Kanexthal: Treatment for Beta-Thalassemia

A one-time gene therapy for transfusion-dependent β-thalassemia.

Kanexthal is designed as a curative treatment for eligible patients with transfusion-dependent β-thalassemia. Kanexthal addresses the root cause of β-thalassemia by restoring the natural hemoglobin production and freeing patients from lifelong tranfusions and iron chelations.

View clinical results

See treatment journey

Program experience & outcomes

Evidence snapshot (Based on available data by October 2025)

70+ patients treated

17 patients treated in IIT, 9 patients in Ph. 1

40+ patients treated in real world

Free from transfusion
All patients treated so far have achieved transfusion independence.
Longest follow up is ~4.5 years.

Accessible in China
The product is commercially available for patients in certified hospital(s) in China based on pilot access program.

Blood stem cells are collected from the patient through apheresis*.

A functional β-globin gene is added to the collected cells using a lentivirus vector**.

Each batch is carefully tested for safety and quality before infusion.

The modified stem cells are infused back into the patient to restore healthy blood production.

1.

2.

3.

4. How Kanexthal works

*Apheresis is a procedure that enables the separation and collection of stem cells from the whole blood using a centrifuge machine.

**A lentiviral vector is a molecular tool used to deliver genetic material (a functional β-globin gene in this case) into

cells. It is essentially a "delivery truck" derived from a lentivirus that has been engineered to be safe and useful for research and medicine.

What is Kanexthal?

Kanexthal is designed to address the root cause of β-thalassemia, not just manage its symptoms.

Using the patient’s own blood stem cells, Kanexthal introduces a functional β-globin gene that enables the body to produce healthy hemoglobin independently. This reduces — and in many cases, eliminates — the need for ongoing blood transfusions.

Unlike lifelong transfusion regimens that can lead to iron overload and other complications, Kanexthal offers a one-time, potentially curative treatment.

A one-time gene therapy for transfusion-dependent β-thalassemia.​

​

Kanexthal is designed as a curative treatment for eligible patients with transfusion-dependent β-thalassemia. Kanexthal addresses the root cause of β-thalassemia by restoring the natural hemoglobin production and freeing patients from lifelong tranfusions and iron chelations.

​

​

Program experience & outcomes​

70+ patients treated 17 patients treated in IIT, 9 patients in Ph. 1 40+ patients treated in real world​​

Free from transfusion All patients treated so far have achieved transfusion independence. Longest follow up is ~4.5 years.​​​

Accessible in China The product is commercially available for patients in certified hospital(s) in China based on pilot access program.

1.

2.

3.

4.

How Kanexthal works