Pharmaceutical News 13 August 2025 - 22 December 2025

Intellia Therapeutics, Inc. is a clinical-stage biotechnology company at the forefront of CRISPRgene editing medicines. Headquartered in Cambridge, MA, Intellia was one of the first companies to harness CRISPR-Cas9 technology to develop potential in vivo therapies – treatments delivered directly into the body to edit genes inside patients’ cells. In fact, Intellia reported the first-ever clinical evidence that CRISPR could provide a therapeutic benefit in humans via in-body gene editing, showing in 2021 that a one-time IV infusion could drastically lower a disease-causing protein in patients ts2.tech. This pioneering achievement, accomplished with their transthyretin amyloidosis program, has put Intellia on the map as a trailblazer in genomic medicine. Today, Intellia’s mission is to develop curative treatments for severe diseases with few options, by editing or knocking out malfunctioning genes at the source. Its pipeline spans both in vivo therapies and ex vivo approaches. Intellia’s platform and know-how – based on Nobel Prize-winning science – have attracted top-tier collaborators. For example, Regeneron Pharmaceuticalspartnered with Intellia back in 2016, providing expertise to co-develop CRISPR treatments. This alliance has already borne fruit: Regeneron and Intellia’s lead candidate NTLA-2001 for ATTR amyloidosis emerged from their collaboration, and the partners even expanded the deal in