Disease: Acute Myeloid Leukaemia, AML

Disease info:

Acute myelogenous leukaemia (AML) is a cancer of the blood and bone marrow — the spongy tissue inside bones where blood cells are made.

The word "acute" in acute myelogenous leukaemia denotes the disease's rapid progression. It is called myelogenous (my-uh-LOHJ-uh-nus) leukaemia because it affects a group of white blood cells called the myeloid cells, which normally develop into the various types of mature blood cells, such as red blood cells, white blood cells and platelets.

Acute myelogenous leukaemia is also known as acute myeloid leukaemia, acute myeloblastic leukaemia, acute granulocytic leukaemia and acute nonlymphocytic leukaemia.

Acute myelogenous leukaemia is caused by damage to the DNA of developing cells in the bone marrow. The bone marrow produces immature cells that develop into leukaemic white blood cells called myeloblasts. These abnormal cells are unable to function properly, and they can build up and crowd out healthy cells.

In most cases, it's not clear what causes the DNA mutations that lead to leukaemia. Radiation, exposure to certain chemicals and some chemotherapy drugs are known risk factors for acute myelogenous leukaemia.

In 2017, there were an estimated 64,512 people living with acute myeloid leukaemia in the United States. The rate of new cases of AML was 4.3 per 100,000 men and women per year. The death rate was 2.8 per 100,000 men and women per year.
Official title:
Study start:
Gene editing method:
Type of edit:
Gene knock-out
Wilms tumour 1- WT1, T Cell Receptor Alpha Constant-TRAC, T Cell Receptor Beta Constant-TRBC ,
Delivery method:
No information - Ex-vivo
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy



NTLA-5001 seeks to treat acute myeloid leukAemia (AML) by engineering autologous T cell receptors (TCR) directed towards the Wilms’ Tumor 1 (WT1) antigen, an over-expressed protein that is often associated with AML and other cancers. We are using CRISPR-Cas9 to selectively and precisely edit the genes of patients’ immune cells, with the goal of improving their performance in treating oncological and immunological diseases. These improved immune cells should more effectively identify and attack cancerous cells upon re-infusion.

Last updated: Jul. 14, 2021
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