Intellia has shown that gene editing works in the body, and now the biotech is going to try to prove the same outside of the body.
The Cambridge, Massachusetts-based biotech’s application to kick start a trial for an ex vivo CRISPR genome editing cancer candidate has been accepted by the FDA. Intellia can now move forward with early human testing of NTLA-5001, an autologous T cell receptor (TCR)-T cell therapy that targets Wilms’ Tumor antigen in patients with acute myeloid leukemia (AML).
The biotech plans to start screening patients for a phase 1/2a study by the end of the year, according to a Thursday release. Up to 54 adult patients with persistent or recurrent AML who have previously received first-line therapy will be admitted to the study.
NTLA-5001 will be Intellia’s first ex vivo candidate to enter the clinic. The company and partner Regeneron reported first-in-human results for the in vivo therapy NTLA-2001 in patients with the rare disease transthyretin amyloidosis back in June.
In vivo therapies are administered directly to the patient to target cells and edit the genome from the inside. For ex vivo therapies, cells are removed from the body and modified before being transferred back to the patient. This method is also referred to as cell therapy. Intellia is using CRISPR/Cas9 to modify the cells once removed from the body.
The biotech is aiming to offer a safer cell-based cancer therapy for AML patients, who have about a five-year survival rate, despite available treatments.
After seeing results for NTLA-2001, this trial start for the AML therapy was the next eagerly-awaited news from Intellia. The acceptance of the application means the company is on its timeline of getting the study underway by year’s end.
Intellia is expected to name another candidate for development before the end of 2021 as well. Shares in the biotech were flat Thursday morning, coming off a fairly steep decline in recent weeks.