



Intellia Therapeutics is teaming up with Italy’s San Raffaele University and Research Hospital to develop engineered T-cell therapies for hard-to-treat cancers. The 3-year research collaboration, option, and license agreement will aim to leverage Intellia’s CRISPR/Cas9 genome-editing platform to generate improved T-cell therapies for both hematologic and solid tumors. The agreement includes options and licenses to key technologies developed at San Raffaele for producing engineered cell therapies.

The collaboration represents the first external partnership for Intellia’s eXtellia division, which it set up in January 2016 to focus on applying the CRISPR/Cas9 genome-editing platform for applications in immuno-oncology and autoimmune and inflammatory disease indications.

Chiara Bonini, M.D., head of San Raffaele’s Experimental Hematology Unit and deputy director of the Division of Immunology, Transplantation, and Infectious Diseases, will lead the scientific work at San Raffaele. “Through this collaboration, eXtellia aims to apply CRISPR/Cas9 genome editing in a multifaceted way to modulate the fundamental properties of engineered immune cells and amplify their anticancer properties far beyond current applications,” said Andrew Schiermeier, Ph.D., svp at eXtellia. “San Raffaele and Dr. Bonini are recognized globally as leaders in cell therapy and immuno-oncology, with excellent track records in translating innovative research into approved therapies.”

Intellia is developing a series of ex vivo and in vivo genome-editing programs in-house and in partnership with industry. The firm’s preclinical in vivo pipeline is focused on using lipid nanoparticles to target the liver and is headed by a Regeneron-partnered program against transthyretin amyloidosis (ATTR). The in vivo pipeline also includes programs targeting alpha-1 antitrypsin deficiency (AATD), hepatitis B virus (HBV), and inborn errors of metabolism (IEMs). Regeneron and Intellia inked their licensing and collaboration agreement to develop CRISPR-based products for up to 10 targets in 2016.

During late 2014, Intellia and Novartis inked a strategic collaboration and licensing agreement focused on developing ex vivo CRISPR/Cas9-based chimeric antigen receptor (CAR) T cells and hematopoietic stem cells (HSCs). Under the terms of the agreement, Novartis has exclusive rights to develop all programs focused on engineered CAR T cells. Novartis and Intellia are jointly developing multiple HSC programs, and Intellia will in addition develop its own proprietary internal HSC pipeline.



























