Intellia's CRISPR-Engineered Cell Therapy Receives FDA Orphan Drug Tag For Bone Marrow Cancer

Intellia Therapeutics Inc's NTLA CRISPR-powered T cell therapy has received an FDA orphan drug designation.

• The autologous T cell receptor therapy, dubbed NTLA-5001, is being evaluated in a Phase 1/2a trial in acute myeloid leukemia, the white blood cell cancer that affects blood and bone marrow.
• A week ago, the company dosed the first patient in the NTLA-5001 Phase 1/2a trial in AML after receiving standard first-line therapy. The trial will enroll 54 patients.
• Read Next: Intellia Shares Updated Data On Gene Editing Durability In Rare Disease.
• 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 green light.
• Related content: Benzinga's Full FDA Calendar. 
• The designation is granted to therapeutics that aim to treat diseases impacting 200,000 or fewer people in the U.S.
• Price Action: NTLA shares closed 1.64% higher at $67.07 during after-hours trading on Wednesday.