If you’re considering gene therapy, you may wonder whether it’s right for you and what the treatment involves. You might also think about the risks, time away from home, and whether treatment could change your experience with sickle cell disease (SCD).

Gene therapy is an option for some people with SCD, but deciding whether to move forward with it involves more than knowing how it works. Here’s what treatment involves, what can make it difficult, and what to ask your healthcare team about the risks and benefits for you.

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What To Expect During the Treatment Process

Gene therapy is intensive. It happens in stages and takes months to collect and modify the stem cells from your body.

It requires multiple appointments, procedures, and extended recovery time. You’ll also need ongoing follow-up visits to monitor your progress.

Evaluation and Preparation

Your sickle cell care team starts by assessing whether gene therapy is right for you. They’ll review your medical history, organ function, and previous treatments like hydroxyurea (Droxia). They’ll also assess your ability to tolerate blood transfusions, stem cell collection, and the medicines to knock out your bone marrow.

You also need to commit to years of follow-up care.

This is also when you can talk through your own goals and what you want out of this treatment. Your healthcare team can walk you through what gene therapy can and can’t do, so you can make an informed decision.

Stem Cell Collection and Manufacturing

Your sickle cell care team collects stem cells from your blood. This process, called apheresis, uses a machine to separate the stem cells from the rest of your blood and return the remaining blood to you.

Before collection, you receive blood transfusions to reduce the amount of sickle hemoglobin (HbS). This transfusion period lasts at least 60 days for Lyfgenia or 8 weeks for Casgevy before you receive medicine to help move more stem cells into your bloodstream.

The collection process usually takes six to eight hours a day for one to three days. You may need additional collection cycles if enough cells aren’t collected initially.

After collection, your stem cells are sent to a laboratory, where technicians edit them or add genetic material, depending on your treatment. Manufacturing can take two to six months.

For Lyfgenia, for example, the process typically takes 10 to 15 weeks. For Casgevy, it can take five to six months.

While your cells are being prepared, you may continue receiving blood transfusions to manage your condition. Your care team will tell you which medicines need to be stopped and when. Your overall treatment timeline will depend on your treatment and how your body responds at each stage.

Conditioning, Infusion, and Hospital Recovery

Before your modified cells are infused, your healthcare team will give you chemotherapy called busulfan. This step is called conditioning. Busulfan destroys existing cells in your bone marrow, creating space for your modified cells to settle in and begin producing new blood cells.

Conditioning with busulfan typically causes side effects like:

• Nausea
• Hair loss
• Fatigue
• Very low blood cell counts requiring transfusions of platelets and red blood cells
• Mouth sores
• Fever and sometimes serious infections

It often causes infertility, which is worth discussing with your sickle cell care team well before this stage.

Gene therapy itself doesn’t change the genes in your eggs or sperm, so you can still pass an SCD-related gene variant to your children. The main known fertility risk comes from the conditioning chemotherapy.

Once the four days of conditioning are complete and the chemotherapy has cleared from your body, your healthcare team infuses your modified cells back into your bloodstream. From there, engraftment begins — your modified cells move into your bone marrow and start producing new blood cells over the following weeks. Most people stay in the hospital from the start of chemotherapy until their blood counts recover.

After infusion, your sickle cell care team will monitor your recovery and blood counts for two to three months. This may take place at the hospital or through regular appointments. You may need antibiotics to prevent infections or blood transfusions during this period.

Long-Term Follow-Up

Long-term follow-up continues well beyond your hospital stay because some risks may not appear right away. Your healthcare team will monitor:

• Blood counts
• Hemoglobin levels
• Organ function
• How your modified cells are working

For example, Lyfgenia carries a risk of blood cancer and requires lifelong monitoring, including blood counts at least every six months for at least 15 years. Casgevy also requires long-term monitoring for potential delayed effects from the gene-editing process and chemotherapy used for conditioning.

Missing follow-up appointments can make it harder to catch complications early. Planning ahead can help — ask how often you’ll need visits, who coordinates your care, and what to do if you move or switch healthcare providers.

What Are the Barriers to Gene Therapy?

Gene therapy may offer benefits, but accessing it isn’t simple for everyone. Costs, travel, medical requirements, and the demands of treatment can all create barriers.

Fertility Preservation

The chemotherapy drugs used during conditioning before gene therapy, such as busulfan, usually damage the ovaries or testes and cause infertility.

If you’re considering having biological children, ask your sickle cell care team early about fertility preservation, including when to decide, whether it could delay treatment, and whether you can get help covering the cost.

Treatment Center Access and Travel

Gene therapy is offered through specialized treatment centers, so you may need to travel for evaluation, treatment, and follow-up.

You may need to consider transportation, accommodations at school or work, child care, and time away from work or school. You may also need someone to help you during recovery.

Ask your treatment center what support is available, including help with insurance questions, costs, and logistics.

Cost and Insurance Coverage

Gene therapy can involve substantial costs for treatment, hospitalization, testing, travel, and follow-up. For some people, these expenses may put treatment out of reach.

Some insurance plans cover certain treatment-related costs. Manufacturer support programs may also help with some nonmedical costs, depending on eligibility.

Some health plans, including Medicare, cover gene therapy, though coverage and costs can vary by location, insurance, and eligibility.

Before moving forward, confirm your coverage with your insurer or treatment center, including what your plan covers and what you may need to pay out of pocket.

Emotional Readiness and Long-Term Commitment

Committing to gene therapy means thinking beyond the treatment itself. Before you start the process, think about whether you’re ready for everything that’s involved.

Recovery can take months, followed by years of monitoring. Consider how treatment may affect your schedule, support system, work, and other responsibilities.

Your healthcare team can explain what treatment can and can’t achieve, what recovery involves, and what ongoing care you’ll need.

Questions To Ask Your Care Team About Gene Therapy

Whether you’re just considering gene therapy or already weighing your options, these questions can help guide your conversation with your healthcare team.

Asking these questions can help you understand what treatment involves, what to expect afterward, and whether gene therapy fits your life and goals right now.