Intellia Therapeutics, Inc. products

Genome editing is efficient, precise and scalable. CRISPR/Cas9 genome editing can make permanent, precisely targeted changes in patients’ chromosomes and repair the underlying genetic mutation, whereas more traditional gene therapy typically involves introducing a non-permanent copy of a gene into patients’ cells. These attributes of CRISPR/Cas9 provide a significant therapeutic edge over other gene therapy and costly earlier-generation genome editing technologies, such as zinc finger nucleases and transcription activator-like effector nucleases. Intellia’s proprietary CRISPR/Cas9 system could potentially address diseases with a single dose of treatment because it permanently repairs the defective DNA. This represents a breakthrough improvement over current therapies, most of which require lifelong administration because they cannot correct underlying causes of the disease.

We plan to initiate patient screening for a first-in-human Phase 1 study of NTLA-5001, a WT1-directed TCR T cell therapy for the treatment of AML, by the end of 2021. Our first ex vivo development candidate seeks to treat acute myeloid leukemia (AML) by engineering autologous T cell receptors (TCR) directed towards the Wilms’ Tumor 1 (WT1) antigen, an over-expressed protein that is often associated with AML and other cancers. We have nominated NTLA-5001 as our initial development candidate for AML and expect to enter the clinic in 2021. Intellia’s lead WT1 TCR also has the potential to target WT1-positive solid tumors, such as ovarian cancer, glioblastoma, lung cancer and mesothelioma.

The first patient was dosed with our investigational genome editing treatment for transthyretin (ATTR) amyloidosis in November 2020. Our modular approach enables us to optimize the power and versatility of the CRISPR/Cas9 technology and, importantly, allows us to rapidly develop therapeutics for numerous diseases that currently have limited treatment options.