About the Disease
Sickle Cell Disease (SCD) is an inherited blood disorder caused by a mutation in the gene that encodes hemoglobin. Normal red blood cells are round and flexible; in SCD they become rigid, crescent or “sickle”-shaped. These sickled cells can block blood vessels, causing pain, anemia, organ damage, and reduced lifespan. Gene-edited stem cell therapy aims to use a patient’s own blood stem cells, correct the faulty gene (via CRISPR or other editing tools), then transplant them back so that the body can produce healthy red blood cells, reducing or eliminating the sickle symptoms.
Institutes / Trials Offering Gene-Edited Stem Cell Therapy for Sickle Cell Disease
UCSF Benioff Children’s Hospital Oakland / UCSF-UCLA-IGI Consortium – Oakland & Los Angeles, California, USA
- Running the CRISPR-SCD trial (also known as CRISPR_SCD001), which uses non-viral CRISPR-Cas9 gene-editing to correct the sickle mutation in the patient’s own blood stem cells, with re-infusion via bone marrow transplant. Home+3UCSF Pediatrics+3Home+3
- Principal investigator: Mark Walters, MD. UCSF Benioff Children’s Hospitals+1
- Contact / info: UCSF Benioff Children’s Hospital Oakland. UCSF Benioff Children’s Hospitals+1
King’s College Hospital / King’s Health Partners – London, UK
- Approved to deliver exa-cel (also known as Casgevy) treatment under the UK’s NHS. This therapy involves collecting a patient’s stem cells, gene-editing them to increase fetal hemoglobin production (which does not sickle), and then infusing them back. Kings Health Partners
- For older children and adults with severe sickle cell disease. Kings Health Partners
Lifespan Comprehensive Sickle Cell Center / University of Alabama at Birmingham (UAB) – Birmingham, Alabama, USA
- Has ongoing clinical trials of gene-edited stem cell therapies. One of the trials is BEACON, evaluating safety and efficacy of autologous (patient’s own) CD34+ stem cells edited (gene edited) for SCD with severe vaso-occlusive crises. The University of Alabama at Birmingham
Cure Sickle Cell Initiative (NHLBI, NIH) – USA
- A collaborative initiative that supports and funds gene therapies in sickle cell, including trials such as CRISPR-SCD. Its goal is to accelerate development of safe, gene-edited, stem cell-based cures. NHLBI, NIH+2Home+2
