered trustworthy and non-biased by SCDAA.

• The Democratizing Education for Sickle Cell Gene Therapy Project created materials with input from individuals with SCD and their families. Read their FAQs and visit their website.
• SCDAA has produced several videos on gene therapy. Watch the gene therapy masterclass and this webinar to learn more.
• OneSCDVoice hosts gene therapy resources on their Gene Therapy 101 page.
• The Dec. 8 FDA approval statement for Casgevy and Lyfgenia.

To learn more about Vertex’s Casgevy (exagamglogene autotemcel), visit casgevy.com. To learn more about bluebird bio’s Lyfgenia (lovotibeglogene autotemcel), visit my bluebird support.