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news.Intellia Therapeutics has secured orphan drug designation from the US Food and Drug Administration (FDA) for its ex vivo investigational T cell receptor (TCR)-T cell therapy, NTLA-5001, to treat acute myeloid leukemia (AML).
The trial of the drug features a dose escalation and expansion phase. Credit: Hal Gatewood on Unsplash
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An autologous TCR-T cell treatment, NTLA-5001 is intended to target the Wilms’ Tumor (WT1) antigen, which is highly expressed in AML and several other hematologic and solid tumours.
In a Phase 1/2a trial, NTLA-5001 is being evaluated in adults with persistent or recurrent AML who have earlier given first-line treatment.
The trial features a dose escalation and expansion phase, with up to 54 total participants.
Intellia president and CEO John Leonard said: “The FDA’s decision to grant orphan drug designation for NTLA-5001 reflects the serious need for novel treatment options for people living with AML, a disease with notably poor long-term survival.
“As part of our full-spectrum genome editing strategy, we seek to leverage our proprietary CRISPR/Cas9-based platform to engineer differentiated cell therapies targeting cancers for which there are currently limited or no treatment options.
“We look forward to advancing our investigational TCR-T cell therapy, NTLA-5001, through the clinic in hopes of improving future treatment options for patients in need.”
AML is a cancer of the blood and bone marrow. It is a common type of acute leukemia in adults in the US, with an estimated new cases of over 20,000 in 2021.
