Exagamglogene Autotemcel · Casgevy, CTX-001

What is Exagamglogene Autotemcel?

Exagamglogene autotemcel is an autologous cell therapy made of CD34-positive hematopoietic stem cells gene-edited ex vivo with CRISPR-Cas9 to disrupt the erythroid enhancer of the BCL11A gene. The edited cells engraft in bone marrow and produce erythrocytes with higher fetal hemoglobin, which compensates for defective adult hemoglobin. It is used in sickle cell disease and beta-thalassemia.

Summary written with AI from the sources listed at the end of this page.

At a glance

Also known as

Casgevy, CTX-001, Exa-cel

Drug type

Cell therapy (Gene-edited cells)

Targets

BCL11A erythroid enhancer (BCL11A)

Mechanism

Gene editing: BCL11A erythroid enhancer (BCL11A)

Pathways

fetal hemoglobin (HbF) expression, reduced intracellular HbS concentration, erythropoiesis

Therapy area

Hematology

Developer

Vertex Pharmaceuticals Incorporated (VRTX)

Also in trials of

CRSP

FDA status

Approved as Casgevy (December 8, 2023), BLA125787 · FDA label

UNII

S53L777GM8

✅ FDA-approved uses

• Sickle cell disease with recurrent vaso-occlusive crises
• Transfusion-dependent beta-thalassemia

From the indications section of the FDA label (version of Jul 7, 2026). Read the full label for the exact wording, limitations and safety information.

🧪 Clinical trials

Trials of the companies BioRadar tracks, from ClinicalTrials.gov, most advanced phase first.

Conditions studied

Beta-ThalassemiaGenetic Diseases, InbornHematologic DiseasesThalassemiaHematological DiseasesHemoglobinopathiesSickle Cell Disease

More from Vertex Pharmaceuticals

Sources

• FDA Purple Book
• FDA label (DailyMed)
• FDA label Casgevy (2026-07-07), indications
• NCI Thesaurus
• ClinicalTrials.gov NCT03745287
• ClinicalTrials.gov NCT05356195

Updated Oct 11, 2026. Something wrong or missing? Report it on the VRTX page.