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Bone marrow aspirate showing acute myeloid leukemia. Several blasts have Auer rods. Credit: VashiDonsk at English Wikipedia.

The US Food and Drug Administration (FDA) has accepted Intellia Therapeutics’ investigational new drug (IND) application to assess NTLA-5001 in a Phase 1/2a study for acute myeloid leukaemia (AML).

NTLA-5001 is the company’s first wholly-owned ex vivo CRISPR genome editing candidate for cancer treatment. The autologous T cell receptor (TCR)-T cell therapy is developed to target the Wilms’ Tumour (WT1) antigen to treat all genetic subtypes of AML.

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The Phase 1/2a study will evaluate NTLA-5001 in adults with persistent or recurrent AML who have previously received first-line therapy. It will include up to 54 participants and will feature a dose escalation and expansion phase.

The study will particularly assess the safety, tolerability, cell kinetics and anti-tumour activity of a single dose of NTLA-5001 among the participants. Intellia plans to start patient screening by year-end.

Intellia president and CEO John Leonard said: “The FDA’s acceptance of our IND for NTLA-5001 is an important milestone in our pursuit of developing advanced cell therapies utilising Intellia’s proprietary engineering platform to treat patients with cancer.

“NTLA-5001 is our first wholly owned ex vivo candidate to enter the clinic, and we expect to initiate this first-in-human study in adults with AML by year-end.

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“Our treatment strategy is to leverage CRISPR/Cas9 genome editing technology to create next-generation engineered immune cells with the potential to attack cancer cells more effectively and safely than previously developed cell therapies.”

A cancer of the blood and bone marrow, AML is the most common type of acute leukaemia in adults. The five-year overall survival rate for AML patients is currently less than 30%.

Earlier this month, BerGenBio concluded subject enrolment in a Phase Ib/II BGBC003 clinical trial of bemcentinib in AML and myelodysplastic syndrome (MDS) patients.

Cell & Gene Therapy Coverage on Clinical Trials Arena supported by Cytiva.

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