An illustration of a CRISPR-Cas9 gene editing protein cutting a DNA helix
Credit: Design Cells/Getty Images

Clinical-stage genome editing company Intellia Therapeutics announced Wednesday that it’s ex vivo investigational T cell receptor (TCR)-T cell therapy, NTLA-5001, for the treatment of acute myeloid leukemia (AML), has received orphan drug designation from the U.S. Food and Drug Administration (FDA).

Intellia develops of novel therapeutic candidates by applying CRISPR technology. It is developing both in vivo therapies—where it delivers CRISPR-based therapy intravenously—as well as ex vivo where it employs CRISPR to engineer cell therapies for the treatment of cancer and autoimmune diseases.

NTLA-5001 is an engineered autologous TCR-T cell therapy designed to target the Wilms’ Tumor 1 (WT-1) antigen, which is highly expressed in AML as well as other hematologic and solid tumors.  It is currently in a Phase I/IIa study in adults with persistent or recurrent AML who have previously received first-line therapy.

“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,” said John Leonard, M.D., president and CEO of Intellia “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.”

The FDA’s orphan drug designation provides incentives for drug developers that include tax credits for clinical testing and prescription drug user-fee exemptions. Orphan drug designation status is defined as those designed the diagnosis, treatment, or prevention of a diseases that affect fewer than 200,000 people in the U.S.

NTLA-5001 is the first wholly owned ex vivo therapeutic candidate for the company. It was developed using its proprietary cell engineering platform and uses a WT1-targeting TCR identified in collaboration with IRCCS Ospedale San Raffaele in Milan, Italy. Based on preclinical results, Cambridge, MA-based Intellia proprietary cell engineering platform has the potential to result in a pipeline of more efficacious and safer cell-based cancer therapies, according to the company.

The Phase I/IIa study of the candidate will evaluate the safety, tolerability, cell kinetics and anti-tumor activity of a single dose in adults who have detectable AML after having received standard first-line therapy. The study includes a dose escalation and expansion phase, with up to 54 total participants. The dose-escalation phase of the study includes two independent arms of up to three cohorts each: Arm 1 consists of adults with AML with lower disease burden, defined as those with less than 5% blasts in bone marrow, while Arm 2 consists of adults with AML with higher disease burden, defined as those with greater than or equal to 5% blasts in bone marrow. Once a dose is identified in each arm, two expansion cohorts will be opened for further safety assessment.

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