Intellia Therapeutics Inc (NTLA) Ganando

Intellia Therapeutics, Inc. is a clinical stage genome editing company, which engages in the development of curative therapeutics using the CRISPR/Cas9 system. The company is headquartered in Cambridge, Massachusetts and currently employs 377 full-time employees. The company went IPO on 2016-05-06. CRISPR is a gene editing technology which is also sometimes referred to as CRISPR/Cas or CRISPR/Cas9 when referring to the use of CRISPR technology with the Cas9 enzyme. Its in vivo product candidates address genetic diseases by deploying its technologies, including CRISPR/Cas9 delivered by LNPs, as the therapy for diseases with high unmet need. Its lead in vivo product candidates, nexiguran ziclumeran (nex-z or NTLA-2001) for the treatment of transthyretin (ATTR) amyloidosis and NTLA-2002 for the treatment of hereditary angioedema (HAE), are the first CRISPR-based therapy candidates to be administered systemically, via intravenous (IV) infusion, for precision editing of a gene in a target tissue in humans. For its ex vivo product candidates, it applies its technologies to create engineered cell therapies to address immuno-oncology and autoimmune diseases.