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Intellia Therapeutics Presents Preclinical Proof of Concept for CRISPR-based In Vivo Editing of Bone Marrow at Keystone eSymposium
- Demonstrates the promise of Intellia’s proprietary non-viral delivery system for in vivo genome editing of tissues outside the liver, with applications to
About this update from Intellia Therapeutics, Inc.
[{"type":"text","content":"- Demonstrates the promise of Intellia’s proprietary non-viral delivery system for in vivo genome editing of tissues outside the liver, with applications to inherited blood disorders such as sickle cell disease - Observed durable, multidose editing of whole bone marrow and hematopoietic stem cells in mouse models at therapeutically relevant levels CAMBRIDGE, Mass., March 10, 2021 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), today announced the presentation of preclinical data establishing proof-of-concept for non-viral genome editing of bone marrow and hematopoietic stem cells (HSCs) in mice. This represents the company’s first demonstration of systemic in vivo genome editing in tissue outside the liver using its proprietary non-viral delivery platform. Gene editing of HSCs in vivo via a non-viral delivery system offers the potential to transform the treatment of sickle cell disease (SCD) and other inherited blood disorders by overcoming the complexity and safety risks of ex vivo approaches. The company is presenting these data today at the Keystone eSymposium: Precision Engineering of the Genome, Epigenome and Transcriptome, being held virtually March 8-10, 2021. “This new data supports the possibility of delivering a safer solution to treat blood disorders, including sickle cell disease, by avoiding the need for bone marrow transplantation,” said President and Chief Executive Officer, John Leonard, M.D. “We’ve demonstrated we can expand our in vivo capabilities originally designed for liver applications to other tissues and achieve therapeutically meaningful levels of gene editing, reinforcing the promise of Intellia’s modular platform to transform the lives of people living with genetic diseases.” Presentation DetailsTitle: “In Vivo Genome Editing of Hematopoietic Stem and Progenitor Cells”Session: DeliveryDate and Time: March 10, 2021, 11:50 a.m. - 12:05 p.m. ETPresenting Author: Sean Burns M.D., senior director of Intellia’s Disease Biology and Pharmacology group The presentation can be found here, on the Scientific Publications & Presentations page of Intellia’s website. CRISPR/Cas9-based genome editing is well suited to the treatment of hereditary blood disorders, such as SCD. However, the current requirement for ex vivo manipulation of HSCs and toxic myeloablative transplantation regimens are significan...