Massachusetts-based biotechnology company Intellia Therapeutics announced Thursday that the U.S. Food and Drug Administration (FDA) has accepted its Investigational New Drug (IND) application for NTLA-5001, an experimental drug that is developed for treating acute myeloid leukemia (AML), according to BioSpace.
NTLA-5001 is Intellia’s first wholly-owned ex vivo CRISPR genome editing candidate.
Intellia CEO and President Dr. 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 utilizing Intellia’s proprietary engineering platform to treat patients with cancer.”
AML is a type of cancer that starts in the bone marrow, but it quickly moves into the blood, according to the American Cancer Society (ACS). It can sometimes spread to other parts of the body including the lymph nodes, liver, spleen, central nervous system (brain and spinal cord), and testicles.
Dr. Leonard told BioSpace, “Importantly, NTLA-5001 is engineered to target the Wilms’ Tumor (WT1) antigen which is overexpressed in an estimated 90% of AML patients regardless of genetic subtype.”
“In addition, WT1 is highly expressed across many hematological and solid tumor types, providing the significant potential to subsequently target a number of additional cancers,” he added.
The company now plans to move quickly and initiate patient screening for Phase I and II trials, evaluating the drug’s safety and efficacy in adults with persistent or recurrent AML.
“NTLA-5001 has been specifically engineered using Intellia’s differentiated and proprietary CRISPR/Cas9 genome editing technology to create a homogeneous T cell product with optimized cell health and function, which we hope will lead to better outcomes for patients as compared to current therapies,” Dr. Leonard explained.
Intellia is also involved in ex vivo programs that are aimed at developing potentially curative therapies for both cancer and autoimmune diseases, according to BioSpace.
In March 2020, Intellia announced that it received the FDA’s IND for its other lead ex vivo asset, called OTQ923, an investigational drug that is being studied for the treatment of sickle cell disease (SCD).
Intellia has teamed up with Novartis to make NTLA-5001.
Dr. Leonard said, “WT1, the target of NTLA-5001, is an intracellular antigen which would be inaccessible by current CAR-T approaches, further differentiating NTLA-5001 from other treatment options in development.” The article first appeared on BioSpace.
