Nothing improves a team’s chemistry like winning, and Intellia Therapeutics is on a winning streak in the CRISPR field. Intellia set the biotech world ablaze on June 26 when it released the first-ever clinical data showing the safety and efficacy of in vivo CRISPR genome editing in human patients. The continuing Phase I study interim data, presented at the Peripheral Nerve Society (PNS) Annual Meeting, showed that Intellia’s primary investigational in vivo candidate, NTLA-2001, significantly reduced the disease-causing protein following a single infusion.
Hereditary transthyretin (ATTR) amyloidosis is a rare, life-threatening protein misfolding disorder caused by specific transthyretin (TTR) gene mutation. NTLA-2001 is being developed in collaboration with Regeneron Pharmaceuticals to treat hereditary transthyretin (ATTR) amyloidosis.
An organization’s culture is established from the top down, as Intellia President and Chief Executive Officer John Leonard, M.D., displays daily. He brings a breadth of scientific and biopharmaceutical experience to the job, as well as a passion for making a difference for patients and the team that is striving to make that happen. That is a significant commitment for a company with over 350 employees. After a 22-year career at Abbott and AbbVie, which included helping the latter’s Humira® become the world’s best-selling medication, Leonard joined Intellia to help turn CRISPR/Cas9 technology into a therapeutic reality.