By The Definition: Sickle Cell Disease

Sickle Cell Disease (SCD) is a group of inherited red blood cell disorders. Healthy red blood cells are round and they move through small blood vessels carrying oxygen to all parts of the body.  In SCD, the red blood cells become hard and sticky and look like a C-shaped farm tool called a “sickle.” Sickle Cells die early, which causes a constant shortage of red blood cells. When they travel through small blood vessels, they get stuck and clog the blood flow. This can cause pain and other serious problems such infection, acute chest syndrome and stroke.

Symptoms and complications of SCD are different for each person and can range from mild to severe.

There is no universal cure for Sickle Cell Disease.

The only treatments that can cure Sickle Cell Disease are the newest gene‑editing therapies and stem cell (bone marrow) transplants. Everything else helps manage symptoms, but does not cure the disease.

GENE THERAPIES

The FDA approved two transformative gene therapies in December 2023 — the first treatments that can potentially cure Sickle Cell Disease by correcting the underlying genetic problem.

Casgevy (CRISPR gene‑editing therapy)

  • Uses CRISPR to edit a patient’s own stem cells.

  • Helps the body produce healthy red blood cells.

  • Considered a functional cure for eligible patients.

  • Expanded in 2026 to treat children as young as age 2.

Lyfgenia (gene‑addition therapy)

  • Adds a corrected gene to help red blood cells function normally.

  • Also considered a functional cure, though it carries a boxed warning.

Blood & Bone Marrow Transplant (Stem Cell Transplant)

A stem cell transplant from a matched donor can cure Sickle Cell Disease by replacing the patient’s bone marrow with healthy marrow that produces normal red blood cells.

  • Highly effective but requires a compatible donor.

  • Carries risks such as graft‑versus‑host disease.

  • Usually offered to patients with severe disease.

Bone Marrow or Stem Cell Transplants are very risky, and can have serious side effects, including death. For the transplant to work, the bone marrow must be a close match.

People with SCD can live full lives and enjoy most of the activities that other people do. Treatment options are different for each person depending on the symptoms and can include receiving blood transfusions, maintaining a high fluid intake, receiving intravenous therapy and medications to help with pain.