Intellia's CRISPR-engineered cell therapy secures FDA orphan drug status after AML trial launch

Intellia Therapeutics' CRISPR-powered T cell therapy, which uses the gene editing method to reprogram a sample of the patient's immune cells, has received an orphan drug designation from the FDA just eight days after the biotech disclosed that its first patient had been dosed with the treatment. 

The autologous T cell receptor therapy, dubbed NTLA-5001, is being evaluated in a phase 1/2a clinical trial in patients with acute myeloid leukemia, the white blood cell cancer that affects blood and bone marrow. The FDA cleared the trial to begin enrollment back in September 2021, and the biotech plans to test the one-time therapy in 54 adults who have persistent or recurrent AML and have previously undergone first-line cancer treatments. 

With the orphan drug status, Intellia snags the potential for tax credits on clinical testing, plus fee exemptions and a seven-year marketing exclusivity should NTLA-5001 secure an FDA greenlight. The designation is granted to therapeutics that aim to treat diseases impacting 200,000 or fewer people in the U.S.

The Cambridge, Massachusetts biotech disclosed the news a minute after market closed Wednesday. Prior to the announcement, Intellia's shares ended trading up 3.68% to $65.99 apiece. The share price rose another 1.62% to $67.06 as of 4:15 p.m. ET. 

Intellia has already shown its gene editing works within in the body, through its in vivo therapy NTLA-2001 designed for patients with the rare disease transthyretin amyloidosis. The treatment reduced a known biomarker of the disease and maintained the effect in study participants who were followed anywhere from two months to 12 months after receiving the therapy. 

Now, it's hoping to prove effectiveness at treating AML through an ex vivo approach, where the cells are removed from the body and modified before being infused back into the patient.