Treatment · Beta thalassemia

Zynteglo (betibeglogene autotemcel)

An approved gene-addition therapy for transfusion-dependent beta thalassemia — the non-editing alternative to Casgevy in the same disease.

Approved approvedgene addition

Approved treatment At least one medicine using this approach has been authorised by a national regulator for this use.

Eenvoudige uitleg

Zynteglo adds a working copy of the beta-globin gene into a patient's own blood stem cells using a modified virus, so their body can make normal haemoglobin again. Most people treated in its trials no longer needed blood transfusions. It treats the same condition as Casgevy by a different route.

Dieper ingaan

Betibeglogene autotemcel delivers a functional βA-T87Q-globin gene to autologous CD34+ cells by lentiviral transduction, restoring haemoglobin production in transfusion-dependent beta thalassemia. FDA approved August 2022. Requires the same myeloablative conditioning as editing-based products.

Sources

U.S. Food and Drug Administration
Zynteglo ↗

Written by The CRISPR Atlas editorial team Last updated Aug 25, 2026

Belangrijkste feiten

Company	bluebird bio
Disease	Transfusion-dependent beta thalassemia
Technology	Lentiviral gene addition — not gene editing
FDA status	Approved August 2022 Source: U.S. Food and Drug Administration
List price	$2.8 million in the United States Announced list price

Citeer deze pagina

The CRISPR Atlas. “Zynteglo (betibeglogene autotemcel).” Updated Aug 25, 2026. https://crisprdecoded.com/treatments/zynteglo

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Technologies

Gene Therapy (gene addition)

Diseases

Beta Thalassemia

Companies

bluebird bio

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