Intellia Therapeutics (NASDAQ:NTLA) (NASDAQ: NTLA) is a clinical‐stage biotechnology company focused on developing potentially curative genome editing therapies using the CRISPR/Cas9 platform. The company’s research spans both in vivo and ex vivo applications of CRISPR/Cas9, aiming to correct or disable disease‐causing genes with a single administration. Intellia’s lead in vivo program targets transthyretin amyloidosis (ATTR) by delivering CRISPR/Cas9 machinery directly to the liver, while additional preclinical efforts pursue treatments for hemophilia A, hereditary angioedema and other genetic disorders.
Beyond its in vivo pipeline, Intellia collaborates with strategic partners to extend the impact of its genome editing approach. The company maintains a collaboration and license agreement with Regeneron Pharmaceuticals to advance in vivo therapies for ophthalmic and other systemic diseases. In the ex vivo space, Intellia has entered into an alliance with Novartis to harness CRISPR/Cas9 for engineering allogeneic CAR-T cells for cancer immunotherapy, with the goal of producing “off‐the‐shelf” cell therapies.
Founded in 2014 as a spin-out from Caribou Biosciences, Intellia went public in 2016 and is headquartered in Cambridge, Massachusetts, with research operations in the San Francisco Bay Area and an innovation center in the United Kingdom. The company’s leadership team is led by President and Chief Executive Officer John Leonard, M.D., who brings extensive experience in oncology and cell therapy development, supported by a seasoned executive cadre in R&D, manufacturing and regulatory affairs.
Intellia serves a global patient population through its proprietary platform and collaborative network, with a focus on creating one‐time treatments that have the potential to transform the standard of care for patients with debilitating genetic diseases. The company continues to advance multiple programs through clinical and preclinical milestones toward regulatory filings and broader commercialization.
