Breakthrough: Intellia Achieved the First CRISPR-based In Vivo Editing of Bone Marrow

Yesterday, Intellia Therapeutics, a biotechnology company founded by Nobel laureate, Jennifer Doudna, presented preclinical proof of concept for CRISPR-based in vivo editing of bone marrow and hematopoietic stem cells (HSCs) in mice at Keystone eSymposium. 

Intellia’s modular in vivo editing platform employs non-viral LNP delivery with large cargo capacity, biodegradable / transient expression, low immunogenicity, redosing capability, scalable synthetic manufacturing and tunable tropism. 
Their preclinical experiment achieved durable, multidose editing of whole bone marrow and hematopoietic stem cells in mouse models at therapeutically relevant levels.

Read more on Intellia website: https://ir.intelliatx.com/news-releases/news-release-details/intellia-therapeutics-presents-preclinical-proof-concept-crispr