FDA Grants Orphan Drug Designation to T-Cell Receptor Therapy for AML

By Sabrina Ahle - Last Updated: February 2, 2023

The US Food and Drug Administration (FDA) has granted orphan drug designation to ex vivo investigational T-cell receptor therapy NTLA-5001 for the treatment of patients with acute myeloid leukemia (AML).

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NTLA-5001 is designed to target the Wilms’ tumor antigen expressed in AML as well as other blood and solid tumor cancers.

Currently, a phase I/IIa study is evaluating the safety, tolerability, cell kinetics, and antitumor activity of NTLA-5001 in adult patients with detectable AML following standard first-line therapy.

Source: Intellia Therapeutics press release, March 9, 2022.

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