The FDA has accepted an NDA application for the ex vivo CRISPR genome editing candidate for the treatment of cancer.
NTLA-5001, an autologous T-cell receptor (TCR)-T cell therapy engineered to target the Wilm’s Tumor antigen, is intended to treat all genetic subtypes of acute myeloid leukemia (AML). It is Intellia’s first ex vivo candidate using its proprietary cell engineering process for the treatment of cancer to enter a clinical study.
“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. 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. 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. Our study is an important first step toward improving treatment for people living with this aggressive form of cancer. AML is the most common type of acute leukemia in adults, that, despite currently available treatments, has a five-year survival rate of less than 30 percent” said Intellia President and Chief Executive Officer John Leonard in a press release.
The planned phase I/IIa study will evaluate the safety, tolerability, cell kinetics and anti-tumor activity of a single dose of NTLA-5001 in adults with detectable AML who have already received standard first-line therapy, with a dose escalation and expansion phase.
Intellia has also submitted a regulatory application for NTLA-5001 in the UK.